er Excellency Dr. Delese Mimi Darko became the inaugural Director General (DG) of the African Medicines Agency (AMA) in October 2025. Dr. Darko engaged with Global Forum African Regional Editor Lorraine Danks on the Agency’s vision and strategic priorities, and how AMA could be a game-changer for access to medicines in Africa.

Lorraine Danks: It was wonderful to see you in June in Philadelphia for the DIA 2026 Global Annual Meeting and to hear you speak about the African Medicines Agency. What was the significance of AMA’s presence there, and what conversations stood out to you?

DG Dr. Darko: AMA’s presence at the DIA Global Annual Meeting is significant because it ensures that Africa is not only participating in global regulatory discussions but actively helping to shape them. For AMA, this is particularly important as we operationalize a continental regulatory system for Africa. It gives us an opportunity to communicate Africa’s regulatory priorities, strengthen relationships with other mature regulatory authorities and industry, and ensure that global product development strategies increasingly consider African regulatory requirements and the needs of African patients from an early stage.

It is also about positioning Africa as a pioneer in regulatory science and innovation, rather than simply a recipient of products developed elsewhere. Through platforms such as DIA conferences, AMA can promote regulatory convergence, reliance, and greater predictability for developers interested in Africa while ensuring that quality, safety, and efficacy remain at the center of access to medical products across the continent.

Lorraine Danks: What is your personal vision for AMA and what would success look like at the end of your tenure?

DG Dr. Darko: My vision is an AMA that is owned and driven by National Regulatory Authorities (NRAs) in Africa. AMA does not replace NRAs; we complement them and amplify their work. Together with Heads of NRAs, we have developed a clear vision, which is to enable a healthy, self-reliant Africa with quality, safe, and efficacious medical products supported by trusted, harmonized regulatory excellence.

That vision is one that is guiding our AMA Five-Year Strategic Plan. Our ambition is to become a credible, 100% ratified, World Health Organization (WHO)-Listed Authority with financial self-reliance by 2030. The public version of the five-year strategic plan is not yet available. It will be made available after approval through internal AMA processes.

Lorraine Danks: What are your immediate priorities for AMA?

DG Dr. Darko: I have previously spoken about three immediate priorities for AMA. The first is universal ratification of the AMA Treaty to bring all 55 AU Member States on board. Currently, 34 countries have ratified.

The second is capacity building for NRAs, which is the objective of AMA.

The third is to support local African manufacturing of medical products, an essential part of our health security and sovereignty.

Lorraine Danks: What are the one or two things you most want DIA members (many of whom are industry, regulatory, and clinical research professionals) to understand about AMA today?

DG Dr. Darko: AMA is building a coherent continental regulatory architecture that supports scientific innovation and provides DIA members with a more predictable and streamlined regulatory pathway to the African market, reducing the need to navigate 55 separate regulatory systems, requirements, and processes.

Lorraine Danks: A core promise of AMA is faster access to safe, effective, quality medicines. What specific mechanisms, such as joint reviews, reliance pathways, or harmonized standards, are already reducing timelines for African patients?

DG Dr. Darko: As stated previously, AMA is not starting from scratch. AMA’s foundation, the African Medicines Regulatory Harmonization (AMRH) program, has already successfully undertaken a continental listing of 12 products based on expression of interest. Through reliance on work done by Continental Technical Committees (TCs), the national approval timelines for some products have been reduced by more than 30%.

From Q4 this year, AMA will put out another expression of interest for manufacturers who want their products to be continentally listed.

We also have our Reference Agencies Network, which facilitates reliance amongst the ML3 (Maturity Level 3) agencies on the continent and also acts as a scientific backbone for AMA.

Lorraine Danks: How is AMA thinking about regulatory pathways for health product innovation, including support for African-led R&D and manufacturing, rather than just approving products developed elsewhere?

DG Dr. Darko: AMA is intentional about supporting innovation and manufacturing of medical products in Africa. The Regulatory Innovation and Manufacturing Readiness Hub, which was officially launched during the inaugural AMA Week in October 2026 in Kigali, Rwanda, is intended to help speed up access by removing regulatory uncertainties through early engagement.

This is complemented by the work of the AMA Capacity Strengthening Hub, also launched during AMA week, which coordinates capacity strengthening for regulatory authorities, including strengthening clinical trial reviews and monitoring. For example, AMA has put together a team of experts from ML3 regulatory agencies to provide a scientific opinion to an Egyptian company that is developing an Ebola vaccine.

DG Dr. Darko: The greatest opportunity is closing the gap between where these products are discovered and where they’re actually needed by embedding Africa earlier in the development pipeline, not just at the point of registration.

Lorraine Danks: How does AMA see its coordinating role differently from or similarly to the European Medicines Agency (EMA) model in Europe?

DG Dr. Darko: Like the EMA, AMA provides a platform for countries to pool scientific expertise, conduct joint assessments, and avoid duplicating high-quality regulatory work.

The key difference lies in the legal and institutional context. AMA does not replace Africa’s national regulatory authorities or take away their decision-making powers. It coordinates regulatory cooperation across the continent and issues rigorous scientific opinions that countries can rely on when making their own sovereign regulatory decisions.

Lorraine Danks: What does AMA add for a multinational sponsor that is already navigating multiple African NRAs; what’s the tangible efficiency gain?

DG Dr. Darko: One application, one rigorous scientific review, and access to multiple markets. Instead of submitting substantially dissimilar dossiers and responding to overlapping questions across numerous regulatory authorities, sponsors can benefit from coordinated assessment, shared scientific expertise, and more consistent requirements and timelines.

Lorraine Danks: How is AMA building trust with NRAs so that the agency is seen as complementary to rather than competing with national jurisdictions?

DG Dr. Darko: NRAs are the backbone of AMA’s mandate, so we work with them on every major strategy and build with them, not around them. A concrete example: In January, we convened the first Heads of NRAs and Regional Economic Communities (RECs) Forum under the theme “Building the African Medicines Agency Together.”

Bringing authority leadership into the room to shape the agency’s direction is how we ensure that there is ownership. It’s those same Heads of Agencies, increasingly, who carry the case for AMA to their own governments.

Lorraine Danks: AMA has drawn significant support from funding and technical partners (including the European Union) to speed up its operationalization. How is AMA planning for long-term, sustainable funding beyond this initial support?

DG Dr. Darko: AMA has a clearly articulated strategy to be financially self-sustaining through fees from its services as well as contributions from AMA States Parties as clarified in the AMA Five-Year Strategic Plan (to be issued at a future date).

The goal is to ensure that AMA, like the EMA, obtains its majority funds from fees charged to industry for continental listing, scientific advice, regulatory inspections, and variations.

Lorraine Danks: What should pharmaceutical companies, CROs, and other DIA members be doing now to prepare for a more harmonized African regulatory environment?

DG Dr. Darko: Engage early with the AMA Regulatory Innovation & Manufacturing Readiness Hub through rimr@au-ama.africa and align to harmonized standards. Sponsors who build dossiers with continental reliance in mind and coordinate with the Hub early have the greatest chance of success.

Lorraine Danks: What role do you see for patient advocacy groups and civil society in AMA’s work going forward?

DG Dr. Darko: It is central. Patients are why AMA exists, and advocates keep us honest about whether access is genuinely improving on the ground. We see them as partners who shape priorities and who tell the human stories that demonstrate the impact of our regulatory work. Those stories are ultimately how the public comes to trust an institution like ours.

Lorraine Danks: Is increasing the volume and frequency of clinical trials conducted in African countries one of your priorities, or do you leave that to the individual Member States?

DG Dr. Darko: Yes, increasing both the number and diversity of clinical trials conducted in Africa is a priority for AMA. Africa remains significantly under-represented in global clinical research, and the number of early-phase trials conducted on the continent is still very low, particularly beyond large public-health trials.

linical trial data integrity failures have become a critical threat to patient safety, regulatory trust, and the credibility of global drug development. Despite the finalized International Council for Harmonisation (ICH) E6(R3) guideline emphasizing rigorous data governance, recent high-profile cases show that fragmented digital workflows continue to create openings for error, inconsistency, and even fraud. AI and blockchain may offer a robust solution to this crucial challenge. 

A recent Alzheimer’s drug investigation revealed medically impossible clinical results, including cognitive improvements in placebo groups that contradict known disease progression. The consequences were severe: a $35 million trial rendered unusable and years of scientific progress lost. At the same time, investigative sites routinely log into six or more disconnected systems per trial, forcing manual data transcription across electronic data capture (EDC), clinical trial management systems (CTMS), and vendor portals. This manual duplication introduces an inherent 1%–4% transcription error rate, directly affecting regulatory submissions and patient safety.

These failures highlight a structural weakness in clinical research: Digital tools have proliferated, but the underlying infrastructure remains fragmented, inconsistent, and vulnerable. To protect data integrity, the industry needs to shift from reactive auditing to proactive, technology-enabled oversight.

ICH E6(R3) shifts clinical-trial quality from retrospective inspection toward prospective design. AI and permissioned blockchain may support that shift when deployed selectively for critical data and processes, but only when they are interoperable, validated, governed, privacy-preserving, and designed to reduce, not add to, site burden.

Persistent Fragmentation in Clinical Trial Systems

Investigative sites operate in hybrid environments where paper, Excel trackers, and disconnected digital systems coexist. Survey data from the Tufts Center for the Study of Drug Development (CSDD) confirms persistent reliance on paper-based workflows for source documentation and regulatory files. The Society for Clinical Research Sites (SCRS) reports severe technology overload, with inconsistent tech stacks driving administrative burden and burnout.

The TransCelerate 2025 Annual Report documents mounting operational complexity and slow progress toward interoperable digital protocols. Large pharmaceutical companies often require years to implement new systems due to validation requirements, global change management, and integration challenges, as noted by analyses from Deloitte, McKinsey, and IQVIA. These slow adoption cycles entrench legacy workflows and delay modernization.

This fragmentation forces sites to manually reconcile data across EDC, electronic Trial Master Files (eTMF), CTMS, safety systems, and electronic clinical outcome assessment (eCOA) platforms. Each manual step introduces risk, increases workload, and reduces the reliability of regulatory submissions.

Documented Data Integrity Failures

The Alzheimer’s case is not isolated. The valsartan hypertension trial at Kyoto Prefectural University of Medicine was retracted after investigators uncovered fabricated patient data and manipulated results, prompting institutional sanctions and broader scrutiny of clinical research practices. These events demonstrate that data fraud, while difficult to quantify, carries profound consequences for public trust and scientific progress.

Regulators have responded decisively. The FDA’s guidance on data integrity and compliance with drug CGMP and the EMA’s guideline on electronic clinical data reinforce the ALCOA+ principles, requiring data to be attributable, legible, contemporaneous, original, accurate, and complete. Both agencies emphasize full traceability across the data lifecycle.

Despite these expectations, fragmented digital ecosystems make traceability difficult to achieve. Without structural modernization, data integrity failures will continue to undermine clinical evidence.

Blockchain: Immutable, Transparent, and Audit-Ready

Blockchain introduces tamper-evident, cryptographically secured audit trails aligned with FDA 21 CFR Part 11, the FDA Decentralized Clinical Trials Guidance, and EMA expectations for trustworthy digital evidence. Once recorded, a blockchain transaction cannot be altered without detection. However, blockchain does not ensure that data entered are accurate or free from fraud. Suspect or erroneous records are typically not deleted; instead, corrective entries preserve a transparent audit trail while maintaining full traceability.

Key capabilities include:

• Immutable audit trails with time-stamped, cryptographically secured entries
• Decentralized validation across multiple nodes
• Smart contracts that automate compliance rules, trigger data queries, validate timestamps, or release site payments.

These features shift data integrity from reactive auditing to proactive enforcement. Blockchain ensures that every data point is traceable, tamper-evident, and audit-ready.

AI: Continuous Monitoring and Anomaly Detection

AI enables continuous monitoring, anomaly detection, and workflow automation across clinical operations. Applications include:

• Automated review of source documents and eTMF content
• Predictive models for operational risk
• Real-time anomaly detection in clinical and safety data
• Generative AI for drafting protocol sections and monitoring reports
• Agentic AI systems capable of executing multistep workflows autonomously.

A McKinsey analysis estimates that advanced agentic AI could reduce operational costs by 30%–50% when deployed effectively. Regulators are increasingly addressing AI in clinical research through the FDA’s Good Machine Learning Practice principles, EMA reflection papers on AI, and the EU AI Act, all emphasizing transparency, explainability, and risk management.

AI strengthens oversight by detecting medically implausible data, identifying operational anomalies, and reducing reliance on manual review.

Synergy Between AI and Blockchain for Data Integrity by Design

AI and blockchain can be combined to support a data-integrity-by-design architecture for clinical research. In this model, blockchain provides cryptographically secured data provenance, tamper-evident records, traceability, and auditable transaction histories. AI contributes continuous data analysis, automated validation, anomaly detection, and workflow support. Together, these technologies may strengthen the reliability, transparency, and operational efficiency of clinical-trial data processes.

This synergy enables:

• Near-real-time identification of anomalous or medically implausible data.
• Automated monitoring of predefined quality, protocol, and compliance controls.
• Reduction of manual data review and reconciliation.
• Transparent and auditable records for authorized stakeholders.
• Continuous verification of data authenticity and protocol adherence.

The result is a potential data-integrity-by-design architecture in which data provenance, validation, analysis, and oversight are more closely integrated throughout the clinical-trial lifecycle. Rather than replacing sponsor accountability, investigator responsibilities, human review, or validated quality systems, AI and blockchain can provide complementary infrastructure for reducing opportunities for undetected manipulation, strengthening data traceability, and supporting more credible regulatory-grade evidence.

Implementation Considerations

Adoption requires careful planning:

• System integration: Blockchain and AI should interface smoothly with legacy EDC, eTMF, CTMS, and safety systems.
• Regulatory acceptance: Sponsors should demonstrate validation, explainability, and audit readiness.
• Standards alignment: Adherence to CDISC SDTM/ADaM, HL7 FHIR, and ISO standards is vital to support structural interoperability.
• Change management: Sites and CROs require targeted training and deliberate workflow redesign.
• Feasibility: Early adoption may be most practical in decentralized trials, digital-first studies, or trials run by smaller sponsors operating without deeply entrenched legacy systems.

Broader Implications and the Human Angle

Data-integrity failures in clinical trials carry severe human and societal costs that extend far beyond regulatory noncompliance. When clinical data are compromised, vulnerable patient communities are exposed to ineffective or unsafe therapies, while public trust in medical research is deeply eroded. The profound impact of data anomalies is illustrated by a recently reported Alzheimer’s investigation, where data-tampering concerns compromised a major pathway of dementia research and delayed alternative therapeutic breakthroughs for families awaiting a cure.

Simultaneously, severe inefficiencies at the clinical site level directly destabilize trial operations. The logistical friction of disparate trackers drives massive workforce exhaustion, causing clinical research coordinator turnover rates to soar between 35% and 61%.

Smaller community-based research sites are disproportionately impacted by this overhead because they lack the capital to hire dedicated data management staff, ultimately bottlenecking industry efforts to improve diverse patient enrollment.

Across the broader healthcare ecosystem, these data inconsistencies severely drag down drug development timelines. Because regulators must pivot to exhaustive data validation and query resolution, patient access to life-saving therapies is routinely delayed. Transitioning to an interconnected data architecture powered by AI and blockchain provides an essential framework to establish data integrity by design. However, this adoption must be executed equitably to ensure that the operational and therapeutic benefits extend to patients, investigators, and sponsors alike.

A Coordinated Path Forward

A modern clinical trial architecture requires coordinated execution across four pillars:

1. Sponsors should launch pilot programs within 12–18 months using automated anomaly detection and immutable ledgers for high-risk endpoints such as eCOA, safety reporting, and digital protocols.
2. Regulators should accelerate harmonized frameworks defining validation, explainability, and audit expectations for agentic AI and distributed ledgers.
3. Clinical Sites should be protected from further technology fatigue through unified platform access, sponsor-funded training, and workflows that eliminate manual duplication.
4. Technology Vendors should prioritize interoperability, transparency, and out-of-the-box integration over proprietary silos.

By adopting AI and blockchain thoughtfully and equitably, the global clinical research enterprise can secure a transparent, automated future, accelerating delivery of therapies that patients and regulators can trust.

Conclusion

Fragmented clinical-trial systems are more than an administrative inconvenience: They can undermine data reliability, increase site burden, and erode patient and regulatory trust. Clinical-trial data integrity must therefore be designed into trial processes, not assessed only after problems emerge. AI and permissioned blockchain may strengthen selected controls for data provenance, traceability, anomaly detection, and workflow verification. Their value, however, will depend on disciplined, equitable implementation using clearly defined intended uses, validated and interoperable systems, robust governance, privacy protections, human oversight, and measurable reductions in site burden. This practical approach can help advance data integrity by design under ICH E6(R3) and support more trustworthy evidence for timely patient access to safe and effective therapies.

health authority in Japan publishes updated post-market surveillance guidance on a Tuesday afternoon. By Wednesday morning, an AI agent has detected the change, cross-referenced three affected products, scored the regulatory risk, and drafted and routed an impact memo to a senior reviewer just walking into their office in the US, fully traceable, start to finish. What once consumed two weeks of coordination now takes hours. This scenario is illustrative, but each step in it is already technically achievable, and it reflects the direction leading regulatory functions are actively pursuing.

Building the Foundation: Capability Maturity in Regulatory Affairs

Reaching a fully governed operating model in regulatory operations does not happen overnight. It follows the same logic of continuous improvement that ICH Q10 establishes for the pharmaceutical quality system: a capability that matures under management responsibility rather than arriving complete. Most organizations today sit somewhere between basic automation and augmented intelligence: They have moved beyond spreadsheets and email-based tracking but have not yet reached the point where their AI systems can independently prioritize updates, score risks, or recommend actions for human approval.

One useful way to frame this journey is through a five-stage capability maturity model: Stage 1, manual tracking; Stage 2, rule-based automation; Stage 3, GenAI-assisted review; Stage 4, AI-prioritized recommendations; and Stage 5, orchestrated execution with human signoff.

• Stage 1: Manual Tracking: Real-world benchmarking data illustrates just how wide the gap is at the earliest stage: When asked how quickly their team could identify a new regulation and execute a response plan, only about one in 10 regulatory leaders said they could do so within two days; most reported response times of a week or more, with over 40% of managers and specialists needing anywhere from one to four weeks, and nearly one in five taking longer than a month, according to RegASK’s 2026 State of Regulatory Affairs and Compliance Report. At this stage, teams still rely on offline, manual processes: tracking regulatory changes through inbox alerts, compiling impact memos by hand, and covering a limited set of jurisdictions with inconsistent accuracy.
• Stage 2: Rule-Based Automation: At the second stage, scripted data collection and rule-based routing bring modest improvements, such as faster first-pass triage and broader monitoring, though turnaround still lags well behind what regulatory volume now demands.
• Stage 3: GenAI-Assisted Review: The third stage introduces Generative AI into the workflow, enabling multilingual search, concise summaries, and materially faster, more consistent output than manual review alone.
• Stage 4: AI-Prioritized Recommendations: The real inflection point comes at the next stage, where AI systems begin to prioritize updates, score risk, and recommend actions for human approval, closing the gap between the “one in 10” who respond in days today and the rest of the field.
• Stage 5: Orchestrated Execution with Human Signoff: The ultimate destination is full orchestrated execution with human signoff, where AI agents handle end-to-end workflows and generate audit trails by default, turning today’s multiweek exception into the standard response timeframe.

Progressing through these stages also redefines what “performance” means for RA teams. Traditional key performance indicators (KPIs), such as hours worked per dossier, number of submissions filed, and revision rounds per document, reflect a sequential, labor-intensive model in which each step awaited manual approval before the next step could begin. Under that model, error detection was measured by how many rounds it took to correct a document rather than by how rarely errors occurred.

Agentic AI reframes this entirely. The new performance baseline is continuous compliance: KPIs shift to right-first-time approval rates, signal detection speed, demonstrably controlled compliance posture, and multijurisdictional filing velocity. Strategic impact is gauged by review cycles shortened and time-to-market acceleration. This KPI evolution is consistent with the GOVERN and MEASURE functions of the NIST AI Risk Management Framework, which focuses on measuring real outcomes and whether a system is fit for its purpose, rather than relying on activity-based measures.

To make this concrete, consider what each stage looks like for a single common task: responding to a labeling change issued by a health authority. Stages 1 through 3 reflect how most organizations operate today. Stage 4 is already within reach using available technology, while Stage 5 is technically achievable for organizations that have integrated their internal systems and put governance in place.

• At Stage 1: Someone on the team notices the change in an email newsletter days after publication, manually compares it against the company’s product portfolio, and drafts a Word document memo for the regulatory lead to review and act on. In many instances, however, no one on the team notices the change at all: Noncompliance may only surface when flagged by a health authority, a distributor, or an external stakeholder, at which point corrective action carries significantly higher regulatory and reputational risk.
• Stage 2: Scripted tools automatically collect the update from the regulatory authority and feed/route it to the relevant team via a workflow system; however, a human must still interpret the change and determine its impact on the product portfolio.
• Stage 3: A GenAI tool surfaces the change automatically, summarizes it in the local language, and flags potentially affected products, but a human still must orchestrate the downstream workflow.
• Stage 4: An AI system not only detects the change and flags affected products but also scores the regulatory risk, prioritizes the response relative to other pending updates, and drafts a recommended action plan for human review and approval before any downstream steps are taken.
• Stage 5: An AI agent detects the change within hours, runs an impact analysis against the full product portfolio across all registered markets, drafts the impact memo, routes it to the appropriate reviewer, and logs every step in an audit trail. The human’s role shifts from doing the work to approving it.

Reaching higher stages requires more than better software. It demands a clear operating model that defines and illustrates how agents coordinate, specialize, and interface with quality management, pharmacovigilance, and submission systems.

Multiagent Systems: The Coordination Layer of Regulatory Operations

If this maturity model describes the destination, multiagent architecture describes the vehicle. AI agents are goal-directed software systems designed to perceive, reason, and act with a degree of autonomy. In regulatory affairs, this means they monitor changes, analyze impacts, and take proactive action, shifting the paradigm from reactive to strategic.

Unlike traditional AI models that follow pre-set instructions and stop, Agentic AI works toward defined goals, breaks complex tasks into smaller steps, adapts its approach as conditions change, initiates actions across connected systems, and learns from the results. A well-designed multiagent system assigns specialized roles: A Research agent handles source ingestion and API connections to health authority databases; an Analytics agent clusters signals and scores risk; a Content agent drafts impact memos, SOP updates, and submission modules; and a Compliance agent ensures policy alignment and generates audit trails. An Orchestrator agent sits above them, decomposing high-level goals into tasks and delegating to the right specialist.

To illustrate how this works in practice, consider an illustrative scenario from global pharmaceutical operations: A health authority in Japan publishes an updated guidance document on post-market surveillance requirements for a specific drug class.

• Within minutes, the Research agent detects the publication through its continuous scan of the PMDA website, ingests the document, and classifies it by therapeutic area and regulatory domain.
• The Analytics agent cross-references the change against the organization’s product portfolio and flags three products with active registrations in Japan that are directly affected. It assigns a risk score based on the magnitude of the change and the compliance deadline.
• The Content agent then generates a draft impact memo summarizing the change, identifying the affected products, and recommending specific actions: updating the labeling (warning and precautions), revising the periodic safety update report timeline, and notifying the regional regulatory lead.
• The Compliance agent verifies that the proposed actions align with the company’s internal SOPs and logs the entire chain of reasoning for audit purposes.
• The Orchestrator packages everything and routes it to a senior regulatory professional for review and signoff.

Global Regulatory Monitoring also meets a constraint that architecture alone does not resolve. Monitoring public regulatory sources travels freely; what agents handle downstream does not. Registration files, internal assessments, and regulatory strategy are commercially confidential, and where agents extend into safety case processing, the data becomes personal and health data are subject to cross-border transfer restrictions. One workable pattern separates the layers: centralized monitoring and classification of public sources, with processing of regulated data kept in jurisdiction and only the assessment crossing the boundary. Cloud versus on premises then becomes a decision per data class, and one that has to be documented. The human in the loop maintains judgment, reviewing and evaluating whether the recommended actions are strategically sound, not assembling the information.

This architecture extends naturally to other high-value workflows. In submission management, agents can automate document generation, version control, and compliance checking, accelerating submission cycles.

In continuous compliance monitoring, agents track compliance posture around the clock, surface deviations as they arise, and route recommended corrective actions for human review. A regulatory illustration of this direction is the FDA’s consolidation of its legacy adverse event reporting systems—covering drugs and biologics, vaccines, veterinary products, and devices—into the Adverse Event Monitoring System (AEMS). FDA describes AEMS as strengthening safety surveillance through advanced case processing workflows, AI-based redaction and digitization tools, enhanced analytics, and comprehensive cross-product surveillance. Notably, the agency’s own signal evaluation cadence has not changed: Potential signals of serious risk continue to be published quarterly under section 505(k)(5) of the FD&C Act. The shift is in access rather than in judgment, and the implication for sponsors is that as fragmented safety data are consolidated and surfaced sooner, the expectation of how quickly an organization sees and acts on the same information rises with it. This “always-on” posture is not a new obligation but a continuous execution of an existing one: ICH E2E already requires a pharmacovigilance plan built on an evolving safety specification, EMA GVP Module VI governs the collection and submission of suspected adverse reaction reports, GVP Module IX governs signal management, and FDA post-marketing reporting of adverse drug experiences sits under 21 CFR §314.80. Faster, always-on execution, however, raises an unavoidable question: If agents act faster and more continuously than people can review, what makes their outputs trustworthy? That is the role of governance.

From GenAI to Agentic AI: Why Governance Is Non-Negotiable

As AI’s capability and governed execution increase, governance becomes the essential infrastructure that ensures trust, compliance, and auditability. Deploying Agentic AI in a regulated GxP environment is not possible without a robust governance framework. ICH E6(R3) already sets this expectation for clinical systems, requiring data governance and validation of computerized systems proportionate to risk. The question is not whether to govern, but how comprehensively to govern.

Governance also requires one boundary to be stated explicitly: which decisions remain human regardless of system capability. No regulator has published a list of decisions that may not be automated, but the boundary follows from where regulation assigns accountability to a named person—as EU pharmacovigilance law does for the Qualified Person responsible for pharmacovigilance, who may delegate tasks but not accountability. On that basis, benefit-risk determination, confirmation of a validated safety signal, final approved product information wording, and signoff on responses to health authorities cannot be delegated to a system, however capable it may be. Agents assemble the evidence and draft the wording; the determination remains a human act and will remain so unless legislation reassigns that accountability.

Consider what can happen without governance:

• An AI agent scanning regulatory databases ingests a corrupted source document and generates an impact assessment based on faulty data.
• Without data provenance tracking, no one can trace the error back to its origin. The flawed assessment flows downstream into a labeling update that passes through automated compliance checks, because the checks validate format, not source integrity.
• By the time a human reviewer catches the error, the organization has already initiated a submission to a health authority based on incorrect information.
• The cost is not just rework; it is regulatory credibility.

This kind of failure is preventable, but only with deliberate safeguards built into the system from the start. Ten governance pillars are critical for building a resilient AI ecosystem in Regulatory Affairs.

Data integrity forms the foundation of any resilient AI ecosystem:

1. Data provenance requires securing and documenting the origin of all data the AI consumes, maintaining a versioned registry of every regulatory intelligence source with update timestamps.
2. Audit trails must be immutable and human-readable, logging every step from signal detection to final approval, a requirement codified in FDA 21 CFR Part 11 for electronic records in regulated environments, and mirrored in the EU by EudraLex Volume 4, Annex 11 (Computerized Systems), which sets equivalent expectations for audit trails and data integrity in GMP-regulated computerized systems.
3. Role-based access enforces granular permissions, ensuring that only senior RA personnel can approve a final submission package.
4. Version control manages changes to AI models, prompts, and associated documents through validated systems, consistent with ISPE GAMP 5, FDA’s draft guidance on the use of AI in drug and biological product regulatory decision-making, and the draft EU GMP Annex 22 on Artificial Intelligence.

Operational safeguards are equally important.

5. Exception handling defines a clear process for routing AI-flagged anomalies to human reviewers: for instance, creating a dedicated queue for any regulatory change with a risk score above a defined threshold.
6. Validation and testing means rigorously checking AI outputs for accuracy, especially after model updates, including periodic adversarial testing of multilingual summarization capabilities, with validation effort proportionate to risk under ICH Q9(R1) as operationalized in ISPE GAMP 5, FDA’s AI credibility assessment guidance, and EU GMP Annex 22.
7. Bias and ethics monitoring watches for unintended consequences in patient-facing communications, as required under the EU AI Act for high-risk AI systems.
8. Incident response prepares teams to investigate and remediate if the system misses a critical safety alert.

Finally, lifecycle management ensures that these safeguards hold over time:

9. Lifecycle monitoring mandates periodic revalidation of AI models to ensure they remain fit for purpose as regulations and data sources evolve, consistent with the MANAGE function of the NIST AI Risk Management Framework and the ISO/IEC 42001 continual improvement cycle.
10. Third-party accountability extends these controls to systems the organization does not build. Suppliers of externally developed models should be assessed on risk and the assessment documented, consistent with ISPE GAMP 5, with contractual rights to notification of model changes before deployment. ICH Q10 establishes that responsibility for outsourced activities is not transferred with the work, and ICH E6(R3) states the same principle for clinical systems: Accountability for oversight remains with the sponsor regardless of what is delegated. Applied to agentic AI, this gives a defined chain: The RA lead owns the regulatory decision and answers for it to the health authority; the AI Governance Committee owns the system’s validation status and the conditions under which it may be used; and the vendor is accountable to the organization under contract, never in its place.

Implementing these safeguards is what separates a pilot from a production deployment. Organizations that treat governance as an afterthought will find their AI systems untrusted, unauditable, and ultimately unusable in regulated environments.

Governed Execution Ahead: How Agentic AI Redefines Compliance by 2028

Adoption is still early. A 2025 survey found that only 7% of senior leaders and 16% of operational teams are currently using Agentic AI. But the upward trajectory is unmistakable. Industry analysts project that by 2030, most organizations will be deploying AI agents across multiple business units, with high-capability, systematic execution deployments growing significantly by 2028, though human-in-the-loop oversight will remain the norm through at least 2027.

The specific implications for RA are substantial. Continuous global surveillance will replace manual tracking, allowing teams to capture regulatory updates more reliably and act with greater confidence. Automated document generation, version control, and compliance checking will shorten submission cycles. These trajectories align with the direction set by regulators themselves: FDA’s guidance on the use of AI to support regulatory decision-making for drug and biological products establishes a risk-based credibility assessment framework, EMA’s reflection paper on AI in the medicinal product lifecycle sets expectations for human oversight across development and post-authorization activities, and ICH Q9(R1) provides the risk-based governance principle underpinning both. Together they point to the same foundation for trusted deployment: human-centric design, risk-proportionate governance, and lifecycle management.

The window of opportunity is narrowing. Organizations that solve foundational challenges around data quality, internal system access, and governance in the near term will convert Agentic AI into a durable operational advantage. Those that delay face a three- to four-year catch-up period with compounding disadvantages. By 2027, early adopters operating at significantly higher efficiency will process substantially more regulatory changes with the same headcount, delivering sustained operational efficiencies.

Realizing these gains requires leadership clarity about the human role. The destination is governed execution at scale, guided by human judgment rather than replacing it. The OECD definition—mirrored in Article 3(1) of the EU AI Act — establishes autonomy as a gradient, which frames the question; the obligation comes from Article 14 of the EU AI Act, which requires high-risk systems to be designed for effective human oversight.

Empowering Humans with AI: The New Era of Regulatory Affairs

As agentic systems absorb monitoring, triage, and first-pass analysis, regulatory value shifts from output volume to decision quality. The professionals who thrive will be those who know what to delegate to agents, what to escalate to humans, and how to govern the boundary between the two. The underlying principle is not new: ICH E6(R3) already requires that accountability for data, and for oversight of the systems producing it, remains with the sponsor or investigator regardless of the tools used. This demands clear operating models, disciplined leadership, and governance infrastructure built before it is needed, not after. This evolution is not a distant future; it is happening now. Organizations adopting Agentic AI today are already reducing regulatory risk, improving audit readiness, and accelerating market access. The shift moves Regulatory Affairs from a heavy operational burden into a strategic capability; from a cost center to a source of competitive advantage.

For organizations ready to move beyond early wins, the path is practical. Start with two focused deployments in high-value, repetitive areas; global labeling change orchestration and Module 1 dossier assembly are strong candidates, as the first article in this series illustrated. Module 1 is a particularly good test case: Because it falls outside the harmonized CTD structure defined in ICH M4 and remains region-specific, its assembly carries exactly the jurisdictional variability that manual processes handle slowly and inconsistently. Within 12 months, stand up an AI Governance Committee with representatives from RA, Quality, Legal, and IT, embedding the governance safeguards outlined above and ensuring all AI-driven processes produce audit-ready evidence. Then scale using the maturity model as a roadmap, tracking progress with clear KPIs: for example, mean time to impact memo, missed-update rate, SLA compliance, first-pass accuracy, and audit-trail completeness.

The future of Regulatory Affairs belongs to teams that combine human expertise with AI-augmented intelligence, not to replace regulatory expertise, but to amplify it. Organizations that begin designing, validating, and governing these capabilities today will not simply keep pace with increasing regulatory complexity; they will convert it into an operational advantage. The time to build that foundation is now.

vidence shows increasingly that patient engagement can improve medicines development and regulatory decisions. The next challenge is to connect fragmented efforts and demonstrate the value of patient knowledge across healthcare systems.

The challenge is no longer simply to demonstrate that patient engagement matters. It is to understand how its benefits can be achieved more consistently, how existing healthcare ecosystems can work together, and what value is lost when decisions are made without sufficient patient input.

Connecting Ecosystems That Remain Fragmented

Healthcare has already established networks that bring stakeholders together. Organizations such as DIA and the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA) connect communities across medicines development, while Health Technology Assessment international (HTAi) and The Professional Society for Health Economics and Outcomes Research (ISPOR) advance collaboration around health technology assessment (HTA) and evidence of value. Each brings valuable expertise, but opportunities remain to align evidence needs, share learning, and ensure that patient knowledge informs decisions throughout the medicines lifecycle.

Yet connecting these communities with the wider healthcare decision-making system remains a challenge.

The regulatory ecosystem provides an important example. Regulators assess the evidence submitted to support medicine approval, but decisions about research priorities, study design, and evidence generation are made much earlier. HTA bodies and payers consider treatment value and access “after the fact” (i.e., after regulatory actions), sometimes requiring evidence that differs from what was generated for regulatory approval.

Patient knowledge has relevance throughout this process, but opportunities for patients to contribute remain disconnected. Evidence generated for one decision may not be suitable or accessible for another, while different organizations develop engagement approaches without consistently sharing what works.

More intentional coordination is needed, but collaboration cannot simply be assumed. Stakeholders operate under different mandates, incentives, and resource constraints. Progress depends on identifying shared problems, agreeing where patient knowledge can improve decisions, and developing practical ways to work across organizational boundaries.

Patient Focused Medicines Development (PFMD) seeks to address these gaps by connecting existing initiatives rather than creating parallel structures. Its Patient Engagement Synapse platform provides a global infrastructure for sharing knowledge and connecting stakeholders. PFMD’s collection of good practices and its annual Made with Patients Awards also help organizations learn from practical experience and adapt successful approaches to their own settings.

The next opportunity is to make this collective knowledge more actionable, connecting stakeholders not only to exchange experience but to improve how healthcare decisions are made.

Evidence That Patient Engagement Changes Outcomes

A growing evidence base demonstrates why earlier and more systematic patient involvement deserves attention.

Evidence compiled by PFMD for its forthcoming Value of Patient Engagement report highlights studies in which systematic early patient input was associated with 31% faster clinical trial recruitment, 1.6 fewer global protocol amendments, and a 46% relative reduction in participant dropout.

The regulatory impact is also becoming clearer. An analysis of European Medicines Agency (EMA) scientific advice between 2017 and 2020 found that approximately one in five scientific advice letters were modified following patient input. Furthermore, 71% of scientific advice coordinators recognized added value from patient involvement.

These findings illustrate that patient knowledge can influence not only the experience of participating in research, but also the evidence and advice that guide medicines development.

The economic implications are significant. In its 2026 investor whitepaper, The Patient Dividend, Gilde Healthcare (a Netherlands-based specialist investment firm focused on healthcare innovation, private equity, and climate solutions) presents patient engagement as an underutilized means of managing risk and creating value. The report highlights one case study in which patient advisory boards were associated with an average 10-week reduction in phase 3 trial duration, with estimated savings of $5.4 million per trial.

These findings come from different research settings and should not be interpreted as universally achievable returns. Nevertheless, they show why patient engagement warrants consideration as part of development strategy, regulatory preparedness, and investment decision-making.

Measuring the Value of Better Decisions

Understanding the value of patient engagement requires moving beyond counting engagement activities to examining what actually changes.

PFMD’s approach follows the connection between patient engagement, its influence on a decision, the resulting outcome, and the value created.

For clinical development, this could mean assessing whether patient input changes eligibility criteria or study endpoints, and subsequently examining recruitment, retention, and protocol amendments. In regulatory processes, it could involve documenting changes to scientific advice, the relevance of submitted evidence, and whether important patient considerations were addressed earlier or early enough.

Measuring the costs of poor-quality decision-making is equally important.

Gilde Healthcare describes patient engagement as an early warning mechanism: an opportunity to identify misalignment between product development assumptions and patients’ needs before course correction becomes expensive.

A treatment may demonstrate clinical efficacy yet prove difficult to use in everyday care. A trial may meet scientific requirements but struggle to recruit because its design places unacceptable demands on participants. Discovering these problems late can impose substantial costs on developers and healthcare systems.

Establishing the contribution of patient engagement therefore requires comparative research, consistent measures, and access to relevant data. Sponsors can provide operational and development data; regulators can contribute insights into assessment processes; and patient organizations can help establish whether decisions reflect patient priorities.

Importantly, faster or less costly decisions should not automatically be considered better decisions. Measures of efficiency must be assessed alongside scientific quality, patient relevance, and the consequences for access and health outcomes.

Building the Infrastructure for Evidence-Based Engagement

The evidence also reveals a gap between recognizing value and acting systematically to achieve it.

Gilde Healthcare identifies the lack of standardized investor-grade metrics and portfolio-level oversight as barriers to adoption. Existing engagement frameworks support good practice, but decision-makers responsible for allocating capital or overseeing development portfolios also need ways to assess engagement quality and track its contribution over time.

Similar challenges arise across regulatory and healthcare systems. Different stakeholders need evidence for different purposes, yet there is considerable value in developing shared methods and making existing knowledge easier to use.

The challenge is not simply whether patient input is recognized, but how consistently it can inform decisions across different institutions. Recent work by the Centre for Innovation in Regulatory Science (CIRS), examining patient involvement in regulatory and HTA decision-making, highlights opportunities to improve coordination, strengthen the visibility of patient contributions, and better align approaches across agencies.

Addressing these gaps requires more consistent methods for generating and using patient experience data, while recognizing that different decisions may call for different types of evidence.

Regulatory authorities increasingly recognize the relevance of patient experience data (PED). The US FDA has undertaken assessments of how PED contributes to regulatory decision-making. The European Medicines Agency (EMA) has also advanced work on methods for collecting and using PED in medicines development and assessment.

PFMD’s Integrated Framework for Generating Patient Experience Intelligence, which remains in development, aims to support this transition by helping stakeholders generate more consistent, fit-for-purpose patient evidence and strengthen its use in decision-making. Building on the Global PED Navigator, this work offers an opportunity to connect the methods used to generate patient knowledge with the decisions that knowledge needs to inform.

It also responds to a broader challenge in digital health. As patient-generated data become more widely available, healthcare systems need trusted governance and better connections between data sources, evidence requirements, and decision-making processes. Greater volumes of data alone will not guarantee that patients’ needs are better understood.

The Organisation for Economic Co-operation and Development (OECD) has highlighted the importance of interoperability, trustworthy data governance, and individuals’ ability to access and use their health information. Its research on people-centered digital health systems reinforces the need to connect digital infrastructure with meaningful participation.

Making Patient Knowledge Part of Sustainable Healthcare

The implications extend beyond the efficiency of medicines development. Health systems face growing pressure to allocate resources effectively while improving access and responding to increasingly complex patient needs.

Patient engagement can contribute by identifying priorities earlier, avoiding unnecessary development activities, and helping decision-makers understand whether innovations will deliver value in real-world care.

For investors, this creates opportunities to assess development risk more accurately. For regulators and HTA bodies, it can improve the relevance of evidence. For patient communities, the ultimate benefit is greater influence over decisions that affect their health.

Achieving these benefits at scale requires going beyond examples of good practice. It requires sustained collaboration, shared infrastructure, and a clearer understanding of where patient knowledge contributes most.

Over the coming years, the priority should be to connect existing ecosystems around common evidence needs, strengthen methods for measuring impact, and make patient engagement/involvement routine while important decisions can still be influenced.

The measure of progress will not be how many patients are consulted, but how consistently their knowledge leads to better decisions, and how clearly healthcare systems can demonstrate the value of those decisions.

generic tablet has the same active ingredient wherever it is sold. The study used to prove that it performs like the original brand-name drug, though, has not been the same from one market to the next.

Testing the same product two or three ways costs money, and every gap between one agency’s expectations and another’s opens the door to a mistake.

ICH M13A, Bioequivalence for Immediate-Release Solid Oral Dosage Forms, is meant to reduce this opportunity for error. It sets a common standard for the bioequivalence of immediate-release solid oral dosage forms, and regulators have started to apply it.

What M13A actually standardizes

M13A reached Step 4 of the ICH process in July 2024, the point at which ICH’s regulatory members formally adopt a guideline. Each region then implements it separately, under its own procedures.

FDA issued its own final guidance adopting M13A in October 2024, and EMA adopted it as a scientific guideline effective 25 January 2025.

Alongside the guideline, the FDA issued a large set of revised draft product-specific guidances to match. A product-specific guidance is FDA’s recommendation on how to design the bioequivalence study for one named drug.

M13A supersedes the parts of the EMA guideline on the investigation of bioequivalence that deal with bioequivalence study considerations and data analysis for a nonreplicate study design. In a nonreplicate design, no volunteer takes the same product twice. M13A applies to immediate-release solid oral dosage forms taken by mouth, such as tablets, capsules, and granules or powders for suspension, and covers both new and generic products, in development and after approval.

The change that most study teams will notice first has to do with food. A bioequivalence study is often run twice: once with the volunteer fasting and once after a meal, because food can change how fast and how completely a drug is absorbed. That expectation has weighed more heavily on FDA submissions than on EMA ones, since EMA’s earlier bioequivalence guideline already treated fasting as the default and called for a fed study only in specified cases. M13A makes the single-study default the harmonized position. For most products one study is now enough: under fasting conditions in the general case, or under fed conditions where the label calls for the product to be taken with food to improve absorption or reduce variability. Both studies are kept for high-risk products, where the drug substance combined with a complex formulation or manufacturing process makes it more likely that the product will behave differently depending on conditions in the gut.

The guideline settles much of the rest of the study design and analysis: which pharmacokinetic measures matter most (peak concentration and total exposure), and the familiar 90% confidence interval of 80.00% to 125.00% for the test-to-comparator ratio of those measures. It also permits designs that use more than one comparator, so that a single study can serve several regions.

M13A also standardizes how the comparator is handled once it has been chosen. It sets expectations for selecting the comparator batch, and it asks that the test and comparator batches match to within 5% in the amount of drug they actually contain, unless a difference can be justified. Comparator selection itself remains governed by each region’s own rules, which M13A does not override, and the multicomparator design is the initial step it takes to ease that. In practice, a sponsor filing in several regions may still need a comparator strategy for each jurisdiction.

Some things are left out on purpose. Highly variable drugs, narrow therapeutic index drugs, and complex study designs sit outside M13A’s scope and go to a later guideline (M13C). Highly variable drugs are those whose blood levels vary widely from one dose to the next in the same person. Narrow therapeutic index drugs are those where the margin between an effective dose and a harmful one is small. Two related topics involve biowaivers, meaning permission to skip a new in-human study for a product strength on the basis of other evidence. Waivers for a product’s additional strengths, the doses beyond the one already tested, are the subject of M13B; waivers based on a drug’s solubility and permeability, its biopharmaceutics classification, stay under ICH M9, Biopharmaceutics Classification System-based Biowaivers.

ICH adopted M13B at Step 4 on August 4, 2026, after a public consultation that ran through 2025. EMA published the final guideline on September 21, 2026, but it does not take effect until March 17, 2027. As of October 2026, M13B is still a draft guidance at FDA, so it does not yet govern a filing in either region. M13C was initiated in February 2025, and the concept paper supplement endorsed by the ICH Management Committee projects Step 2, the draft stage, in June 2027 and Step 4 in February 2029. As a result, M13A does not yet have the answer for a highly variable drug, or an additional-strength biowaiver; those questions sit with M13C and M13B.

Why it matters, and where it stops

For a company filing across markets, the payoff is direct, though how much it is worth depends on the product and on how many markets are in play. One well-built study can increasingly cover submissions across ICH regions as each puts M13A into effect; Health Canada adopted it for submissions filed after December 27, 2025, following FDA and EMA. That can avoid duplicate studies and save money and time. Consider a product that used to need a fasting-only study for one agency and a fasting-plus-fed study for another. Under one common standard, that kind of mismatch is much less likely to surface halfway through review.

The new standard has two real limits, and both are easy to underestimate.

First, it does not cover every product. M13A is written for immediate-release forms, and it hands highly variable and narrow therapeutic index drugs to M13C. Extended-release and complex generics still lean on product-specific guidances that differ by region.

FDA’s own numbers show why that matters. In an Office of Generic Drugs analysis presented in April 2025, 157 abbreviated applications across eight extended-release products, submitted through the end of 2024, drew 224 first-cycle bioequivalence deficiencies, concentrated in dissolution (37%), bioanalysis (35%), pharmacokinetics (17%), and formulation (11%). Extended-release is the space M13A does not reach, and it is where a great deal of design judgment still sits.

Second, even for immediate-release products, a common standard only helps teams that deliberately design to it. The categories in that analysis are about how a study is designed and run rather than about the dosage form, and the same design and analysis gaps recur from one submission to the next:

• A dissolution method that cannot tell a good batch from a bad one, so a weak product slips through.
• Bioanalytical records that arrive without the raw data needed to verify the numbers.
• Subjects dropped from the analysis with no reason on record.

These are scientific and analytical failures, not clerical ones. A dissolution method that cannot discriminate has not been developed properly, bioanalytical records without the raw data cannot be checked, and an analysis whose exclusions are unexplained cannot be assessed at all. Harmonization does not fix any of them, and it raises the cost of a design mistake, because the same error can now sink a study in several markets at once.

Nor does the harmonized guideline change who reviews the result. Each region still conducts its own assessment, under its own product-specific guidances and its own inspection and facility expectations. Even with a common study standard, these reviews stay separate. Whether an application clears its first cycle still pivots on the strength of the science in the dossier and on how completely it is presented.

For patients, the argument is more supply, sooner. When one solid study travels, a lower-cost generic can reach people in more countries without waiting for a separate study in each. For manufacturers in emerging or newly harmonizing markets, M13A lowers a real barrier to entering highly regulated markets, provided the study is designed to the harmonized standard from the outset. Adoption outside ICH regions remains variable, however, and local requirements still decide whether a study is accepted there.

What regulatory teams can do now

A few practical steps:

• Read and compare M13A against current bioequivalence study templates, and reset the default protocol designs that most design teams start from, including the move to a single study for most lower-risk products.
• Separate immediate-release work from extended-release and complex product work in planning, and focus on the relevant product-specific guidances for anything that M13A does not cover.
• Follow M13B and M13C, so that biowaiver and complex-design study plans do not rest on ground that is still shifting.
• Turn the recurring deficiency areas (dissolution discrimination, complete bioanalytical data, and justified subject exclusions) into a checklist that the study team reviews and approves during protocol design, before the protocol is finalized.
• When a product, such as a highly variable drug or a narrow therapeutic index drug, falls outside M13A’s core, expect region-specific requirements and settle the design with the relevant agency before the study begins.

For immediate-release products, M13A brings a stack of market-by-market study designs much closer to a single one, though regional product-specific guidances and local requirements still apply. Generics developers stand to save real time and money. The gain goes mostly to study teams that build to the standard from the first protocol, since retrofitting it onto a study already designed captures much less; and ultimately to patients, who can access these products sooner, with equivalence demonstrated to the same standard wherever the product is sold.

Supplemental Resources

• Federal Register notice of availability, M13A final guidance, October 31, 2024
• EMA, ICH guideline M13B, Step 5, EMA/CHMP/ICH/85092/2025 (published September 21, 2026; legal effective date March 17, 2027)
• ICH, Supplement to the M13 Concept Paper covering M13C, endorsed by the ICH Management Committee January 27, 2025 (source for the projected M13C Step 2 and Step 4 dates)

he history of multinational pharmaceutical regulation is often told as a progression toward harmonization: from national requirements to shared standards, from isolated assessments to regulatory reliance, and ultimately toward more integrated systems capable of delivering safe, effective, quality medicines to patients faster.

Yet this narrative overlooks an essential reality: Harmonization is neither a single destination nor a uniform process. It is shaped by history, politics, institutional maturity, public-health priorities, and the sovereign responsibilities of individual countries.

Europe: From Harmonization Pioneer to Regulatory Reinvention

Europe represents perhaps the most mature example of regional pharmaceutical regulatory integration. Europe’s journey from the European Economic Community (EEC) in 1958 through the European Community (EC) to today’s European Union (EU) reflects decades of progressively deeper economic, political, and regulatory integration. Political commitment, scientific collaboration, and legislative development culminated in the establishment of the European Medicines Agency (EMA) in 1995. The European regulatory network, combining the EMA, national competent authorities, and the European Commission, created an enduring framework for pooling expertise, sharing assessments, and reaching coordinated regulatory decisions.

Since 1995, working with national regulatory authorities and the European Commission, the EMA has contributed to the centralized authorization of more than 1,900 human medicinal products across the EU. This illustrates the enduring value of shared scientific expertise, coordinated assessments, and mutual trust within Europe’s regulatory network.

Europe’s achievement was never simply the creation of a supranational agency. It was the development of a culture in which regulators learned to overcome centuries of tension and come to trust one another’s scientific judgments while operating within a common legal framework. That culture helped establish Europe as a global leader in regulatory harmonization and an influential participant in the development of international standards, notably ICH.

Yet institutional maturity does not confer immunity from change.

In her July 2026 Sir Alasdair Breckenridge Lecture, EMA Executive Director Emer Cooke challenged regulators to fundamentally reimagine their role. Rapid advances in cell and gene therapies, personalized medicines, artificial intelligence, and novel clinical-trial methodologies demand more than incremental procedural improvements. Regulators must evolve from being predominantly gatekeepers into proactive enablers of responsible innovation, without compromising scientific independence or patient protection.

ED Cooke’s vision embraces agility, digital transformation, earlier engagement with developers, and deeper international cooperation. Her memorable six C’s—communication, cooperation, coordination, collaboration, convergence, and coalitions—capture a philosophy increasingly relevant well beyond Europe.

The lesson is compelling: Even the world’s most sophisticated regulatory networks must continue to learn. Harmonization is not an accomplishment to preserve unchanged, but a capability to continually renew.

Africa: Building Continental Ambition on National Strength

Africa’s regulatory journey reflects a different history and a visionary ambition.

With 55 African Union Member States, diverse legal systems, considerable differences in regulatory capacity, and substantial unmet medical needs, Africa faces challenges that are fundamentally different from those encountered during European integration.

Rather than replicating the EMA, Africa has developed its own institutional response: the African Medicines Agency (AMA), established through an African Union Treaty adopted in 2019. Building on years of African Medicines Regulatory Harmonization initiatives, AMA is now transitioning from political aspiration to operational reality.

It is noteworthy that the EU and EMA have provided substantial financial assistance, scientific expertise, and technical support to the AMA, while recognizing full African leadership and ownership of its regulatory responsibilities, operations, and decisions.

The inaugural AMA Week, held in Kigali in October 2026, provides a particularly powerful illustration of this transition. The launch of the Regulatory Innovation and Manufacturing Readiness Hub and the inauguration of the Technical Committees underscore an ambition extending beyond product assessment to encompass African innovation, local manufacturing, supply chain integrity, drug safety monitoring and enforcement, regulatory capacity, and health sovereignty.

In an interview published in this issue of Global Forum, AMA’s inaugural Director General, Dr. Delese Mimi Darko, articulates a crucial distinction: AMA does not replace national regulatory authorities; it complements and amplifies their work.

This is not a minor institutional detail. It is the foundation of Africa’s regulatory model, which was driven, “piloted,” and informed by the seminal work of the East African Community (EAC) Medicines Regulatory Harmonization (MRH) initiative.

AMA seeks to coordinate scientific expertise, facilitate joint assessments, strengthen regulatory capabilities, and generate rigorous scientific opinions on which national authorities can rely while retaining their decision-making responsibilities.

There is already evidence that collaboration can deliver tangible results. According to DG Darko, the African Medicines Regulatory Harmonization program has supported the continental listing of 12 products, with reliance on shared technical assessments reducing national approval timelines by more than 30% for some products.

Her priorities, such as universal Treaty ratification, capacity building, and support for local manufacturing, reflect a distinctly African understanding of regulatory excellence. With 34 countries having ratified the Treaty at the time of her interview, the task remains substantial. Nevertheless, AMA’s emergence demonstrates what determined leadership and continental solidarity can accomplish.

Africa is not merely importing regulatory models; it is actively adapting global experience to African realities and positioning itself to contribute original solutions to global regulatory science.

Latin America: Progress Through Pragmatic Cooperation

As a third distinct regulatory framework, Latin America is characterized by established national institutions, significant scientific capabilities, diverse legislative traditions, and persistent fragmentation.

Unlike Europe, Latin America has not developed a comparable overarching medicines agency. Unlike Africa, it has not established a continent-wide treaty-based institution with AMA’s mandate. Instead, regulatory convergence has evolved through national initiatives, regional networks, international partnerships, and increasingly sophisticated discussions about mutual reliance.

This approach may appear hesitant, even frustratingly incremental. But it would be a mistake to confuse institutional fragmentation with an absence of progress.

As highlighted in this issue’s article from the 2026 DIA Latin America Annual Meeting in Mexico City, regulators and stakeholders throughout the region are advancing risk-based regulation, digitalization, pharmacovigilance modernization, artificial intelligence, and pragmatic reliance.

However, the distinction between adopting reliance policies and implementing them effectively is especially revealing. An assessment involving 10 Latin American countries found that all had a legal basis for reliance (despite lacking a multinational framework like the EU and the African Union), but only half had established standard operating procedures to operationalize it.

This gap illustrates the challenge of converting regulatory goodwill into routine institutional practice.

Nevertheless, authorities such as Brazil’s ANVISA and Mexico’s COFEPRIS are advancing regulatory modernization, while the Pan American Health Organization continues to support convergence and collaboration across national systems.

Latin America’s opportunity may lie not in creating another EMA or AMA (although this cannot be ruled out in the fullness of time) but in building a sufficiently connected ecosystem of sovereign regulators capable of sharing scientific work, reducing duplication, and deploying limited resources where local expertise contributes most.

Reliance, properly understood, is not an abdication of responsibility; it’s an expression of confidence in the scientific competence of others.

Different Frameworks, Shared Principles

Comparing these three regions reveals differences in institutional architecture, but striking similarities in purpose:

• Europe demonstrates the power of sustained integration and the necessity of continuous reinvention.
• Africa shows how political commitment and shared ambition can transform regulatory fragmentation into coordinated continental action.
• Latin America reflects the potential of pragmatic cooperation among sovereign authorities, even without a single political framework (there is no “Latin American Union”) or regional institution (there is no “Latin American Medicines Agency”).

None offers a universal blueprint. Each has lessons to share—and to learn.

Their common denominator is trust: trust in scientific evidence, in professional competence and rigor, in institutional integrity, and in the legitimacy of different jurisdictions. Trust makes reliance possible, leadership makes cooperation durable, and innovation makes regulation responsive. And patients give these efforts their ultimate purpose.

Yet maintaining trust depends on the everyday work, dialogue, constructive dissent, and ultimate collaboration of legislators and regulators to convert industry innovation into patient benefit. In Europe, the CHMP has exemplified the successful application of these crucial elements for decades.

Regulatory convergence should never become a contest over whose model is most advanced. Nor should harmonization be mistaken for uniformity. Respecting local epidemiology, healthcare systems, resources, and legal responsibilities is not an obstacle to global cooperation; it is essential to its success. The most promising future is therefore neither one global regulator nor hundreds of disconnected national authorities. It is a worldwide community of regulators capable of learning from one another, sharing scientific expertise, and exercising their responsibilities with confidence and mutual respect.

Europe, Africa, and Latin America are demonstrating, each in its own way, that the future of pharmaceutical regulation will be built through collaboration rather than competition, and through mutual reliance rather than institutional imitation.

When I entered the pharmaceutical industry world in the early 1980s, there was no regulatory harmonization. Each country had its own legal and regulatory requirements and, importantly for regulatory professionals, its own format for Marketing Authorization Applications (paper only, and tons of it). The sheer inefficiency of multiple overlapping and duplicative nonclinical and clinical test requirements and application formats was recognized and addressed by ICH (more than 80 Guidelines to date, and the work continues). The very existence of the eCTD today is a testament to the value of multinational visionary collaboration, and a massive advance in quality and speed compared to the “bad old days.”

Three hundred million years ago, the continent we now know as North America was contiguous with Europe, Africa, and South America. They all existed as a single continent called Pangea. It is a stretch to consider the medicines frameworks already in place or evolving today as a gradual movement towards a human pangea focused on the public health—or is it? We may never have a World Medicines Agency (which may or may not be a good idea in any case). But the advances (scientific, political, legislative, regulatory, and more) of the past four decades and expected in the near and distant future are bringing the regions of the world closer together.

Continents are learning globally, acting locally—and, above all, striving to serve their patients. And that’s what matters.

ive vaccine candidates against Bundibugyo ebolavirus are funded. Two are in human trials. Around 620,000 doses of the University of Oxford’s candidate have already been manufactured and are waiting at the Serum Institute of India. What no one has yet addressed is how a successful vaccine will be approved and registered in the countries that need it.

Everything is Moving Except Regulation

In June, the Coalition for Epidemic Preparedness Innovations (CEPI) committed more than $60 million, with cofunding from the US Biomedical Advanced Research and Development Authority (BARDA), to four vaccine candidates developed by the International AIDS Vaccine Initiative (IAVI), Moderna, the University of Oxford with the Serum Institute of India, and Public Health Vaccines; furthermore, up to $16.5 million was announced in August to support Minapharm Group’s candidate. CEPI’s goal is to advance at least two of them through emergency authorization. Oxford’s ChAdOx1 BDBV candidate became the first Bundibugyo vaccine ever tested in humans in July, and Moderna’s mRNA-1469 entered its own first-in-human trial in August.

On the demand side, Gavi, the Vaccine Alliance, has made up to $50 million available, including $40 million in pull financing, which creates a guaranteed buyer for doses ready to be deployed if trials succeed.

Push funding, pull funding, and at-risk manufacturing are in place. However, there has been minimal public discourse on regulatory preparation. This missing piece can and should be put in place in advance, and the registration of the first Ebola vaccine offers a starting point. After the ERVEBO vaccine was licensed in 2019, WHO wrote down what worked and what did not. This record—published in the WHO’s Weekly Epidemiological Record (WER) in August 2020, seven months into the COVID-19 pandemic—outlines the lessons learned by both WHO and Merck in approving ERVEBO and includes recommendations for future public health emergency responses; this record is still waiting to be used in the current Ebola outbreak.

The ERVEBO Precedent

ERVEBO, Merck’s rVSV-ZEBOV vaccine against Zaire ebolavirus, became the first licensed Ebola vaccine after being developed in the shadow of the 2014-2016 West African epidemic, when no licensed vaccine existed. The European Medicines Agency (EMA) granted conditional marketing authorization in November 2019, WHO pre-qualified the vaccine within days, and five African countries, including the DRC, registered it within 90 days. WHO later published a lessons-learned review of how ERVEBO’s pre-qualification and registration were expedited. As well as detailing the successes, the document diagnoses the causes of delay and proposes solutions to be adopted in advance of future outbreaks—such as today’s.

What Worked, and What Did Not

While imperfect, the registration of ERVEBO broke records as the fastest vaccine to be pre-qualified by the WHO at the time. Global coordination was a primary driver of this fast pace. In May 2019, WHO published a roadmap for licensing ERVEBO in African countries. It coordinated the manufacturer, the WHO pre-qualification team, the EMA, national regulators, and the African Vaccine Regulatory Forum (AVAREF) around a single dossier, enabling near-parallel reviews. Early regulator engagement also played a part. ERVEBO entered the EMA’s PRIME (PRIority MEdicines) scheme and received US Food and Drug Administration Breakthrough Therapy Designation nearly three years before the EMA submission, thereby opening scientific dialogue and expedited review long before the data matured.

The same review is candid about what failed. The platform for information exchange was insufficiently defined, with Merck unexpectedly having to provide the same dossier to African regulators multiple times in different formats due to some regulators’ unfamiliarity with password-protected USB flash drives. There were delays in the completion of confidentiality agreements and the nomination of representative reviewers to attend meetings. Legal frameworks were also missing, since not all affected countries had emergency-use provisions that allowed accelerated regulatory decisions.

What Has Changed Since 2019

On paper, much of this has been fixed. The African Medicines Agency (AMA) is now being operationalized, and on June 3, it joined forces with the EMA and African national regulators on the outbreak response, the first public health emergency in which the two agencies have collaborated. On safety, AfriVigilance launched on June 29, 2026, as a continent-wide platform that connects safety evidence across a product’s entire lifecycle, from clinical development to real-world use, answering the WHO review’s call for robust pharmacovigilance. On legal frameworks, the recently revised African Union’s Model Law on Medical Products Regulation is being promoted across the continent to embed emergency use authorization, facilitated approval pathways, and reliance (the practice of informing national decisions with trusted assessments by other regulators). Uganda has moved: Its National Drug and Health Products Authority (NDHPA) Act, which was assented to in April 2026, replaces a framework dating back to 1993. It places a statutory duty on the new Authority to rely on the decisions of other regulators and of regional agencies, and creates a route to authorize unregistered products in an emergency or during a pandemic. The Act, however, awaits a commencement instrument, and the detail of any emergency pathway is left to regulations yet to be made. The DRC has no equivalent reform in place.

There has also been progress towards improving information exchange. In December 2024, CEPI committed $19.1 million to the nonprofit Accumulus Synergy as part of a global regulatory preparedness framework aimed at public health emergencies. The cloud-based platform lets a sponsor share one standardized dossier with many regulators at once, so they can review the same data, questions, and answers simultaneously and in real time. This is precisely the type of global platform for centralized posting and submission the WHO review called for.

The Countries Least Ready May Wait Longest

WHO’s proposed solutions rest on conditionals. A centralized, cloud-based platform helps only where reviewers have reliable internet connections and appropriate training. Reliance on a reference authority’s assessment works only where national law permits a regulator to act on it. Furthermore, the two countries affected by the current outbreak are asymmetrically prepared. Uganda, which declared its own outbreak over on July 28, has a National Drug Authority on a WHO-supported journey toward maturity level 3 (out of 4) of WHO’s global benchmarking scale, and its national quality control laboratory was recently benchmarked at maturity level 4. The DRC, which carries more than 99% of the caseload, has a young regulator, the Congolese Pharmaceutical Regulatory Authority (ACOREP), that is still assuming responsibilities from the former Directorate of Pharmacy and Medicines and has not reached maturity level 3.

Preparedness built around innovative solutions will help the countries already best positioned to use them. Without a clear picture of what each national regulatory authority can and cannot do, no one will notice which countries are being left behind until doses arrive.

What Must Happen Now

WHO, through AVAREF and in coordination with the AMA, should assess regulatory preparedness to authorize a Bundibugyo vaccine in the DRC, Uganda, and at-risk neighboring countries, covering review capacity and expertise, internet connectivity, and supported submission formats. Funders should resource these organizations to do this, since capacity is constrained. The output should be an updated African licensing roadmap, copublished by WHO and the AMA, before pivotal trial data arrive. The primary lesson of the ERVEBO vaccine is that the roadmap came before the data. The doses are ready. The question is whether the regulatory system that receives them will be.

sia represents approximately 60% of the world’s population, and more than 5 million people in Asia died from cancer in 2022. Although numerous Asia-led regional and local initiatives aim to reduce the cancer burden in the region, access to cancer care still varies considerably between and within countries. Furthermore, due to major socio-economic disparities and differences in healthcare systems across the world, a cancer diagnosis can still represent the start of a traumatic journey and sometimes even a death sentence in various Asian countries.

Are we listening to cancer patients?

This article shares discussions among three Asian cancer patients and patient representatives from highly different settings, who offered their perspectives, lived experiences, and aspirations for tomorrow’s normal in Asia at the most recent DIA Japan Annual Meeting.

Asian Cancer Patient Realities and Aspirations

Three Asian patients from Cambodia, Singapore, and Japan shared concrete examples of their lived experiences, ongoing activities, and aspirations with stakeholders including clinicians, researchers, industry representatives, and regulators.

The speaker from Cambodia, a long-term breast cancer survivor, highlighted many challenges to cancer patient care, including ineffective communication between patients and physicians, distrust in healthcare systems, the substantial risks of self-medication, and the questionable reliance on traditional herbal treatments in some rural areas. This speaker also explained that limited availability of medications, equipment, specialists, and technical capacities actually forces some low-income patients to borrow money or sell property to access treatment at private clinics or abroad.

A brain tumor survivor from Singapore who is also a patient representative illustrated the important role of patient support groups. She founded an association which serves as a community for brain tumor patients and provides various services including monthly support group meetings, awareness walks, and education talks and excursions. Other key support activities include psychosocial services and financial assistance programs for Brain Tumour Society Singapore (BTSS) members such as welfare grants for needy families and tuition grants for children with brain tumors.

Patients who receive a cancer diagnosis often enter an unfamiliar and frightening world where access to accurate information becomes empowering. The Japanese patient representative established a patient support group for diffuse gastric cancer with her late husband who passed away from this disease. Her group activities include cancer awareness events and active collaboration with healthcare professionals through webinars for patients, patient involvement in clinical guidelines and development of plain language summaries of research findings, and international patient exchanges aimed at broadening perspectives and possibilities.

Strengthening Education for Patients and Their Advocates/Representatives

These three patients then explored their aspirations in panel discussion together with a Japanese physician highly involved in patient engagement, as well as the session chairs, one regulator and one researcher/patient representative. Several common priorities emerged from these conversations.

One important priority was the strengthening of education not only for patients and patient representatives, but also for healthcare professionals. Patients must better understand their disease and treatment to participate more actively in their own care, while healthcare professionals and patient representatives would also benefit from more informed mutual understanding and communication.

Because many cancer patients primarily access information that is available within their own country, patient representatives play a key role in providing timely, relevant, understandable information to patients and families. The Japanese representative also pointed out that patients may be particularly vulnerable at the time of diagnosis to seeking “information wrapped in sweet words,” choosing information based more on emotions than reliability. At the same time, education of healthcare professionals regarding the importance of patient engagement remains essential to support this two-directional relationship.

The panel discussion also underscored the importance of closer collaboration between healthcare professionals and patient representatives to better align efforts toward patient needs and more responsive healthcare systems. The experience of the patient from Singapore (mentioned above) illustrated how involving patient representatives in public hospital settings and decision-making processes can contribute to practical institutional changes, including improvement in workflows and patient support systems, by placing patients’ and caregivers’ needs at the center of service design and decision-making. Mutual understanding, collaboration, and safe space for dialogue between stakeholders is critical throughout these interactions.

Despite major disparities for patients across the region, such as access to innovative medicines, clinical trials, and specialist cancer care, common priorities emerged during the session, including improving access to trustworthy information, strengthening cancer awareness and education, and fostering collaboration among stakeholders. Over time, such connections will support regional collaboration to address challenges particularly relevant to Asia, including inequalities in access to care, linguistic and cultural diversity, urban-rural gaps, and the rapidly evolving cancer burden across the region.

Sustainable Cross-Border Dialogue and Patient Engagement in Asia

Another session topic illustrated a concrete approach to fostering constructive cross-border dialogue in Asia: bringing various stakeholders together within the same space. Beyond sharing experiences, these exchanges also create opportunities to openly discuss unmet needs, practical barriers, and possible solutions across the region’s highly different healthcare environments.

Such interactions may be particularly important in Asia, where neighboring countries face a growing cancer burden despite major differences in healthcare resources, infrastructure, and patient engagement, while often sharing cultural and social context that extend across borders. Creating environments where people with lived experience can speak openly may therefore help stakeholders better understand realities that are not always visible through clinical, regulatory, or policy perspectives.

These interactions also demonstrated that patient engagement is not limited only to supportive roles, but may contribute to shaping research priorities, strengthening access to clinical trials, improving patient-centered information and communication, and enhancing the responsiveness of healthcare systems and cancer policies.

Self-Knowledge through Learning from Others

One way to transform these shared learnings into sustainable regional progress is to develop similar sharing platforms within regional and international scientific meetings, clinical trial networks, healthcare institutions, and public health initiatives. Beyond raising awareness of the disease and importance of patient advocacy, these efforts may help ensure that lived experience is more meaningfully integrated into cancer policies, research priorities, clinical trial design, communication strategies, and healthcare delivery across the region. Documentation and sharing of key cooperative efforts, practical examples, and lessons learned may also help avoid duplication and support continuity across countries and stakeholders.

The Japanese representative reflected during the panel discussion that by learning from other countries, we also learn more about our own. Future initiatives could build on efforts led by organizations such as the World Health Organization (WHO) and the Union for International Cancer Control (UICC) while adapting them to Asian realities and disparities. Meaningful patient engagement in Asia and beyond will likely continue to grow through local champions, shared long-term commitments, and continuous opportunities for regional and international exchanges.

ustralia’s cell and gene therapy (CGT) sector is at an inflection point. With strong foundations and growing momentum, Australia has a unique opportunity to become a regional leader in CGT through coordinated national efforts, while expanding patient access to these life-saving medicines throughout the country, as discussed at a recent Summit (see sidebar).

Australia is approximately three years behind the US FDA in granting marketing authorization for patient access to approved CGT products. According to this same Medicines Australia report, only eight Therapeutic Goods Administration (TGA)-licensed good manufacturing practice (GMP) production facilities exist in the nation of Australia, and 83% of its industry stakeholders expect workforce talent shortages to negatively impact their strategies within the next five years. Finally, while Australia has established several initiatives to support collaboration and advancement of the CGT sector, such as the Solutions for Manufacturing Advanced Regenerative Therapies Cooperative Research Centre (SMART CRC), opportunities remain to strengthen national coordination through a unified, whole-of-sector strategy supported by dedicated resources and sustained engagement across government, industry, academia, healthcare, and patient communities.

Behind each of these previous figures is a patient: often a child or young adult with a rare genetic condition, or a person living with a blood cancer who has run out of options, together with a family that may spend months navigating referral and travel while waiting for a solution.

Patient-centricity served as the guiding principle and foundation for the following recommendations, each of which was tested against these three important questions raised at the Summit:

1. Does it shorten the time to patient access?
2. Does it reduce the burden of receiving treatment?
3. Does it improve the equity of who is treated?

It is wise to remember that access is not only a regulatory and reimbursement question; it is also a question of geography, knowledge dissemination, and getting support before, during, and after treatment.

Currently, Australia has a strong base in the life sciences sector with world-class universities, an efficient clinical trial regulatory pathway, and a 43.5% R&D tax incentive that attracts international investors and global companies. In 2024, 185 active CGT clinical trials were registered in the country. In October 2025, the $238 million SMART CRC launched. That movement, combined with growing investment in RNA manufacturing infrastructure, signals a real momentum for regional CGT development.

In comparison, other regions are moving faster.

• The UK Cell and Gene Therapy Catapult has upskilled over 3,800 people and leveraged more than £850 million in investment at Stevenage.
• The US FDA has launched its Plausible Mechanism Pathway for individualized genome-editing therapies, and the FDA Rare Disease Framework Initiative announced in October 2026 also overlaps into CGT development. In addition, the US Advanced Research Projects Agency for Health (ARPA-H) is investing hundreds of millions of dollars in distributed precision-medicine manufacturing.
• The EU Biotech Act is streamlining development timelines for Advanced Therapy Medicinal Products (ATMPs) across Europe. This is all happening while Asia Pacific has surpassed North America in the number of active CGT clinical trials, driven primarily by the explosion of CGT development in China.

Summit participants acknowledged that additional work is needed to streamline the development and approval processes for CGTs in Australia and further agreed that global regulatory divergence imposes duplicative costs due to CMC misalignment, among other issues. In fact, the lack of standardization around CMC requirements is the leading cause of late-stage regulatory delays globally as well.

Cell and gene therapies represent a fundamentally different class of medicine. Unlike a small-molecule tablet or a monoclonal antibody produced in bulk and dispensed from a pharmacy shelf, a CGT product may be extracted from the patient’s own blood, genetically modified in a specialized manufacturing facility, transported under strict cold-chain conditions, and infused back into that same patient within a defined treatment window. Every step of this process (from apheresis to conditioning chemotherapy, from vein-to-vein logistics to post-infusion monitoring) is time-critical, technically complex, and clinically demanding. The consequences of these differences ripple through the entire clinical development, regulatory approval, and access system. That is why clinical trials for CGTs cannot rely on the same endpoints, follow-up periods, or trial designs as those used for conventional therapeutics. Additionally, health technology assessment (HTA) bodies face a valuation problem because it is hard to model the cost-effectiveness of a one-time curative treatment over a lifetime horizon when follow-up data may extend only two or three years instead of the required 15 years of long-term follow-up required after the administration of human gene therapy by many global regulatory agencies.

In Australia, hospitals must be specially accredited to deliver CGT therapies. Patients in regions or states without treatment centers face barriers of distance and logistics. Australia’s Clinical Trial Notification (CTN) scheme provides a streamlined regulatory pathway for eligible clinical trials. Unlike the US FDA’s Investigational New Drug (IND) process, which generally includes a 30-day waiting period before clinical investigations may begin, the CTN scheme does not require a separate scientific evaluation by the TGA before trial initiation.

This is why many cell and gene therapy companies, especially new gene editing companies and in vivo CAR-T companies, have now come to Australia. The HOYA/University of Melbourne 2024 CGT Landscape Report has identified 22 Australian CGT therapy developers, with CAR-T representing 31% of the modality mix, alongside over 20 supply-chain, service, and product-provider entities. Approximately 80% of trials are phase 1 or phase 2, with relatively few progressing to phase 3 or commercial development domestically; this translation gap defines much of the sector’s current challenge. Most of the programs originate overseas and license their intellectual property offshore, with Australia participating as a site rather than as the originating clinical trial developer.

CGT manufacturing is where Australia’s ambitions run directly into several structural constraints. The eight national, TGA-licensed GMP manufacturing facilities focus primarily on T cells, mesenchymal stem cells, cord blood, plasmid DNA, autologous chondrocytes, and peripheral blood stem cells. However, all these remain at a much smaller scale compared to the Catapult facilities in Stevenage and Braintree (UK), ACTRIS in Singapore, or OmniaBio in Canada.

The 2025 Deloitte/Cell and Gene Catalyst Australian Cell and Gene Therapies Innovation Investment Report found that 58% of respondents believe the lack of onshore manufacturing will negatively impact their CGT development strategies over the next five years, with 83% citing supply chain disruptions as a significant challenge, and 70% viewing manufacturing challenges (including capacity, scalability, and raw material costs) as a serious obstacle. As previously mentioned, the SMART CRC launched in October 2025, representing the most significant national investment in advancing CGT capability to date. With $238 million over 10 years and 63 partners spanning universities, CDMOs, supply-chain companies, and end-user healthcare organizations, its four research programs (biomanufacturing, bioseparation, biomaterials, and clinical enablement) are designed to bridge the gap between university innovation and scalable GMP-ready clinical manufacturing.

Australia’s health technology assessment and reimbursement system was designed decades ago to handle the evaluation and approval of conventional medicines. It was not designed for one-time, curative, hospital-administered, autologous therapies that cost hundreds of thousands of dollars per patient and generate durable health benefits over the patient’s lifetime. The result is a system that tries to apply tools inadequately and aims at addressing the wrong problem, resulting in delays in patient access. Of 72 CGT products approved globally, only 14 have been approved in Australia in early 2026. Of those 14, only 12 have managed to get the Medical Services Advisory Committee recommendation. Twenty percent get TGA approval, and even fewer seem to reach Australian patients.

For patients, these delays carry real costs: extended waits while their disease progresses, difficult travel logistics to access a small number of accredited centers, time away from work, school, and family, and related challenges, resulting in significant out-of-pocket expenses. Due to these challenges, patients with rare diseases and patients living in regional and remote areas are the most likely to be left behind by scientific advances. Access to a CGT should not depend on a patient’s zip code, or on how well their family can navigate a complex system.

The regulatory environment for CGTs is changing rapidly all around the globe, with major agencies moving at different speeds and in different directions with the aim of creating more efficient pathways for advanced therapies. In this global landscape, it is important for developers to select indications with clear unmet medical needs, so HTA bodies and regulators both engage with them early. It is also important to maintain a regulatory mindset (with application to batch-to-batch variability, potency assays, GMP-adjacent activities, etc.) from day one. It is also useful to get parallel advice from regulators and HTA bodies, and to use expedited regulatory pathways wherever possible, for an eligible CGT product. Qualification is then nearly certain.

In the Asia-Pacific region, China is now clearly the leader in CGT clinical trials. Meanwhile, Singapore is positioning itself as the APAC regulatory and manufacturing hub: The Advanced Cell Therapy and Research Institute, Singapore (ACTRIS) facility, the National Health Innovation Centre, and Singapore’s RNA National Initiative collectively present a coherent regional strategy that Australia has not yet matched. Since many clinical trials are now coming out of China and looking to run in Australia to get data from more diverse patient populations, the Australian ecosystem needs to be equipped with an appropriately trained workforce.

CGT manufacturing, clinical delivery, and regulatory navigation each require specialized skills that take years to develop. Skilled workforce development has emerged from a background concern into a first-order constraint on everything else: Manufacturing scale-up, clinical trial expansion, regulatory engagement, and patient access all ultimately depend on people with the right expertise. The SMART CRC’s Future Workforce Program and the University of Melbourne’s CGT training offerings are the right starting points, but synergy is needed between these stakeholder groups and industry to help create a recognizable career pathway for people entering the CGT sector.

Prioritized Recommendations

Summit discussions developed six recommendations for government officials, regulatory policy experts, health system administrators, and CGT industry leaders in Australia. Each is aimed at bringing approved therapies to Australian patients sooner and more equitably. In order of priority:

1. Establish a national CGT coordinating body with patient representation. The Australian government should mandate and resource a whole-of-sector body, linked to the proposed National Life Sciences Strategy, that brings together regulators, HTA bodies, clinicians, manufacturers, and researchers, with patient and carer representatives, to generate a national CGT roadmap, creating shared priorities and accountability for progress. In one recent report, 91% of respondents advocated for a nationally coordinated approach to strengthen Australia’s CGT capabilities.
2. Align regulation and HTA requirements as early as possible to reduce duplication. TGA and HTA bodies should offer joint or parallel scientific advice and run parallel review processes wherever possible, so that evidence generated for approval also serves reimbursement decisions. Greater reliance on assessments by trusted overseas regulators, and closer international alignment on CMC requirements, would reduce the duplicative costs that currently drive late-stage delays.
3. Adopt funding and evaluation models built for one-time therapies. Where long-term data are immature, managed-entry and outcomes-based agreements, with payment linked to durable benefit and staged over time, can allow earlier access while evidence accrues. These should be supported by a national CGT patient registry combined with a real-world evidence (RWE) framework, so that outcomes are collected once and used by regulators, payers, and clinicians alike.
4. Bring treatment closer to patients. A hub-and-spoke model should link accredited treatment centers with regional hospitals for referral, apheresis, monitoring, and follow-up, supported by telehealth and shared-care protocols. Funded travel and accommodation support for patients and carers should be a standard part of access, not an afterthought.
5. Embed the patient voice in decisions. Patients and caregivers should sit on HTA committees, regulatory advisory forums, and trial design groups, and patient-relevant outcomes (including quality of life and burden of care) should be captured in trials and registries. Clear, accessible information will help patients and families make more informed decisions on their options and pathways.
6. Build sovereign, scalable manufacturing. Targeted investment should build on the SMART CRC (see above) to expand onshore GMP capacity and support pilots of point-of-care and distributed manufacturing. Shorter, more resilient supply chains would reduce vein-to-vein times and dependence on offshore supply.

Additionally, a few distinct steps could be taken in Australia toward strengthening the CGT workforce. Because people are the rate-limiting step for every priority listed above, workforce development should be treated as core infrastructure rather than a background activity.

1. Develop a national CGT workforce strategy and skills framework. This would ideally be codesigned with employers, universities, regulators, and clinicians to define the competencies needed across manufacturing, clinical delivery, regulatory science, and HTA, and map existing offerings, including the SMART CRC Future Workforce Program and university training, to meet them.
2. Create recognized credentials and visible career pathways. Nationally recognized microcredentials, traineeships, and apprenticeships in GMP manufacturing and CGT operations, aligned to employer expectations, would make the sector an attractive and portable career choice.
3. Train the clinical delivery workforce, especially outside the major centers. Structured training for hematologists, apheresis and infusion nurses, pharmacists, coordinators, and allied health staff, with support for regional hospitals joining shared-care networks, is essential for safe and equitable delivery. Patient navigator roles should be recognized as part of the CGT care team.
4. Grow regulatory and HTA capability through exchange. Secondments and joint training among regulators, HTA bodies, industry, and academia, and international exchange with partners such as DIA, the UK Cell and Gene Therapy Catapult, and APAC regulators, would build the expertise needed to assess novel therapies efficiently. The Catapult’s record of upskilling thousands of people shows what a coordinated national effort can achieve.
5. Attract and retain scarce skills. Migration and mobility settings, research-to-industry fellowships, and partnerships across Asia Pacific should help fill critical gaps while domestic training capacity grows.

Summary and Next Steps

In summary, the CGT sector in Australia could benefit from organizing over three horizons:

• Within the next 12 months: Convene a national coordinating body with patient representation, publish the national CGT skills map, and agree on a pilot for providing joint TGA and HTA advice.
• Within two to three years: Stakeholders convene to establish the first hub-and-spoke treatment networks, pilot outcomes-based funding agreements for selected therapies, and launch a national CGT patient registry.
• Within five years: National establishment of scalable onshore manufacturing and a recognized national CGT career pathway, with a measurable narrowing of the gap between overseas and Australian patient access.

As the CGT field evolves quickly, in vivo CAR-T, gene editing, and individualized genome-editing therapies will test regulatory, reimbursement, and delivery systems around the world. The systems that are designed around patients, and flexible enough to prepare for new modalities, will be better positioned to handle the advancements. It is suggested that the National CGT Strategy for Australia could be successful with tracking progress in the field locally through a set of patient-centered indicators: the time between first approval overseas and access in Australia, the proportion of eligible patients treated outside the capital-city centers, patient-reported experience and outcomes, and the size of the trained CGT workforce.

Toward a Better Tomorrow

Australia’s CGT sector currently has significant local scientific talent, the clinical trial infrastructure, the regulatory credibility, and the manufacturing investment to be a meaningful player in the global CGT landscape. However, the translation gap from early-phase clinical trials to commercial-scale manufacturing, and then to approved and reimbursed patient access, is real, documented, and widening relative to peers.

The absence of a national coordinating body means that the sector’s collective capacity is less than the sum of its parts.

The HTA system’s fragmentation means that three years separate Australian patients from the same therapies their counterparts in the US or Europe receive.

The workforce shortage means that all ambitions for scale, whether in manufacturing, clinical delivery, or regulatory science, are constrained by the supply of people who know how to do the work.

Since innovation alone is not going to bring about improved patient access, it is important to be able to deliver innovative therapies equitably in Australia, across a very large and diverse territory for the country. Placing patients at the center, and acting on the recommendations above, offers a practical way to turn that shared understanding into measurable progress.

The CGT Summit did not resolve these challenges; instead, it created a shared understanding of the landscape that has been built and allowed multiple stakeholders to discuss the gaps and suggest important future priorities. These insights provide the basis for coordinated action, which can be shared within Australia through the LifeSciences Australia membership as well as globally through DIA’s stakeholders and their scientific work.

lobal drug development is becoming more international, data-driven, and scientifically complex, while regulators and industry face growing pressure to accelerate access without compromising quality or patient safety. Harmonization is more than alignment of technical requirements. Its broader implications include reducing duplication and improving efficiency to support quicker patient access.

The 2026 DIA ICH Outlook Conference in Shanghai provided a timely view into how regulatory harmonization is evolving in practice against this backdrop.

China is playing an increasingly active role in this process, not only through implementation of international guidance, but also through wider technical dialogue and standards development. For regulators, standards-setting bodies, and companies, the implication is that the next phase of harmonization will depend not only on formal convergence, but also on practical implementation, regulatory trust, and deeper collaboration on emerging technologies and quality standards.

China’s Implementation: From Catch-Up to Co-Creation

Since joining ICH in 2017, per figures on the ICH Guideline implementation website, China has listed 125 ICH guideline items, with 118 already implemented, for an overall implementation rate of about 94%. Reported implementation rates by domain were 100% of the quality guidelines, 96% of the efficacy guidelines, 97% of the safety guidelines, and 83% of the multidisciplinary guidelines.

This progress matters not only because of scale but because it changes China’s role in global regulation: China is no longer merely catching up; it is contributing regulatory experience, clinical practice insights, and technical expertise to the global system.

For example, at a 2021 National Medical Products Administration (NMPA) ICH symposium in Beijing on China’s contributions to the process and prospects of the ICH, representatives of the Pharmaceutical Research and Manufacturers of America (PhRMA), the International Generic and Biosimilar Medicines Association (IGBA), and similar organizations praised the China regulator and government for their leadership in the ICH process. In 2024, NPMA was elected as a member of the ICH Management Committee for the third time (first in 2018, then again in 2021), another illustrative example of the international scientific community’s high regard for China’s regulator.

This transition is particularly important because China now plays a larger role in innovation, clinical development, and manufacturing. As a result, harmonization is no longer a one-way process of adoption. It is increasingly a process of cocreation.

Modernizing China’s Clinical Trial Ecosystem

China now conducts clinical development at very large scale, with more than 5,000 registered clinical trials, more than 1,900 trial institutions, and more than 30,000 principal investigators.

The challenge is more than growth; it is improving quality and efficiency while growing. China’s response has been to strengthen a full lifecycle regulatory framework covering clinical trial applications, ethics review, clinical trials, safety oversight, and inspections. At the same time, reform measures are designed to speed development for eligible products (for example, Class 1 innovative drugs under simultaneous global development), including a 30-day review channel for certain clinical trial applications.

A key milestone is the revision of China’s GCP, aligned closely with E6(R3). This new framework emphasizes several core concepts: quality by design, fit-for-purpose approaches, risk proportionality, and critical-to-quality factors (see related article in this issue). It also aims to clarify responsibilities, improve safety reporting, support digital data governance, and create clearer pathways for new technologies and methods in clinical trials. Together, these changes indicate that ICH implementation in China is becoming more practical and action-oriented, rather than merely formal.

Pharmacopeia Harmonization: Progress and Pragmatism

If clinical harmonization is one pillar of global development, quality standards are another. In the 2025 edition of the Chinese Pharmacopoeia, after nearly a decade of work to implement ICH Q4B harmonization in the Chinese Pharmacopoeia, China had achieved full coordination across the 16 general test methods covered by Q4B, a significant milestone recognized internationally.

Unlike guidance documents, pharmacopeial standards often have direct legal force and affect a large number of marketed products. Even when methods appear similar, historical practice, parameter settings, microbial strains, and implementation pathways can differ in ways that matter operationally. A pragmatic future based not only on full harmonization but also on mutual recognition, especially where scientific principles are equivalent but technical details differ, may be particularly useful in fast-moving areas where complete textual alignment is unrealistic. Areas of considerable concern include molecular biology testing, rapid microbiological testing, in vitro animal testing, and joint development of alternatives to animal testing.

Quality Standards as Enablers of Innovation

Standards can be more than compliance tools; they can also be enablers of innovation. As products become more complex, from monoclonal antibodies to mRNA and cell and gene therapies, traditional approaches to quality testing are no longer sufficient on their own. In this respect, quality and innovation are interdependent: Without quality, innovation is unsafe; without innovation, quality systems may become outdated. More flexible quality standards frameworks will combine public standards, analytical reference materials, best practices, and lifecycle thinking.

This perspective aligns closely with the broader conference theme: Harmonization works best when it creates trust, lowers development burden, and supports rapid scientific progress.

Implications for Global Development and Regulatory Strategy

Early planning will become increasingly important for multinational developers so that their clinical protocols, evidence packages, and CMC strategies can support multiple regions yet still account for local implementation differences. For cross-border clinical trials, the combination of E6(R3) and M11 may improve consistency, but operational success will still depend on ethics review processes, data governance readiness, and inspection preparedness in each market.

For China-based innovators pursuing global submissions, continued ICH implementation will support its overall objective of reducing rework and supporting more efficient global filing strategies. Even so, the benefits of ICH implementation go beyond mere compliance: Industry may benefit from engaging earlier in public consultation, pharmacopeial discussion, and technical standard development, especially in areas such as RWE, advanced therapies, and analytical lifecycle management.

Another implication for regulators and standards organizations is that trust-building mechanisms may now matter as much as new guideline development; in some cases, mutual recognition of scientifically comparable standards could reduce duplication faster than waiting for full harmonization.

or clinicians in China, two important good clinical practice (GCP) signals arrived in one week in 2026: On June 3, the International Council for Harmonisation (ICH) adopted E6(R3) Annex 2 at Step 4. On June 8, China’s National Medical Products Administration (NMPA) released the 2026 revision of China’s Good Clinical Practice for Drugs (2026 China GCP), effective September 1. Both incorporate decentralized clinical trial (DCT) and digital methods into GCP.

The practical question is how to use a common digital solution across regions, including China, while adapting its configuration and operational workflows to local requirements.

What ICH E6(R3) Annex 2 Adds

Annex 2 adds GCP considerations for trials using decentralized or pragmatic elements and real-world data beyond the “site-centered” model. It provides a decision framework: Sponsors and investigators should match oversight to risk, ensure that methods are fit for purpose—that is, suitable for their intended use in the trial—and generate sufficient quality information to support good decision-making.

Decentralization is not a technology package but a reconfiguration of trial operating models. Annex 2 covers electronic consent, home nursing, remote assessments, direct-to-patient (DTP) supply, local laboratory testing, digital health technologies, pragmatic elements, and healthcare data. Each should be integrated into protocol design and quality planning according to trial objectives, endpoint criticality, participant burden, and data risk.

Regulatory Convergence in China

Published on June 8, the 2026 China GCP incorporates quality by design, fit for purpose methods, and proportionate risk controls. Through its 2023 patient-centered clinical trial implementation guidance and 2024 DCT elements in Rare-Disease guidance, the Center for Drug Evaluation (CDE) provided broader expectations for incorporating participant needs, feasibility, and communication into trial design. As a general guidance, it has broader relevance.

A 2026 three-year industry analysis by the China Society for Drug Regulation drew on independent, repeated cross-sectional surveys conducted in 2023–2025 (2023 n=889; 2024 n=989; 2025 n=898; total N=2,776). Respondents included professionals in China from trial sponsors, contract research organizations, clinical research sites, DCT technology providers, and site management organizations. Research institutions or hospitals accounted for 55.9% of respondents in 2023 and 57.4% in 2024; the three-year report does not provide the corresponding breakdown by organization type for 2025.

The surveys reported the following percentages for 2023, 2024, and 2025, respectively:

• Reported use of DCT elements was 28.8%, 37.4%, and 33.7%;
• Electronic consent, 11.4%, 12.8%, and 15.8%; and
• Digital health technologies, 5.7%, 9.7%, and 9.0%, respectively.

These survey results provide descriptive context for DCT implementation in China. However, these self-reported surveys reflect respondent experience, not longitudinal change, national prevalence, or DCT maturity; differences in sample composition may affect year-to-year comparisons.

Beijing’s DCT pilot, organized by the Beijing Municipal Medical Products Administration, provides a practical example of local implementation. Its outputs included an expert consensus on DCT management documents, published by the Beijing Pharmaceutical Association in February 2026, and an inspection manual covering 15 inspection areas and 88 items. These tools provide practical references for trial management and local regulatory oversight.

Operational Differences in China

While Annex 2 and the 2026 China GCP converge on participant protection, reliable results, and risk-based quality management, several notable operational differences remain:

• Investigational product distribution: Annex 2 permits sponsor-arranged shipment to participants where local rules allow. The 2026 China GCP assigns to the principal investigator and institution responsibility for receipt, storage, dispensing, use, return, and destruction. DTP models therefore require a China-specific distribution pathway.
• Off-site oversight: Annex 2 requires risk-proportionate investigator oversight away from the trial site. The 2026 China GCP requires prior sponsor consent and institutional supervision for outside parties; the principal investigator and institution retain final responsibility (Article 20), and medical decisions remain with a qualified principal investigator or authorized physician (Article 25). Home-based activities therefore require China-specific delegation and oversight.
• Third parties: Both frameworks require service provider oversight. Under the 2026 China GCP, outside delegation requires prior sponsor consent, effective supervision, and retained ultimate responsibility; certain investigator decisions, confirmations, and reports are not delegable (Article 20). Sponsor outsourcing requires qualified providers, contracts, oversight, and prior written consent for subcontracting; the sponsor remains ultimately responsible (Article 36).
• Digital source data: Annex 2 recognizes remote data collection and requires traceability and source access. The 2026 China GCP requires patient trial records in outpatient or inpatient medical-record systems and direct source access for monitors, auditors, ethics reviewers, and inspectors (Articles 29 and 49). Remote-data plans should pre-define source location, record integration, and access.
• Cross-border data: Annex 2 requires compliance with privacy and data-protection rules but does not prescribe a transfer route. In China, GCP operates alongside the Personal Information Protection Law and the Measures for Data Export Security Assessment, read with the 2024 cross-border data provisions; human-genetic-resource rules may also apply. Before transfer or remote access, sponsors should classify data, determine the transfer mechanism or exemption, and implement required notice, consent, and safeguards.

What Should Global Sponsors Do?

Global sponsors should incorporate China’s requirements into protocol and digital design. A core solution with China-specific configurations can support evidence packages and regulatory submissions.

• Design for convergence (quality by design): Identify critical-to-quality factors and China-specific requirements before locking the protocol, platform, and data architecture. Localize decentralized pathways without changing the scientific core.
• Localize controls (risk proportionality): Assign responsibilities according to participant and data risk, and reflect them in contracts, procedures, training, safety communications, escalation, and monitoring.
• Make the solution and data fit for purpose: Validate the data and solution for Chinese sites and workflows. Pre-define source-record integration, metadata, audit trails, direct access, data standards, and cross-border pathways to keep China data traceable and interoperable.
• Engage early and manage change: During protocol design, assess participant, site, technical, and regulatory feasibility. For novel DCT elements, consider CDE communication under its nonbinding patient-centered guidance; reassess risks and controls throughout conduct.

Conclusion

Global sponsors need only one—not a separate—digital strategy to accommodate the ICH and China GCP guidelines: a common digital solution grounded in participant protection, reliable results, and proportionate risk management, with China-specific operational pathways for investigational product supply, third-party oversight, digital source-data verification, and cross-border data transfer in China. When designed early and reviewed throughout conduct, these pathways can preserve the scientific core while serving participant needs and best available care in each setting. “Fit for purpose” means continuously refining trial methods and controls so they remain suitable for their intended use and support reliable regulatory and clinical decisions across regions. Above all, digital strategy in clinical trials must remain patient-centered and serve patient needs.

n January 2026, the Facilitating and Accelerating Strategic Trials in the EU (FAST-EU) program was launched with the aim to speed up joint clinical trial application (CTA) review to 10 weeks (70 calendar days) from submission to final conclusion, including sponsor response time. This program is intended to support Europe’s competitiveness in multinational clinical trials.

Initial experience within the FAST-EU pilot is summarized on the Clinical Trials Coordination Group (CTCG) website. A total of 144 expressions of interest have been received since the launch of the project, with 34 clinical trials already selected for the accelerated assessment. In response to this high level of interest, and due to the substantial number of applications received, participating Member States agreed to accept more applications beyond the two per month initially planned.

The EU Commission has acknowledged the urgent need to increase the attractiveness of Europe for clinical trials. This has led to a proposal for a draft Regulation 2025/0406 (COD), called the Biotech Act. The FAST-EU pilot was developed in parallel to speed up and simplify the assessment and authorization of multinational clinical trials, addressing long-standing delays and fragmented national procedures. FAST-EU is part of the Accelerating Clinical Trials (ACT) overarching work program 2026/2027.

FAST-EU aims to demonstrate the EU’s capacity for coordination and rapid response, reinforce Europe’s global competitiveness in biomedical research, and ensure that European patients benefit sooner from scientific advancements and therapeutic innovation. It also sends a clear signal of commitment to innovation and cooperation within and across member states. In contrast to other regions in the world, Ethics Committees’ opinions of all participating member states are already built into this ambitious process.

Multinational clinical trials account for a significant share of authorized studies in Europe. These studies are important, as they enable a sufficient number of patients to be recruited across different countries to obtain robust results and accelerate the development of innovative treatments. Facilitating clinical trials in Europe also guarantees the inclusion and representation of European patients in the development of these therapies.

Project and slot coordination is handled by the CTCG. As outlined by the FAST EU Guidance, sponsors wishing to participate in the FAST-EU pilot must submit an Expression of Interest (Letter of Intention) by e-mail to the FAST-EU Coordination Office (FAST-EU@hma.eu) prior to submission of the CTA in the Clinical Trial Information System (CTIS). Of note, this requires careful planning: If an application is accepted for this procedure but the sponsor misses the submittal deadline, then the standard procedure will apply. FAST-EU applies parallel workflows, starting with CTIS submission, where the Validation as well as Part I and Part II assessments are done in parallel. Timelines have been simplified according to the “rule of 7”: All National Competent Authority (NCA) deadlines are adjusted to be multiples of 7 days, providing enhanced predictability. FAST-EU operates completely within the Clinical Trial Regulation (CTR, EU 536/2014) and uses the CTIS workflows.

First results confirm feasibility: The CTCG under the umbrella of the Heads of Medicines Agencies (HMA), and MedEthics EU under the auspices of the European Commission, published the first interim results in May 2026. By then, the process had been set up and three procedures had already been completed, all of which met or came close to meeting the target of 70 calendar days. These results demonstrated early that the 70-day timeframe is operationally feasible for different study types, indications, and constellations of Member States. The studies span all development phases (phase 1 to 3) and involve an average of 7.33 participating Member States per procedure, with up to 14 Member States per study.

A total of 26 EU Member States and EEA countries are currently participating in the FAST-EU project. The distribution of Reporting Member States (RMS) underscores the pan-European nature of the initiative, as 19 Member States have been involved as RMS (see CTCG Website Joint Actions FAST-EU). A comprehensive final report will be published when the pilot project is complete.

FAST EU is only the pilot: The draft European Biotech Act contains a proposal for overhauling the Clinical Trials Regulation.

The positive results showing feasibility support the objectives of the European Biotech Act by helping clinical trial sponsors navigate the EU clinical trials framework more effectively. Basically, they anticipate some of the many changes to the current CTR proposed by the Biotech Act (Commission proposal COM (2025) 1022 final / 16 Dec 2025). This is a legislative proposal for which the co-decision procedure (Ordinary Legislative Procedure) has started under Procedure Number 2025/0406 (COD). The next step will be negotiations with the European Parliament and the European Council. The measures proposed are amongst others intended to facilitate such accelerated assessment of CTAs beyond the current pilot cases.

Overall, the FAST-EU interim results support the conclusion that such a model is operationally feasible, even within the existing CTR framework. Observers have noted that changes to CTIS must be planned by EMA because certain functionalities such as timelines and separate validation processes are not yet supported by the CTIS infrastructure; hence an update of CTIS has been built into the Biotech Act for this.

The ongoing evaluation of the FAST-EU pilot will include qualitative findings from structured interviews with Reporting Member States on coordination efforts, application of the reliance principle, and challenges for the integration of Ethics Committees. As the pilot was planned for a year, the final report can be expected in early 2027. The plan was to extend the pilot to also cover substantial modifications and proceed in an EU4Health Joint Action (download). This will allow regulators to bridge from the end of the FAST-EU pilot until the improvements to the CTA process introduced by the Biotech Act are implemented, which would be 2028 at the earliest.

In summary, Europe has a path forward to simplify and accelerate Clinical Trial Applications collaboratively. Sponsors should continue to use these opportunities.

he value of Risk Management Plan Educational Materials (RMP EMs) depends on real-time access to the latest approved version. If paper-based materials are lost, delayed, or outdated after a safety update, users may not have current advice on monitoring, precautions, or actions to take. Electronic access can reduce delays linked to printing and physical distribution and can help keep safety information available beyond the clinic visit.

Thailand has made RMP EMs available on the Thai Food and Drug Administration (Thai FDA) website, providing an additional digital access channel alongside traditional paper-based distribution through Quick Response (QR) codes.

Electronic RMP EMs give patients and caregivers continued access to approved risk-minimization information beyond the clinic visit, allowing key risks, precautions, monitoring advice, and actions to be revisited when needed to support safe medicine use. Hosting the materials on regulatory websites together with e-labeling further enhances the value by creating a centralized and trusted pathway. Hosting the materials on regulatory websites together with e-labeling further enhances their value by creating a centralized and trusted pathway to access.

Why Is Paper Alone Not Enough?

RMPs outline how a company will identify, characterize, and minimize risk of a medicinal product throughout its lifecycle. EMs are additional risk-minimization tools used to communicate practical safety information to HCPs, patients, and caregivers, including prescriber guides, patient or caregiver guides, checklists, patient cards, and key safety actions. Electronic access to RMP EMs helps keep safety information readily available to users.

Regulatory context also matters. European Medicines Agency (EMA) Guidance on Good Pharmacovigilance Practices (GVP) describes RMPs and risk-minimization measures, and its addendum on effectiveness evaluation emphasizes assessing whether risk-minimization activities work in practice. In Thailand, approved electronic RMP EMs can be hyperlinked to the official Thai FDA drug information portal, providing a centralized public route for product prescribing information.

Benefits to HCPs, Patients, and Caregivers

Thailand provides a particularly relevant setting for this alternative accessible model because HCPs and the general public increasingly rely on digital channels for accessing healthcare information, while regulators continue to expand the availability of online medicine information resources. This makes QR-code-enabled access to approved safety materials both practical and aligned with the broader direction of medicine information access in Thailand.

Thailand’s story is important because this alternative accessible model creates practical benefits for three groups.

• For patients and caregivers, digital approaches provide easier access to current, approved RMP EMs containing key safety information. The RMP EMs continue to be available after the clinic visit, reducing reliance on misplaced or outdated documents and supporting consistent discussions with HCPs on key risks, monitoring, precautions, and safe use. Patients and caregivers can revisit the materials whenever needed.
• For HCPs, centralized national platform and QR-code access can simplify accessing and retrieving current prescribing information and RMP EMs during consultations or follow-up discussions, reducing reliance on possibly outdated stored paper copies.
• For regulators, centralized hosting on the Thai FDA platform can support consistent public access to the latest approved versions of risk-minimization materials from a trusted regulatory source, while creating a more sustainable update pathway when safety information changes.

At the same time, electronic-first should not mean electronic-only. Patient and caregiver needs vary, and some users may still prefer or require printed materials. The Thailand model, which combines Thai FDA-hosted electronic access with targeted paper support for patients who wish to receive paper, can modernize safety communication while preserving inclusivity.

What Thailand Implemented

in January 2025, in response to an informal suggestion from the Thai FDA, the Thailand-based subsidiary of a multinational pharmaceutical and biotechnology corporation introduced digital distribution of RMP EMs for four medicinal products. Once approved by the Thai FDA, the EMs were uploaded by the company and published on the Thai FDA website together with e-labeling which refers to the Thai FDA-approved product information made available electronically, including prescribing information (PI), patient information leaflets, and other approved product information where applicable. QR codes and HCP notification letters supported access, while HCPs could direct patients or caregivers to the same materials during clinical visits.

This model is designed to be nonpromotional: The materials are intended to support safe and appropriate use of medicines, not to promote products or make product claims. The operational goal was to align safety communications across letters, QR codes, and the Thai FDA website so that updated risk-minimization information remains current and easier to locate.

Survey Results

Following the implementation of digital distribution for RMP EMs, this subsidiary conducted a voluntary, quantitative, cross-sectional survey among HCPs prescribing the four medicinal products included in the initiative. Invitations were sent to 310 HCPs; 112 responded to the survey, a response rate of 36.1%. Data were collected from July 2025 to March 2026 using a Microsoft Forms questionnaire accessed by QR code. The survey explored HCP preferences for electronic versus traditional paper-based formats for both HCPs and patient/caregiver materials, HCPs’ views on patients’ preference for the format of patient/caregiver materials, and the HCPs’ expected future use of electronic formats.

• HCPs demonstrated a clear preference for electronic RMP EMs when the materials were intended for their own use. More than two-thirds of respondents (70.5%) accessed materials through QR code-linked electronic format, compared with 8.9% who preferred paper-based materials, while 20.5% reported no specific preference.
• Preferences for patient/caregiver materials were more evenly distributed, with 49.5% favoring electronic format, 27.9% preferring paper-based materials, and 22.5% indicating no specific preference.
• When asked to consider the preferences of patients or caregivers themselves, respondents perceived a near-even split between electronic materials (39.3%) and paper-based (37.5%) formats, with 23.2% indicating no specific preference.

Survey responses also showed strong perceived usefulness. Nearly all respondents agreed that electronic formats made risk-management information easier, more convenient, and faster to access, with 68.8% (n=77) strongly agreeing and 30.4% (n=34) moderately agreeing. Most respondents also expected to use electronic materials more often than paper materials in future practice (67.6% strongly agreed, n=75; 30.6% moderately agreed, n=34).

Digitalization Changing More Than Delivery Format

The findings are consistent with those of a previously published Singapore case study that demonstrated positive acceptance of electronic RMP EMs and improved dissemination timeliness following digital implementation. Thailand advances this approach by adopting a health authority-hosted model, with approved RMP EMs accessible through the Thai FDA website together with e-labeling. This creates a centralized, trusted, up-to-date source of risk-minimization information for HCPs, patients, and caregivers.

Thailand’s experience suggests that digitalization of RMP EMs is more than a change in delivery format: It offers an opportunity to strengthen the accessibility, sustainability, and consistency of safety communication. Future efforts should focus on optimizing user-friendly access through the Thai FDA website, maintaining clear communication of updates, preserving alternative access options for users who require them, and continuing to evaluate whether digital access translates into greater awareness and utilization of key safety information in routine clinical practice. Ultimately, the goal is not simply to reduce reliance on paper-based materials but to ensure reliable, real-time access to key safety messages to ensure patient safety.

Supplemental Resources

• EMA Guideline on Good Pharmacovigilance Practices Module XVI, Risk Minimisation Measures
• EMA Guideline on Good Pharmacovigilance Practices Module V, Risk Management Systems
• EMA GVP Module XVI Addendum II, Methods for Evaluating Effectiveness of Risk Minimisation Measures
• Turning the Page on Hardcopy Risk Management Plan Educational Materials: Digitalization Made Possible