ne of the overarching themes of DIA Europe 2024 was the imperative of alignment and harmonization across regulatory bodies and stakeholders. Representatives from the European Medicines Agency (EMA), World Health Organization (WHO), US Food & Drug Administration (FDA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) echoed the importance of increasing regulatory convergence, particularly in real-world evidence (RWE) guidance for drug safety assessments. Collaborative efforts at the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) and the International Coalition of Medicines Regulatory Authorities (ICMRA) have proven instrumental in driving this agenda forward and have paved the way for more streamlined processes and enhanced regulatory coherence.

The significance of sustained dialogue and collaboration is exemplified by ongoing DARWIN EU studies. DARWIN EU is a federated network of data, expertise, and services that supports better decision-making throughout the medicine lifecycle by generating reliable evidence from real-world healthcare data. The multitude of stakeholders actively engaged in this platform continues to generate a palpable sense of commitment towards fostering continuous evolution and advancement. Recent onboarding of new data partners to grow the number of studies further underscores the collaborative ethos driving DARWIN EU, promising valuable insights and opportunities as the network aims to become fully operational in 2024 and routinely support the evaluation work of EMA’s scientific committees and the national competent authorities. The call for new partners to invite different data partners over Europe to join DARWIN EU is currently open and is seeking partners from primary and secondary care data sources, claims databases, biobanks, etc.

As the healthcare landscape continues to evolve, the integration of AI in medicines development is also pivotal. The complex legislative landscape surrounding AI necessitates multistakeholder dialogue to craft frameworks that foster innovation and ensure responsible and safe applications. Ongoing efforts from regulatory bodies such as the EMA and the European Medicines Regulatory Network (EMRN) shed light on navigating this terrain, with a particular focus on developing fit-for-purpose frameworks. These include the European Commission’s Artificial Intelligence Act and EMA’s Draft reflection paper on the use of Artificial Intelligence (AI) in the medicinal product lifecycle.

Derisking and Decentralizing Development

Progress and lessons learned from the first year of the EU Clinical Trials Regulation (CTR), consolidated across regulator, ethics committee, trial sponsor, and patient perspectives, keep the impact of decentralized clinical trials (DCTs), innovative clinical trial designs, and the regulatory acceptability of platform trials and external controls moving forward. Initiatives aimed at derisking early medicine development include the 3Rs, early target derisking, and Clinical Trials-Simultaneous National Scientific Advice, all designed to enhance efficiency, safety, and sustainability in the development ecosystem.

The evolving landscape of manufacturing and quality assurance in the pharmaceutical industry highlights the need for an enabling regulatory framework that fosters innovation while upholding sustainability goals. Concepts such as “Goldilocks” regulation, which advocates for striking the right balance between oversight and flexibility, resonate throughout the pivotal role of regulatory bodies such as EMA in enabling innovation and driving sustainable practices within manufacturing processes including supporting the new EMA Quality Innovation Group (QIG), finalizing updates to the CMC Regulatory Framework such as GPL Annex II, new guidelines on variations, ICH M4Q(R2), and making sustainability in manufacturing a priority topic for the EMA QIG and DG SANTE.

New directions in patient safety incorporate behavioral science and qualitative approaches for setting risk management’s “intended use” and emphasize interaction between healthcare professionals and patients in developing interventions. Global work-sharing on medicine benefit-risk assessments can also sustain the generic and biosimilar markets. Inclusivity in clinical trials, particularly including pregnant women, is crucial for safe and effective medication use. Evolving our safety approaches and engaging diverse stakeholders in pharmacovigilance and patient safety initiatives remains critical.

The inherent complexity and intersectionality of Europe’s regulatory landscape remain another key area of focus, especially the intersections between different regulatory frameworks and the need for clarity and consistency to effectively navigate them.

Medical products increasingly include medicine and medical device components, yet their respective legislations have developed independently, creating barriers such as differing responsibilities at the EU and Member State level. Additionally, the European Health Data Space (EHDS) aims to facilitate the use of health data while balancing General Data Protection Regulation (GDPR) considerations, necessitating the careful drafting of legislation to avoid adding complexity and ensure alignment with other pertinent regulations.

The new Directive and new EU Pharmaceutical Regulation, which revise and replace the existing general pharmaceutical legislation, present an opportunity to further align with other regulatory frameworks. The importance of frameworks and tools such as PRIME and the Quality Toolbox, along with the significance of established frameworks and initiatives like IHI, support early stakeholder engagement that anticipates changes and challenges. Collaboration and dialogue across EU sectors and bodies (e.g., the EMA, European Chemicals Agency [ECHA], and European Food Safety Authority [EFSA]) have become indispensable for future-proofing regulations and ensuring patient-centric approaches.

Optimizing Patient Preferences and Care Pathways

The importance of optimizing care pathways and integrating patient preferences into decision-making processes is center stage in HTA and access discussions in Europe. Ensuring that patients understand the significance of their involvement in preference studies remains at the heart of these discussions. The outcomes of a single preference study have the potential to significantly influence decisions made by industry, regulators, HTA bodies, and payers, and patient preference data can complement but should not fully replace other clinical evidence. Empowering patients by involving them as research partners in preference studies is also becoming more important; this collaborative approach not only enhances the relevance, feasibility, and acceptability of study designs but also improves study conduct and the interpretation of study findings. Maintaining close alignment between EU Market Access and Regulatory teams to avoid duplication remains of great value.

Raising the education and awareness levels of HTA and of the Joint Clinical Assessment (JCA) or Joint Scientific Committee (JSC) in every corner of the life sciences communities in Europe will be essential to the success of these initiatives. Policy makers can support JCAs by increasing communication with national stakeholders to discuss specific national changes required as the EU environment evolves, developing methodologies for consolidating Population, Intervention, Comparison, and Outcome (PICO) frameworks, establishing pragmatic rules of stakeholder interaction, and reflecting on how Health Technology Dialogue (HTD) interactions could benefit all parties.

Central to all these visions is cultivating sustainable global partnerships. Characterized by their multidimensional nature, global partnerships can tackle complex challenges in innovative and holistic ways to drive transformative change. For example, the UK Medicines and Healthcare products Regulatory Agency’s (MHRA) involvement in both national and international spheres to advance the Net Zero agenda underscores collaboration toward sustainability in healthcare. Other initiatives aimed at promoting sustainability in the healthcare sector include Net Zero, the UK Medical Device Regulations, and the British Pharmacopoeia Environmental Sustainability Information Pack. These initiatives collectively reflect a shared commitment to fostering a more sustainable future for healthcare systems globally.