Proceedings: DIA Europe 2019

Pictures of Transformation:
Snapshot of DIA Europe 2019

Ursula Busse
Global Head, Quality Intelligence & External Affairs

DIA Europe 2019

paradigm shift in healthcare was evident at DIA Europe 2019 in Vienna. Well over a thousand attendees from industry, regulatory authorities, patient associations, and suppliers participated in multiple tracks and sessions over three exciting days. This is what we learned.

Healthcare System Transformation

The current healthcare model is unsustainable.

Current healthcare systems are not sustainable: Over the last 100 years, our life expectancy grew by 35 years. This came at a cost that is outpacing GDP. This cost will double again in the next twenty years. High pressure on public spending will only grow.

Sustainable health systems need: 1) individual empowerment and responsibility, people making life choice decisions; 2) greater accountability of governments and healthcare providers to deliver effective, sustainable health services; 3) data-driven, evidence based value and outcomes.

In the future, therapies will be defined as synergistic interactions between different elements of care. Business models are needed to measure outcomes of healthcare intervention. Future healthcare systems require changes in incentive systems, and for people to value their health while they still have it. 45 percent of diseases are related to attitudes/behaviors.

Transformation Enabled by Digital Solutions

Current trends include evolutions from volume to outcome/value; from cure/treatment to prevention and prediction that results in reduced demand for healthcare.

Many solutions for future healthcare systems relate to digital technology (such as robotics, telepresence, etc.), and come out of the private sector. They will impact jobs, market structure, and value proposition.

High precision tumor diagnosis is now possible thanks to innovative DNA/RNA sequencing techniques. Precision medicine techniques such as CMTA (comprehensive molecular tumor analysis) are available to stratify tumors. Predictive in silico modelling using a virtual twin concept can measure response to single drugs and combinations of drugs.

Data will become the main asset (the “new oil”). Access to healthcare data will become increasingly important. Access and ethical governance rules must be defined. The data ecosystem could adopt open sourcing for technologies and share data in a non-competitive space for the benefit of all.

New methodologies are being developed for clinical trials. Today’s gold standards were created 100 years ago! TransCelerate works on a new data sharing platform, DataCelerate, to augment a study with smaller placebo populations (placebo studies are major hurdle to getting patients enrolled) and accelerate clinical trials.

2018 was a record year for digital health M&A. Data vertical integration (mergers) will happen, and more collaboration between pharma and tech is expected. Regulation, social acceptance (cyber risk, trust, health system actors, etc.) and cost savings versus increased demand will challenge the digital transformation.

Increased Power of Patients

The consumer, no longer the facility, is our new center. Patients are the driving force to change legislation; they are involved in almost all lifecycle activities, from bench to bedside. They have the motivation, focus, and (increasingly) access to knowledge. Expect patients to become the major driver for change in the healthcare system.

Europe’s Readiness to Face the Requirements of Future Therapies

Europe needs to act quickly.

Several warning signs point to the need to act quickly to make the EU an attractive place for innovation: The EU lags in fast track approvals and takes a longer time to approve. EU’s supply chains are under pressure; they are increasingly complex and not secure enough. Medicine availability is limited by pricing and reimbursement.

Regulatory Science on the rise

Regulators need optimal tools to keep pace with scientific and technological advances and ensure the sound assessment of more complex, groundbreaking therapies. There is a need for very strong collaboration in the healthcare sector and Regulatory Science is becoming increasingly important. EMA has released its Regulatory Science Strategy to 2025 which includes challenges to regulatory systems and how the EU intends to overcome them.

EU Patient Associations contribute to these efforts

The EU Patient’s Forum (EPF) developed a strategy to match the 2025 strategy, focusing on patients. They worked on “Paradigm,” a new framework for engaging patients focused on three items: 1) prioritize research, 2) novel clinical trial design, and 3) critical early dialogue with HTAs (reimbursement).

EU Telematics Strategy implementation continues

EMA’s continues to deploy its telematics strategy towards a common IT infrastructure in the EU (examples include EudraVigilance, SPOR, and upcoming MDR implementation). EMA has published an extension to the EU Telematics strategy and implementation roadmap to cover 2018–2019.

EMA’s Innovation Task Force and the EU Innovation Network intend to catalyze innovation

EMA’s Innovation Task Force (ITF) has an evolving mission, amplifies opportunities for early advice, and collaboratively conducts horizon scanning. EMA works on a closer collaboration with HTA and payers. EMA also partners more with academia to undertake fundamental research in strategic areas.

The EU Innovation Network (EU-IN) fosters research and the uptake of innovative methods in the development of medicines. 24 national offices participate in the network. Horizon scanning helps to ensure that the network is able to regulate novel therapies and associated technologies such as nanomedicines, novel manufacturing strategies (such as 3D printing), and mobile/ digital apps in clinical trials.

The EU-IN also collaborates at the ICMRA level and encourages regulatory participation in Innovative Medicines Initiative (IMI) projects. EU regulators provide expertise and regulatory advice to IMI projects to develop new regulatory concepts and solutions. The latest IMI projects focus on digital and other innovative technologies.

New legislative framework for HTAs to reconcile innovation with access

EU-wide projects for collaboration between HTAs have been launched. The European Commission proposes a new legislative framework for HTAs that includes: joint clinical assessments, joint scientific consultations, horizon scanning of emerging health technologies, and voluntary cooperation in other areas.

Access to ATMPs in the European Union

Remaining challenges in the EU legislative framework

The EU has been building a regulatory framework for Advanced Therapy Medicinal Products (ATMPs) for over a decade to facilitate access to these innovative therapies (EC 1394/2007). The EU provides ample information on ATMP classification, and the EMA Committee for Advanced Therapies (CAT) can provide additional advice.

However, the EU legislative framework is not yet fully adapted to ATMPs and their inherent complexity. EMA is working on clarifying details related to genetically modified organism (GMO) legislation. The implementation of EU Medical Device Regulation (MDR) Article 117 for drug-device combination ATMPs must be defined. Finally, provisions in the legislation pertaining to the “hospital exemption” must be clarified.

Need for patient registries

To evaluate the risk benefit for ATMPs, long-term safety follow up (fifteen years) is needed. An overall framework for patient registries and the quality of post licensing data (“real world evidence”) must be defined and implemented.

Need for flexible pricing & reimbursement models

Additional main challenges to access include HTA feasibility, financial sustainability, and innovative pricing feasibility. Access to ATMPs is still limited since regulatory and reimbursement agency (HTA) evidence needs are disconnected.

Flexible reimbursement models are also needed. The Institute for Clinical and Economic Review (ICER) has started a new project to develop and test alternative methods for evaluating potentially curative treatments and for translating the results of cost-effectiveness analyses into recommendations for value-based price benchmarks.

Finally, GDP variation between EU member states results in differential pricing for treatment coming from a single treatment center. A possible solution could be cross-region funding channels.

International Collaboration

ICH reforms successfully completed

Since 2016, new regulatory authorities have joined ICH both as members (e.g., China, Brazil) or observers (e.g., India, Iran, Russia, South Africa, Turkey).

Membership expansion has an impact on the size of expert working groups (EWG), an issue that ICH is addressing. These reforms, however, reduce industry’s influence in the ICH process: EWGs have more regulatory and therefore less industry representation, and industry is only involved up to Step 2a.

ICH will perform a survey on the implementation status of ICH guidelines in ICH regions, and share its results in June 2019. Multiple work streams have been launched to engage with training organizations and institutions