How can we make the joint EU HTA more than just a sum of the currently fragmented national work (Guest blog)
Returning from the HAS Symposium on "Together for HTA in Europe" organised under the auspices of the French Presidency of the European Union, I feel energised by the excellent discussions we had. We heard from those that established the legal basis of the EU HTA Regulation during the past couple of years, from the European Commission (EC), Member State authorities and experts, and from those that are supposed to use and benefit from the jointly produced future EU HTA outputs.
Policy makers and stakeholders clearly articulated their expectation that the EU HTA Regulation presents a unique opportunity for patients and health care systems across the EU with the potential for more timely and broader access to innovative medicines. EFPIA shares this perspective and ambition.
The EU HTA regulation is considered to be the very first output of the EU Pharmaceutical Strategy with the declared objectives to foster patient access to innovative and affordable medicines and to support the competitiveness and innovative capacity of the EU’s pharmaceutical industry. We share these objectives.
But during the discussions in Paris, it also was evident that the EU HTA regulation will only deliver against its promise, if all stakeholders collaborate during the next coming years on implementing a future-proof system that delivers high quality outputs that are relevant for decision making in Member States. Managing the 3-year transition period of implementation of the HTA Regulation towards a workable system so that the key objectives of the Regulation can be achieved is a shared responsibility of EC, Member States, HTA agencies and stakeholders, including patient representatives and industry.
During the implementation phase it will be critically important to ensure that experience, insights and recommendations of all stakeholders including industry are heard and considered. Given industry’s operational experience in navigating HTA processes and patient access pathways in all EU Member States, industry brings unique insights on how to ensure that the EU HTA regulation delivers against its objectives.
Looking at our industry’s innovation pipeline, a wide range of medicines and technologies with unique potential to improve the life of patients across the EU is expected to come to market. Increasingly, development programs target smaller, better defined target populations. Out of the 53 new active substances recommended by EMA for authorisation in 2021, 21 were for new orphan medicines or advanced therapy medicinal products (ATMPs). A wide range of clinical trial designs were used to support the marketing authorisations of these products, reflecting the changing drug development paradigm and evolving regulatory-scientific concepts. Regulatory pathways have evolved over time to better reflect the specifics of the context of a disease or an indication to ensure that patients can benefit as early as possible while maintaining high regulatory standards in terms of quality, safety and efficacy. The changing face of biopharmaceutical innovation offers unique additional treatment opportunities for unmet medical needs but at the same time adds complexity to the work of technology developers, regulators, national HTA bodies and payers.
In light of these advances and in light of the ambition of the EU Pharma Strategy, EU HTA cooperation has to be established in a way that current and future patients can benefit from the unique opportunities of innovative medicines in the most timely manner. EU HTA has to become a driver of these opportunities. It cannot become an additional bottleneck for patient access.
Now what does this mean for the implementation of the EU HTA framework. Please let me highlight a number of areas more specifically:
- There is need for a methodological framework that can reflect the specifics of new medicines, the unmet needs they target, and the context in which these are developed. The EU HTA methods needs to be able to address situations when some data may not be available, the regulation mentions explicitly the use of real-world data. A review of HTA experts highlights the methodological challenges that warrant resolution for a future HTA system to work.
- It needs sufficient Joint Scientific Advice capacity to ensure that all technology developers that seek advice on how to create clinical development plans that need to serve both the needs of regulators and HTA can get it. This capacity should ideally become available already 2022 and 2023 in order to prepare for Joint Clinical Assessments in 2025 and beyond.
- It needs a balanced conflict of interest framework to ensure that the unique expertise and knowledge of patients, clinicians and technology developers on disease context and evidence development can be reflected in the joint EU HTA work.
- It needs recognition of the health technology developer’s role as a key contributor in the scoping process. This enables that technology developer and authors of a JCA can discuss the research questions that need to be answered, and how to best do so given the data at hand. It also needs the ability for manufacturers to comment on the draft EU HTA outputs.
- A proper legal framework needs to be in place to ensure legal certainty, including the right to appeal to the relevant entity in case of disagreement.
- Member States need to create the conditions so that Joint Clinical Assessment can effectively be used in decision making. It is essential that Member States remove inefficient duplicative parallel processes and limit time consuming complementary clinical assessment activities. This is not something that the EC can do, it is fully dependent on the commitment of Member States at home.
- Progress in terms of implementation and specifically the added value of the regulation needs to be systematically and continuously monitored. Identified shortcomings should trigger relevant adjustments as early as possible, not just years after its initial application in 2025.
Together, we need to make sure that joint EU HTA work is more than the sum of the currently fragmented national work with all its shortcomings.
Based on the experience gathered in the various EUnetHTA Joint Actions we know that the production of high-quality joint EU clinical assessments is possible. The summary of our experience (see CRA report) and also the learnings form the various Joint Actions published as "A Future Model of HTA Cooperation” by EUnetHTA have identified key issues and recommendations that can guide to the development of a functioning EU level framework for EU HTA cooperation.
And there are encouraging examples of functioning EU cooperation in other areas. While the remit of EMA is different from that HTA agencies cooperating under the EU HTA regulation, the successful work of EMA for more than 25 years provides significant evidence of the Member States’ ability to collaborate on the basis of a single joint clinical assessment of innovative medicines in the best interest of patients all across the EU.