EFPIA statement on adoption of Health Technology Assessment Regulation
14.12.21
EFPIA, representing the research-based pharmaceutical industry in Europe, notes that, after many years of negotiations, the EU HTA Regulation has cleared the final hurdle in the legislative negotiations after the adoption in the European Parliament and will be published in the Official Journal soon. EFPIA still regrets that the original ambition set forth by the European Commission in its original proposal has been diluted and that we continue to see the risk of an unpredictable system whereby Member States decide on a case-by-case basis if and how they commit themselves to use jointly conducted clinical assessments.
EFPIA strongly supports a system to pool resources and high level expertise so that medicines can be assessed jointly for their relative effectiveness on the basis of clinical evidence and that joint early scientific dialogue can take place with HTA agencies. In order to preserve the spirit of such a system it is of utmost importance that the process leads to high-quality joint clinical assessment reports and their de-facto use by decision-makers at national level.
The recently released EPSCO Council Conclusions acknowledged that the future HTA Regulation can support Member States in their decisions to secure access to innovative health technologies, a statement echoing the European Commission Pharmaceutical Strategy call for the HTA Regulation to support Member States in securing timely and evidence-based access to innovative medicines.
As we move forwards into the implementation period, we must continue to keep the objective of timely access to innovative treatments in mind and avoid further delaying timelines at national level. Feasibility is key in this respect and the implementing arrangements and guidelines for the future system must ensure that:
- National adaptation is foreseen and streamlined to be able to use the EU level outputs; we call on Member States to ensure new processes are put in place, that existing templates and national policies are adapted (where needed) to optimize the timing of the overall EU and national full HTA process.
- Manufacturers are meaningfully included in the assessment process as experience has shown that HTA assessments are set up for less optimal outcomes if it there is no explicitly defined opportunity for the manufacturer to consult with assessors if the assessment plan cannot be "informed" by the knowledge of a manufacturer about the technology. Insights from manufacturers, throughout the process (e.g. scoping, PICO definition, touch-points throughout the process, opportunity to comment on evidence interpretation, appeals mechanism etc.) is a key element of a high-quality assessment.
- It is underpinned by ‘fit-for-purpose’ methodology leading to high quality outputs that can be used by diverse national HTA bodies. Methodological guidelines should be aligned to approaches required for highly innovative portfolios (Cancer, ATMP, OMP, etc.)
- Sufficient capacity and appropriate resources for joint scientific consultations is enabled to allow any manufacturer access to such a process. Joint scientific consultations remain a key element in the framework. As well as providing an opportunity to establish evidence requirements for the marketing authorization procedure and joint clinical assessment at an early stage during the drug development process, it is also crucial as a means of allowing companies to plan and carry out studies in a manner that meets the criteria for marketing authorization and HTA specifications.
EFPIA stands ready to meaningfully contribute through the work of EUnetHTA-21, the work of the Coordination Group, the development of implementing acts and at national level towards ensuring that the future system for joint clinical assessments delivers for patients and that the objective of timely access to innovative medicines is secured.