The EMA has published the minutes of its face-to-face meeting with the EUnetHTA (European Network for Health Technology Assessment) which was held on 8 May 2015.
There were in-depth discussions on various items during this meeting including:
Update on Medicines Adaptive Pathways to Patients’ (MAPPs) and Adaptive Pathways (AP) Pilots: The EMA announced that the review of stage 2 proposals is currently ongoing (for which 8 products have been selected as of March 2015). Several aspects of engagements across stakeholders were discussed including economic model, which is of very high importance to payers. Participation in pilots is of interest for HTA and payer organisations, however resourcing is a bottleneck (conflicting with resources dedicated to the parallel EMA/HTA scientific advice, for which there has seen a substantial increase of requests this year).
Regarding MAPPs, the EMA reported on the progress with the IMI proposal ADAPT SMART that was submitted in response to the “coordination and support action” call topic on MAPPs
The action points of the discussion:
• EUnetHTA to facilitate contribution of different HTA institutions to the initiatives on Adaptive Pathways / MAPPs.
• EUnetHTA to continue exploring with payers how they can be involved in the development.
• EUnetHTA will continue to clarify how to can get information exchange with additional HTA organisations including from new Member States (MS).
• EMA to explore feasibility of defining “sample” scenarios to be discussed with HTAs in the context of the ADAPT-SMART project.
During the recent TOPRA DIA meeting held on 30 June 2015 on AP, Hugo Hurts (from Dutch MEB) and Leeza Osipenko (from UK’s NICE) also shared their experience with the pilots and highlighted the issue of low involvement of pricing and reimbursement authorities (because of resourcing issues mentioned above and early commitment). They also outlined the need for the companies to “think very carefully about the value propositions of their product…as early as the end of Phase I” (for more details on this workshop, please refer to the communication sent yesterday by Lindsay, Sabine and Karl).
Update on Parallel EMA/HTA Scientific Advice: Updates on both the parallel EMA/HTA scientific advice as well as the SEED project were provided. It was generally noted that a challenge is the definition of objectives of early dialogue given the different backgrounds and remits of the organisations involved. The feasibility of a permanent model for the SEED will be discussed at the next plenary EUnetHTA meeting (joint reports on both parallel EMA/HTA Scientific Advice and SEED expected)
Update on on-market evidence collection: An overview of opportunities for regulatory involvement and for potential HTA-regulatory collaboration for on-market evidence generation was provided. Of particular interest was the use of registries and their conduct in countries with interest from a clinical practice perspective. The CHMP is also developing principles for indication wording, with particular reflection on broadening and narrowing compared to the study population investigated in the clinical trials supporting the application. In such case, some key elements considered are the study population (e.g. inclusion criteria, representativeness) and benefit risk assessment (e.g. effect size, uncertainties, concerns in subpopulations, pharmacogenomic considerations, knowledge of mechanism of action) with additional fine-tuning based on factors like disease characterising, predictability of biomarkers. In this respect, the grounds of broadening/narrowing indications are therefore of high importance for the HTA’s own assessment.
Orphan medicines: Divergences were noted between the regulatory/HTA’s approach of the major contribution to patient care or “ease of use”. Indeed, this criterion is seen as sufficient by the COMP to support the “significant benefit”, and maintain the orphan drug designation, whereas its use is exceptional for HTAs, where the demonstration of improved effectiveness is generally required as a result of “ease of use”. A comparison of both the regulatory significant benefit assessment and the HTA’s Relative Effectiveness Assessment (REA) has been then requested for the next meeting.