TY - JOUR
T1 - Exploring the opportunities for alignment of regulatory postauthorization requirements and data required for performance-based managed entry agreements
AU - Eichler, Hans-Georg
AU - Adams, Roisin
AU - Andreassen, Einar
AU - Arlett, Peter
AU - van de Casteele, Marc
AU - Chapman, Suzannah J
AU - Goettsch, Wim G
AU - Martinsson, Jonathan Lind
AU - Llinares-Garcia, Jordi
AU - Nachtnebel, Anna
AU - Pean, Elias
AU - Rasi, Guido
AU - Reksten, Tove Ragna
AU - Timmers, Lonneke
AU - Vreman, Rick A
AU - van de Vijver, Inneke
AU - Wenzl, Martin
PY - 2021/8/23
Y1 - 2021/8/23
N2 - Performance-based managed entry agreements (PB-MEAs) might allow patient access to new medicines, but practical hurdles make competent authorities for pricing and reimbursement (CAPR) reluctant to implement PB-MEAs. We explored if the feasibility of PB-MEAs might improve by better aligning regulatory postauthorization requirements with the data generation of PB-MEAs and by active collaboration and data sharing. Reviewers from seven CAPRs provided structured assessments of the information available at the European Medicines Agency (EMA) Web site on regulatory postauthorization requirements for fifteen recently authorized products. The reviewers judged to what extent regulatory postauthorization studies could help implement PB-MEAs by addressing uncertainty gaps. Study domains assessed were: patient population, intervention, comparators, outcomes, time horizon, anticipated data quality, and anticipated robustness of analysis. Reviewers shared general comments about PB-MEAs for each product and on cooperation with other CAPRs. Reviewers rated regulatory postauthorization requirements at least partly helpful for most products and across domains except the comparator domain. One quarter of responses indicated that public information provided by the EMA was insufficient to support the implementation of PB-MEAs. Few PB-MEAs were in place for these products, but the potential for implementation of PB-MEAs or collaboration across CAPRs was seen as more favorable. Responses helped delineate a set of conditions where PB-MEAs may help reduce uncertainty. In conclusion, PB-MEAs are not a preferred option for CAPRs, but we identified conditions where PB-MEAs might be worth considering. The complexities of implementing PB-MEAs remain a hurdle, but collaboration across silos and more transparency on postauthorization studies could help overcome some barriers.
AB - Performance-based managed entry agreements (PB-MEAs) might allow patient access to new medicines, but practical hurdles make competent authorities for pricing and reimbursement (CAPR) reluctant to implement PB-MEAs. We explored if the feasibility of PB-MEAs might improve by better aligning regulatory postauthorization requirements with the data generation of PB-MEAs and by active collaboration and data sharing. Reviewers from seven CAPRs provided structured assessments of the information available at the European Medicines Agency (EMA) Web site on regulatory postauthorization requirements for fifteen recently authorized products. The reviewers judged to what extent regulatory postauthorization studies could help implement PB-MEAs by addressing uncertainty gaps. Study domains assessed were: patient population, intervention, comparators, outcomes, time horizon, anticipated data quality, and anticipated robustness of analysis. Reviewers shared general comments about PB-MEAs for each product and on cooperation with other CAPRs. Reviewers rated regulatory postauthorization requirements at least partly helpful for most products and across domains except the comparator domain. One quarter of responses indicated that public information provided by the EMA was insufficient to support the implementation of PB-MEAs. Few PB-MEAs were in place for these products, but the potential for implementation of PB-MEAs or collaboration across CAPRs was seen as more favorable. Responses helped delineate a set of conditions where PB-MEAs may help reduce uncertainty. In conclusion, PB-MEAs are not a preferred option for CAPRs, but we identified conditions where PB-MEAs might be worth considering. The complexities of implementing PB-MEAs remain a hurdle, but collaboration across silos and more transparency on postauthorization studies could help overcome some barriers.
KW - Drug regulation
KW - Managed entry agreement
KW - Pricing and reimbursement
KW - Real-world data
UR - http://www.scopus.com/inward/record.url?scp=85114846942&partnerID=8YFLogxK
U2 - 10.1017/S026646232100057X
DO - 10.1017/S026646232100057X
M3 - Article
C2 - 34424152
SN - 0266-4623
VL - 37
SP - 1
EP - 11
JO - International Journal of Technology Assessment in Health Care
JF - International Journal of Technology Assessment in Health Care
IS - 1
M1 - e83
ER -