Exploring the opportunities for alignment of regulatory postauthorization requirements and data required for performance-based managed entry agreements

Abstract

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.

The impact of level of documentation on the accessibility and affordability of new drugs in Norway

“…The discrepancy between perceived and documented value can be addressed through the implementation of managed entry agreements, as evidenced by the increasing adoption of such agreements ( Jommi et al, 2020 ; Efthymiadou and Kanavos, 2022 ). The complexity associated with managed entry agreement implementation remains a challenge and contributes to extended time frames for the final reimbursement decision ( Kang et al, 2020 ; Eichler et al, 2021 ; Fens et al, 2021 ). To optimize the utilization of managed entry agreements, it is essential to incorporate them into the pricing strategies of pharmaceutical companies.…”

Outcome-based reimbursement in Central-Eastern Europe and Middle-East

“…The momentum for outcome-based reimbursement models is strengthened by the ongoing initiative to enhance regulatory post-authorization requirements, especially in those cases where only conditional market authorization is granted for new medicines which respond to huge unmet medical needs with uncertain clinical value (43). Implementation of outcome-based reimbursement models is challenging, especially in resource constrained health care systems of lower income countries.…”

Manufacturers’ views on outcome-based agreements

“…Recently, Eichler et al[ 9 ] explored the feasibility of OBAs by interviewing reviewers from seven competent authorities for pricing and reimbursement. The type of product that can be considered as an OBA is described as a novel treatment with easily measurable outcomes for an unmet medical need but with uncertainty regarding its budget-impact or clinical value.…”

“…Nonetheless, in situations where the price and budget-impact are the main issues, other financial agreements can be used such as discounts, budget-caps or price-volume agreements. In general, OBAs were not often the preferred option for payers due to practical hurdles including manufacturers’ unwillingness to collaborate, differences in perception of value and administrative workload[ 9 ].…”