Performance-based managed entry agreements (AP-AME) could give patients access to new medicines, but practical barriers make competent pricing and reimbursement authorities (CAPRs) reluctant to implement AP-MEAs. We investigated whether the feasibility of PMAs could be improved by better adapting post-authorization regulatory requirements to PB-MEA data generation and using them through active collaboration and data exchange. Reviewers from seven CAPRs provided structured assessments of the information available on the European Medicines Agency (EMA) website on the regulatory requirements for the re-authorisation of fifteen recently authorised products. The evaluators assessed the extent to which post-authorization regulatory studies could contribute to the implementation of PB-MEAs by addressing safety deficiencies. The areas of study studied were: patient population, intervention, comparators, outcomes, time horizon, expected data quality and expected robustness of the analysis. Reviewers provided general comments on the pMEAs for each product and on collaboration with other CAPRs. The auditors assessed the post-authorisation regulatory requirements for most products and across domains, at least partially useful, with the exception of the area of comparison. A quarter of the responses indicated that the public information provided by the EMA was not sufficient to support the implementation of the PB-MEAs. There were few PB MEAs for these products, but the potential for PB PMA implementation or collaboration between CAPRs was considered more favourable. The reactions helped to delineate a number of conditions under which PB MEAs can help reduce uncertainty.
In summary, AP-MEAs are not a preferred option for CAPRs, but we have identified conditions under which PB-MEAs may be worth considering. The complexity of implementing PB MEAs remains an obstacle, but collaboration between silos and greater transparency in post-authorisation studies could help overcome some obstacles. The seven participating CAPRs provided responses. In nine product indication pairs, one CAPR was unable to exchange information specifically on whether pb-MEA is present; for another product-indication pair, two CAPRs were unable to share this information. The inability to provide this information was due to confidentiality agreements. Responses to the remaining questions were not limited by confidentiality considerations. MEAs are most effective when they are tailored to the specific circumstances in which they are used and the preferences of the decision-maker concerned. This adjustment should include a careful examination of the characteristics of the therapy to be reimbursed, the characteristics of the health care payment system, the possibilities of different payment structures and the choices of the decision-maker with regard to financial agreements versus outcome-based agreements, individual or population-based agreements and the combination of several mechanisms. Our comprehensive analysis of the feasibility of MEAs in each of these situations can help in selecting a combination of agreements. Managed entry contract, reimbursement, payment, price, policy, cost, price, innovative, atmp, new, gene/cell therapy, expensive, payer, financing.
However, there is another limit at which transparency needs to be improved: the OECD report (Wenzl and Chapman Reference2) pointed out that the results of multilateral environmental agreements, including those of multilateral environmental agreements, are often confidential and that information generated under the agreements is not shared with other CAPRs and third parties to broaden knowledge. We believe that the secrecy about clinical, but not financial, outcomes of post-authorization data generation plans is not only a missed opportunity to advance our understanding of the effects of drugs, but also violates an ethical imperative to share what can be considered common property. In recent years, enormous progress has been made in the transparency of pre-authorisation studies. Now it`s time to do the same for the information generated after launch. the need for detailed and accurate documentation in protocols and reports to allow for the interpretation of post-authorisation (observational) studies has been developed several times (12; Reference Wang, Pinheiro, Hua, Arlett, Uyama and Berlin13). In summary, PB MEAs are often not the preferred option for CAPRs to deal with uncertainty. Our study identified a number of barriers, but also the conditions under which a PB-MEA might be worth considering. It remains to be seen whether this can help CAPRs identify products for which the PB-MEA would help fill security gaps at launch to an extent that would justify the implementation of such complex agreements.
We are aware of the many problems in the implementation of AME-PB, including legal obstacles, the reluctance of manufacturers to conclude such agreements, the different perceptions of benefits between payers and the administrative burden. .