Ronco Virginia, Dilecce Myriam, Lanati Elena, Canonico Pier Luigi, Jommi Claudio
Market Access Provider Srl, Via V. Monti, 3, 20123, Milan, Italy.
Dipartimento di Scienze del Farmaco, Università del Piemonte Orientale, Largo Donegani 2, 28100, Novara, Italy.
J Pharm Policy Pract. 2021 Mar 19;14(1):30. doi: 10.1186/s40545-021-00311-0.
Advanced therapy medicinal products (ATMPs) represent an important cornerstone for innovation in healthcare. However, uncertainty on the value, the high average cost per patient and their one-shot nature has raised a debate on their assessment and appraisal process for pricing and reimbursement (P&R) purposes. This debate led experts providing for recommendations on this topic. Our primary objective is to investigate the ATMPs P&R process in the main five European countries and to understand if this process is consistent with published P&R expert recommendations. We also investigated the current ATMP pipelines to understand if future ATMPs will create challenges for their P&R process.
P&R framework for ATMPs in the European Major five (EU5) countries was investigated through a literature search on PubMed, institutional websites of National Health Authorities and grey literature. The ATMPs pipeline database was populated from a clinical trial database (clinicaltrials.gov), relying on inclusion and exclusion criteria retrieved from the literature.
Reimbursement status of ATMPs is different across the EU5 countries, with the exception of CAR-Ts which are reimbursed in all countries. Standard P&R process in place for other medicinal products is extended to ATMPs, with the exception of some cases in Germany. List prices, where available, are high and, tend to be aligned across countries. Outcome-based Managed Entry Agreements (MEAs) have been extensively used for ATMPs. Extra-funds for hospitals managing ATMPs were provided only in Germany and, as additional fund per episode, in France. The accreditation process of hospitals for ATMPs management was in most countries managed by the national authorities. As far as ATMPs pipeline is concerned, ATMPs in development are mostly targeting non-rare diseases.
Expert recommendations for ATMPs P&R were partially applied: the role of outcome-based MEAs has increased and the selection process of the centres authorized to use these treatments has been enhanced; additional funding for ATMPs management to accredited centres has not been completely considered and annuity payment and broader perspective in cost considerations are far from being put in place. These recommendations should be considered for future P&R negotiations to pursue rational resource allocation and deal with budget constraints.
先进治疗医药产品（ATMPs）是医疗保健创新的重要基石。然而，其价值的不确定性、每位患者的平均成本较高以及一次性性质引发了关于其定价和报销（P&R）评估和评价过程的争论。这场争论促使专家们就该主题提出建议。我们的主要目标是调查五个主要欧洲国家的ATMPs定价和报销流程，并了解该流程是否与已发表的定价和报销专家建议一致。我们还研究了当前的ATMPs研发管线，以了解未来的ATMPs是否会给其定价和报销流程带来挑战。
通过在PubMed上进行文献检索、国家卫生当局的机构网站以及灰色文献，对欧洲主要五个国家（欧盟五国）的ATMPs定价和报销框架进行了调查。ATMPs研发管线数据库来自临床试验数据库（clinicaltrials.gov），依据从文献中检索到的纳入和排除标准进行填充。
除嵌合抗原受体T细胞（CAR-Ts）在所有国家均有报销外，ATMPs在欧盟五国的报销状况各不相同。适用于其他医药产品的标准定价和报销流程也适用于ATMPs，但德国的某些情况除外。如有标价，价格较高且各国往往趋于一致。基于结果的准入管理协议（MEA）已广泛应用于ATMPs。仅在德国为管理ATMPs的医院提供了额外资金，在法国则是按每次事件提供额外资金。在大多数国家，医院管理ATMPs的认证过程由国家当局负责。就ATMPs研发管线而言，正在研发的ATMPs大多针对非罕见疾病。
针对ATMPs定价和报销的专家建议得到了部分应用：基于结果的MEA的作用有所增加，授权使用这些治疗方法的中心的选择过程得到了加强；尚未完全考虑向经认证的中心提供用于ATMPs管理的额外资金，年金支付以及成本考虑中的更广泛视角也远未到位。在未来的定价和报销谈判中应考虑这些建议，以实现合理的资源分配并应对预算限制。
