Critical Assessment of Belgian Reimbursement Dossiers of Orphan Drugs
Critical Assessment of Belgian Reimbursement Dossiers of Orphan Drugs
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DOI：
10.2165/11585980-000000000-00000
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发表时间：
2011-01-01
影响因子：
4.4
通讯作者：
Simoens, Steven
中科院分区：
文献类型：
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作者：
Denis, Alain;Mergaert, Lut;Simoens, Steven
背景资料：孤儿药产品旨在诊断或治疗严重、危及生命或慢性衰弱的罕见疾病，并且在欧盟每10万人中影响50人或更少。在比利时，药品报销委员会（DRC）根据多个标准评估孤儿药的报销申请：治疗价值、价格和拟议的报销费率;药品在临床实践中的重要性;以及药品对预算的影响。本研究旨在评估比利时孤儿药的报销档案，并将其与提交给欧洲药品管理局（方法：一项定性分析检查了2002年1月至2008年6月期间在比利时提交的所有孤儿药报销档案。从每个档案中提取以下信息：孤儿药描述;适应症;报销状态;治疗价值和需求;预算影响;注册适应症数量。对于选定的孤儿药，深入的分析提取和比较有关的临床试验，其主要终点和结果的EMA文件（即上市许可申请文件，欧洲公共评估报告和产品特性的摘要）和比利时报销dossiers.Results的信息：报销被授予大多数孤儿药。除了官方标准外，其他可谈判的因素，如价格调整、就业激励、患者人数限制和公司对诊断测试的资助，似乎也在报销决定中发挥作用。尽管患者数量较少，但对许多孤儿药进行了随机对照试验。预算影响分析过于简单，没有考虑到对多种适应症的影响。一些差异也观察到提交给EMA的临床证据和提交给比利时DRC.Conclusions：除了官方标准，其他可谈判的因素，如价格调整和就业激励，可能会发挥作用，比利时孤儿药的报销决定。EMA文件中报告的临床证据与比利时孤儿药报销档案中包含的证据之间也存在一些差异。看来有必要使比利时的报销申请进一步标准化，并在分享孤儿药临床证据方面进行欧洲合作。
Background: Orphan medicinal products are designed to diagnose or treat rare diseases that are serious, life threatening or chronically debilitating and that affect 50 or fewer people in every 100 000 in the EU. In Belgium, the Drug Reimbursement Committee (DRC) evaluates reimbursement requests for orphan drugs based on multiple criteria: the therapeutic value, price and proposed reimbursement tariff; the importance of the drug in clinical practice; and the budget impact of the drug.Objectives: This study aimed to assess reimbursement dossiers of orphan drugs in Belgium and to compare them with the clinical evidence submitted to the European Medicines Agency (EMA).Methods: A qualitative analysis examined all reimbursement dossiers of orphan drugs that were submitted in Belgium between January 2002 and June 2008. The following information was extracted from each dossier: description of the orphan drug; indication; reimbursement status; therapeutic value and needs; budget impact; and number of registered indications. For selected orphan drugs, an in-depth analysis extracted and compared information about the clinical trials, their primary endpoints and results from EMA documents (i.e. the marketing authorization application file, European public assessment report and summary of product characteristics) and the Belgian reimbursement dossiers.Results: Reimbursement was awarded to the majority of orphan drugs. In addition to the official criteria, other negotiable factors, such as price adjustments, employment incentives, patient population restrictions and funding of diagnostic tests by the company, seemed to play a role in the reimbursement decision. Despite the low number of patients, randomized controlled trials were conducted for many orphan drugs. Budget-impact analyses were simplistic and did not consider the impact across multiple indications. Some differences were also observed between the clinical evidence submitted to the EMA and that submitted to the Belgian DRC.Conclusions: In addition to the official criteria, other negotiable factors, such as price adjustments and employment incentives, may play a role in Belgian reimbursement decisions of orphan drugs. Some differences have also been noted between the clinical evidence reported in EMA documents and the evidence included in Belgian reimbursement dossiers of orphan drugs. There appears to be a need for further standardization of Belgian reimbursement applications and for European cooperation in sharing clinical evidence of orphan drugs.