To maintain orphan drug status at the time of market authorization, orphan medicinal products (OMPs) need to be assessed for all criteria, including significant benefit, by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). Subsequently, health technology assessment (HTA) organizations evaluate the same OMPs in their relative effectiveness assessments (REAs). This review investigates the similarities and differences between the two frameworks for six HTA organizations, including the European Network for HTA. We discuss differences between both assessment frameworks within five domains (clinical evidence used, patient population, intervention, comparators, and outcome measures) for all drugs. Five illustrative cases studies were selected for a qualitative review.

为了在市场授权时保持孤儿药状态，孤儿药产品 (OMP) 需要由欧洲药品管理局 (EMA) 的孤儿药产品委员会 (COMP) 评估所有标准，包括显着益处。随后，卫生技术评估 (HTA) 组织在其相对有效性评估 (REA) 中评估相同的 OMP。本次审查调查了六个 HTA 组织（包括欧洲 HTA 网络）的两个框架之间的异同。我们讨论了所有药物在五个领域（使用的临床证据、患者人群、干预、比较和结果测量）内的两种评估框架之间的差异。选择了五个说明性案例研究进行定性审查。