Department of Health Policy and LSE Health-Medical Technology Research Group (MTRG), London School of Economics and Political Science, UK.
Soc Sci Med. 2022 Aug;306:115119. doi: 10.1016/j.socscimed.2022.115119. Epub 2022 Jun 8.
Access to medicines treating rare diseases ('orphan medicines') has proven challenging due to high prices and clinical uncertainty. To optimise market access to these medicines, some healthcare systems are implementing specialised pathways and/or processes during marketing authorisation (MA) and/or health technology assessment (HTA). Comparing one setting where these medicines are classed as "orphan" (Scotland) to another where they considered "non-orphan" (Canada), this study aims to explore whether the presence of specialised pathways and processes at MA and HTA levels is associated with more favourable funding recommendations and faster time to market access. A matched sample of 116 medicine-indication pairs with MA approval from 2001 to 2019 in Europe and Canada was identified, and publicly available sources were used for data extraction. Descriptive statistics were used for data analysis. All medicines were commercially marketed in both countries, except one instance in Scotland. In Scotland, more orphan medicines (68.1%) had a favourable HTA recommendation than in Canada (60.4%), while Canada issued more negative HTA recommendations (20.7%) than Scotland (15.5%). Low levels of agreement on HTA recommendations and the main reasons driving recommendations were found between settings. In both countries, medicines with specialised MA approval were less likely to receive negative HTA recommendations than medicines with standard MA. Time to market access was faster in Canada than Scotland, though medicines with specialised MA approval had slower timelines than medicines with standard MA approval in both countries. However, it is unclear whether the presence of orphan designation and HTA specialised processes alone could result in favourable funding recommendations without accounting for other healthcare system-related factors and differences in the decision-making processes across settings. Holistic approaches and better alignment of evidentiary requirements across regulators are needed to optimise access to orphan medicines.
由于价格高昂和临床不确定性，治疗罕见病（“孤儿药”）的药物的可及性一直具有挑战性。为了优化这些药物的市场准入，一些医疗保健系统在营销授权（MA）和/或卫生技术评估（HTA）期间实施了专门的途径和/或流程。本研究将一个将这些药物归类为“孤儿”的环境（苏格兰）与另一个将其视为“非孤儿”的环境（加拿大）进行比较，旨在探讨 MA 和 HTA 层面专门途径和流程的存在是否与更有利的资金建议和更快的市场准入时间相关。从欧洲和加拿大 2001 年至 2019 年获得 MA 批准的 116 种药物-适应症对中确定了一个匹配的样本，并使用公开来源进行数据提取。数据分析采用描述性统计。除了苏格兰的一个例子，所有药物都在这两个国家商业化销售。在苏格兰，有更多的孤儿药（68.1%）获得了有利的 HTA 建议，而在加拿大（60.4%），而加拿大发布的负面 HTA 建议（20.7%）多于苏格兰（15.5%）。在这两个国家，在设置之间发现了对 HTA 建议的低水平一致性以及推动建议的主要原因。在这两个国家，具有专门 MA 批准的药物不太可能获得负面 HTA 建议，而具有标准 MA 批准的药物。与苏格兰相比，加拿大的市场准入时间更快，尽管在这两个国家，具有专门 MA 批准的药物的时间表比具有标准 MA 批准的药物要慢。然而，尚不清楚仅存在孤儿指定和 HTA 专门流程是否可以在不考虑其他医疗保健系统相关因素和不同决策过程的情况下导致有利的资金建议。需要采取整体方法并更好地协调监管机构的证据要求，以优化孤儿药的可及性。
