Health Sector Management, Duke University, Durham, North Carolina, USA
Deutsche Stiftung Weltbevölkerung, Brussels, Belgium.
BMJ Glob Health. 2024 Jan 30;9(1):e013686. doi: 10.1136/bmjgh-2023-013686.
Neglected diseases are a significant global health challenge. Encouraging the development of therapeutics and vaccines for these diseases would address an important unmet medical need. We propose a priority review voucher programme for the European Union (EU). The developer of a drug or vaccine for a neglected disease would receive a voucher for accelerated assessment of a different product at the European Medicines Agency (EMA).
This study uses retrospective observational data to estimate the potential commercial value of the proposed voucher programme using a five-step approach: (1) estimating the time saved in the EMA accelerated regulatory review; (2) gauging time reductions in accelerated pricing and reimbursement decisions by EU member states; (3) selecting 10 high-revenue products launched between 2015 and 2020 representing typical voucher users; (4) analysing IQVIA MIDAS sales data for the selected products and (5) calculating the net present value (NPV) of the voucher based on the 10 products.
The accelerated EMA review would reduce regulatory time by an average of 182 days. Additionally, products could save more than a year in many member states through an expedited 120-day pricing and reimbursement review. The estimated NPV of regulatory acceleration by two quarters would be €100 million. In addition, if France, Italy and Spain reviewed pricing and reimbursement in only 120 days, then the value would double.
An EU voucher estimated at more than €100 million, coupled with a US$100 million counterpart, offers a meaningful incentive for novel product development. However, the voucher programme should be part of a comprehensive strategy for tackling neglected diseases, rather than a standalone solution.
被忽视的疾病是全球健康面临的重大挑战。鼓励针对这些疾病开发治疗方法和疫苗将满足重要的未满足医疗需求。我们建议为欧盟（EU）设立优先审查券计划。开发治疗或预防被忽视疾病的药物或疫苗的公司将获得一张券，可用于加速在欧洲药品管理局（EMA）评估另一种产品。
本研究使用回顾性观察数据，通过五步方法估计拟议的券计划的潜在商业价值：（1）估计在 EMA 加速监管审查中节省的时间；（2）衡量欧盟成员国加速定价和报销决策的时间缩短；（3）选择 2015 年至 2020 年期间推出的 10 种高收入产品，代表典型的券使用者；（4）分析选定产品的 IQVIA MIDAS 销售数据；（5）基于 10 种产品计算券的净现值（NPV）。
加速 EMA 审查将使监管时间平均缩短 182 天。此外，通过加快 120 天的定价和报销审查，产品在许多成员国的时间可节省一年以上。两个季度的监管加速估计 NPV 为 1 亿欧元。此外，如果法国、意大利和西班牙仅在 120 天内审查定价和报销，那么价值将翻一番。
价值超过 1 亿欧元的欧盟券，加上 1 亿美元的美国券，为开发新产品提供了有意义的激励。然而，券计划应该是解决被忽视疾病的综合战略的一部分，而不是独立的解决方案。
