Human Medicines Division, Scientific Evidence Generation Department, European Medicines Agency, Domenico Scarlattilaan 6, 1083 HS, Amsterdam, The Netherlands.
Office of Global Policy and Strategy, US Food and Drug Administration, Silver Spring, MD, USA.
Ther Innov Regul Sci. 2023 Mar;57(2):321-328. doi: 10.1007/s43441-022-00475-0. Epub 2022 Oct 28.
The United States Food and Drug Administration and the European Medicines Agency (EMA) each have programs to expedite development of products identified as having potential to address unmet medical needs: the Breakthrough therapy (BT) and Regenerative Medicines Advanced Therapies designation programs in the US and the Priority Medicines (PRIME) scheme at EMA. We reviewed commonalities and differences in requests submitted and products designated through these programs, with the intent to explore ways to better support global development. During the period from PRIME's launch in April 2016 to 31 December 2020, 151 requests were made to both BT and PRIME programs and the agencies reached concordant outcomes to grant or deny requests for almost two thirds of the cases (93/151, 62%), suggesting similar perspectives across international regulators on the potential of the products under study. Forty-two (42/151, 28%) products were granted both BT and PRIME, thus found by both Agencies to have the potential to address an unmet need for a serious condition, and thereby products for which efficient development would be highly desirable. Working toward better engagement on global development strategies is in the best interests of patients and public health. With this in mind, Agencies and sponsors should take advantage of existing collaborative opportunities, such as parallel scientific advice, and work to identify fresh approaches to support global development of products for unmet medical needs.
美国食品和药物管理局（FDA）和欧洲药品管理局（EMA）都有计划加快开发被认为有潜力解决未满足医疗需求的产品：美国的突破性疗法（BT）和再生医学先进疗法指定计划，以及 EMA 的优先药物（PRIME）计划。我们审查了这些计划提交的请求和指定的产品之间的异同，旨在探索更好地支持全球开发的方法。在 2016 年 4 月 PRIME 推出到 2020 年 12 月 31 日期间，向 BT 和 PRIME 计划提出了 151 项请求，这两个机构对近三分之二的案例（93/151，62%）达成了一致的结果，这表明国际监管机构对研究产品的潜力有类似的看法。有 42 种（42/151，28%）产品同时获得 BT 和 PRIME 的批准，因此这两个机构都认为这些产品有潜力解决严重疾病的未满足需求，因此是非常需要高效开发的产品。努力制定更好的全球发展战略符合患者和公共卫生的最佳利益。考虑到这一点，各机构和赞助商应利用现有的合作机会，如平行科学咨询，并努力确定支持满足未满足医疗需求的产品全球开发的新方法。
