Department of Health Policy, London School of Economics and Political Science, London, UK.
Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Harvard Medical School, Boston, Massachusetts, USA.
Clin Pharmacol Ther. 2020 Mar;107(3):541-552. doi: 10.1002/cpt.1669. Epub 2019 Nov 22.
The Priority Medicines (PRIME) scheme was launched by the European Medicines Agency (EMA) in 2016 to expedite the development and approval of promising products targeting conditions with high unmet medical need. Manufacturers of PRIME drugs receive extensive regulatory advice on their trial designs. Until June 2018, the EMA granted PRIME status to 39 agents, evaluated in 138 studies (102 initiated before and 36 after PRIME eligibility). A third of the studies forming the basis of PRIME designation were randomized controlled trials, and a quarter of the studies were blinded. There was no statistically significant difference between trials initiated before and after PRIME designation in terms of randomized design and use of blinding. However, significantly more efficacy studies included a clinical end point after PRIME designation than before, and significantly fewer included surrogate measures alone. There were no statistically significant differences between the trial designs of PRIME and non-PRIME-designated products.
优先药物（PRIME）计划由欧洲药品管理局（EMA）于 2016 年推出，旨在加快针对高未满足医疗需求疾病的有前途产品的开发和批准。PRIME 药物制造商在其试验设计方面收到了广泛的监管建议。截至 2018 年 6 月，EMA 已将 PRIME 地位授予了 39 种药物，对 138 项研究进行了评估（102 项在 PRIME 资格之前发起，36 项在 PRIME 资格之后发起）。作为 PRIME 指定基础的三分之一的研究是随机对照试验，四分之一的研究是盲法的。在随机设计和使用盲法方面，PRIME 指定之前和之后发起的试验之间没有统计学上的显著差异。然而，在 PRIME 指定之后，包含临床终点的疗效研究明显增多，而仅包含替代指标的研究明显减少。PRIME 和非 PRIME 指定产品的试验设计之间没有统计学上的显著差异。
