Abstract

After witnessing extraordinary scientific and regulatory efforts to speed development of and access to new COVID-19 interventions, patients facing other serious diseases have begun to ask “where’s our Operation Warp Speed?” and “why isn’t Emergency Use Authorization an option for our health crises?” Although this pandemic bears a number of unique features, the response to COVID-19 offers translatable lessons, in both its successes and failures, for non-pandemic diseases. These include the importance of collaborating across sectors, supporting the highest-priority research efforts, adopting rigorous and innovative trial designs, and sharing reliable information quickly. In addition, the regulatory response to the pandemic demonstrates that lowering standards for marketing authorization can result in increased safety concerns, missed opportunities for research and treatment, and delays in determining what works. Accordingly, policymakers and patient advocates seeking to build on the COVID-19 experience for non-pandemic diseases with unmet treatment needs should focus their efforts on promoting robust and efficient research designs, improving access to clinical trials, and facilitating use of the Food and Drug Administration’s existing Expanded Access pathway.

摘要

在目睹了为加速开发和获得新的 COVID-19 干预措施而做出的非凡的科学和监管努力后，面临其他严重疾病的患者开始问“我们的操作翘曲速度在哪里？” 和“为什么紧急使用授权不是我们健康危机的一种选择？” 尽管这种大流行具有许多独特的特征，但对 COVID-19 的反应为非大流行疾病提供了可转化的经验教训，无论是成功还是失败。其中包括跨部门合作、支持最高优先级的研究工作、采用严格和创新的试验设计以及快速共享可靠信息的重要性。此外，对大流行的监管反应表明，降低上市许可标准可能会导致安全问题增加，错过了研究和治疗的机会，并延误了确定什么有效的方法。因此，政策制定者和患者倡导者寻求在治疗需求未得到满足的非大流行性疾病的 COVID-19 经验的基础上，应将精力集中在促进稳健有效的研究设计、改善临床试验的可及性以及促进食品和药物的使用上。管理部门现有的扩展访问途径。