Cooperative Agreement to Support Regulatory Research Related to the 2018 Prescription Drug User Fee Act and the 21st Century Cures Act (U19)

项目总结 美国食品和药物管理局的药物评价和研究中心(CDER) 正在寻求合作研究和召集活动的建议，以推进和促进监管 科学高效地为有需要的患者开发安全有效的药物。在近十年的基础上 与FDA在这些主题上合作多年的经验，杜克-罗伯特·J·马戈利斯医学中心 卫生政策(杜克-马戈利斯大学)建议继续与CDER合作，以解决一系列政策， 2018年美国食品和药物管理局、制药业和国会优先考虑的科学和操作挑战--S 第六号《处方药使用费法案》和2017年《S 21世纪治疗法》。通过这一新的 合作协议，并与PDUFA VI和Cures的目标保持一致，通过以下方式加快发展 Trusted Science，杜克-马戈利斯公司建议通过与以下公司合作来扩大我们与FDA的现有合作 CDER、C-Path和杜克生态系统内的专家共同推进以下目标： 具体目标1：实施多学科协作研究和召集能力，以 支持FDA及其利益相关者应对复杂的药物开发和监管审查挑战，如 在PDUFA VI和治疗中概述。我们的方法将纳入广泛的利益相关者视角， 促进确定实际可行的解决方案，并支持食品和药物管理局的公共卫生使命。 具体目标2：综合和提炼核心研究、利益相关者的意见以及研讨会或会议 目标1中进行的讨论，为所有人确定具体、可操作的下一步和建议 利益相关者团体，而不仅仅是FDA。 具体目标3：将根据目标2制定的调查结果和建议以 透明和及时的方法，着眼于不断吸引利益相关者，以进一步完善和 在可行和适当的情况下实施关键概念和建议。

PROJECT SUMMARY The Center for Drug Evaluation and Research (CDER) at the U.S. Food and Drug Administration’s (FDA) is seeking proposals for collaborative research and convening activities to advance and promote regulatory science and more efficient development of safe and effective drugs for patients in need. Building on almost ten years of collaborative experience working with the FDA on such topics, the Duke-Robert J. Margolis, MD, Center for Health Policy (Duke-Margolis) proposes to continue collaborating with CDER to tackle a range of policy, scientific, and operational challenges as prioritized by FDA, the pharmaceutical industry, and Congress in 2018’s Prescription Drug User Fee Act VI (PDUFA VI) and 2017’s 21st Century Cures Act (Cures). Through this new cooperative agreement, and in keeping with goals in PDUFA VI and Cures to expedite development through trusted science, Duke-Margolis proposes to expand on our existing collaboration with FDA by partnering with CDER, C-Path, and experts within the Duke ecosystem to advance the following aims: Specific Aim 1: Implement a multidisciplinary and collaborative research and convening capacity to support FDA and its stakeholders in addressing complex drug development and regulatory review challenges as outlined in PDUFA VI and Cures. Our approach will incorporate a wide array of stakeholder perspectives, facilitate the identification of practical and actionable solutions, and support the public health mission of FDA. Specific Aim 2: Synthesize and distill the core research, stakeholder input, and workshop or meeting discussions pursued in Aim 1 to establish concrete, actionable next steps and recommendations for all stakeholder groups, not just FDA, to pursue. Specific Aim 3: Disseminate the findings and recommendations developed under Aim 2 to the public in a transparent and timely method, with an eye toward continually engaging stakeholders to further refine and implement key concepts and recommendations where feasible and appropriate.