Cooperative Agreement to Support Regulatory Research Related to the 2022 Prescription Drug User Fee Act and Biosimilar User Fee Act (U19)

项目摘要 美国食品和药物管理局的药物评估和研究中心(CDER)是 寻求合作研究的建议，并召集活动以促进和促进监管科学 在食品药品监督管理局、医药行业和国会2022年重点关注的几个领域，S处方 第七号药物使用费法案(PDUFA VII)和生物相似使用费法案(BsUFA III)。建立在超过14个 在优先主题上与FDA合作多年，杜克-罗伯特·J·马戈利斯医学博士， 健康政策中心(杜克-马戈利斯)建议继续与FDA合作，以解决 PDUFA VII和BsUFA III中确定的政策、科学和业务挑战。 杜克-马戈利斯公司将以其过去的经验为基础，与FDA合作，确定优先主题 开发和交付最多九项协作研究和召集活动，包括年度 最多完成四次大型公开会议、两次私人专家讲习班和三次其他会议 根据需要和在年度预算下可实现的活动(如智囊团、网络研讨会)。取决于 根据预期的召集目的或需要，召集可以是虚拟的、混合的或面对面的。通过这一新的 合作协议，并与PDUFA VII和BsUFA III的目标保持一致，通过 值得信赖的科学，杜克-马戈利斯建议在我们现有的基础设施上扩展，广泛的利益相关者 关系、召集经验和专业知识，以及与众多FDA合作的积极记录 中心和办事处以实现以下目标： 具体目标1：实施多学科、多利益攸关方和协作研究和召集 支持FDA及其利益相关者解决复杂药物开发和监管审查的计划 PDUFA VII和BsUFA III中概述的挑战。 具体目标2：综合和提炼核心背景研究、利益相关者意见以及研讨会或会议 目标1中进行的讨论，以促进和确定具体的、可操作的后续步骤和建议 适用于所有利益相关者群体。 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。 杜克-马戈利斯公司是唯一适合与FDA合作的公司，以推进 PDUFA VII和BsUFA III。我们寻求建立在我们高级团队14年工作经验的基础上 通过其他此类合作协议与FDA合作，让利益相关者在高 感兴趣的优先问题，制定取得进展的建议和模式，并推动 监管科学，更高效地为有需要的患者开发安全有效的药物。

PROJECT Summary The Center for Drug Evaluation and Research (CDER) at the U.S. Food and Drug Administration (FDA) is seeking proposals for collaborative research and convening activities to advance and promote regulatory science in several key areas prioritized by the FDA, the pharmaceutical industry, and Congress in 2022’s Prescription Drug User Fee Act VII (PDUFA VII) and Biosimilar User Fee Act (BsUFA III). Building on more than fourteen years of collaborative working experience with the FDA on priority topics, the Duke-Robert J. Margolis, MD, Center for Health Policy (Duke-Margolis) proposes to continue collaborating with the FDA to tackle the range of policy, scientific, and operational challenges identified in PDUFA VII and BsUFA III. Duke-Margolis will build on its past experience to work collaboratively with FDA to identify priority topics for development and delivery of up to nine collaborative research and convening activities that include the annual completion of up to four large public conferences, two private expert workshops, and three other convening activities (e.g., think tanks, webinars) as needed and achievable under the annual budget. Depending on the intended convening purpose or need, convenings may be virtual, hybrid, or in-person. Through this new cooperative agreement, and in keeping with goals in PDUFA VII and BsUFA III to expedite development through trusted science, Duke-Margolis proposes to expand on our existing infrastructure, extensive stakeholder relationships, convening experience and expertise, and positive track record of collaboration with numerous FDA Centers and Offices to achieve the following aims: Specific Aim 1: Implement a multidisciplinary, multi-stakeholder, and collaborative research and convening program to support FDA and its stakeholders in addressing complex drug development and regulatory review challenges as outlined in PDUFA VII and BsUFA III. Specific Aim 2: Synthesize and distill the core background research, stakeholder input, and workshop or meeting discussions pursued in Aim 1 to promote and establish concrete, actionable next steps and recommendations for all stakeholder groups. 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. Duke-Margolis is uniquely suited to collaborate with the FDA to advance research in the key areas identified in PDUFA VII and BsUFA III. We seek to build on our senior team’s fourteen years of experience working collaboratively with the FDA through other such cooperative agreements to engage stakeholders on high- priority questions of interest, develop recommendations and models for making progress, and advance regulatory science and more efficient development of safe and effective drugs for patients in need.