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

Description

RESEARCH OBJECTIVES The FDA Center for Drug Evaluation and Research seeks to support efforts to research, identify key issues, and convene appropriate subject matter experts to help advance regulatory science to promote the increased availability of safe and effective drugs to the public. This effort will inform regulatory science by advancing the scientific discussion of key research topics of interest to both FDA and external stakeholders such as the pharmaceutical industry, healthcare professionals, patients, and academic researchers. FDA seeks support to address research needs identified in the 2018 reauthorization of the Prescription Drug User Fee Act (PDUFA VI) and requirements under the 21st Century Cures Act. The research areas identified in PDUFA and the 21st Century Cures Act represent key scientific priorities identified by FDA, Congress, and stakeholders that, if advanced, will help expedite the development of drugs and improve efforts to assess their safety and effectiveness, thereby increasing the availability of safe and effective drugs to the public. More information about the research areas identified under PDUFA VI can be found at the following website: https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM511438.pdf SPECIFIC AREAS OF RESEARCH INTEREST In the most recent reauthorization of PDUFA, FDA identified specific areas of research interest in consultation with drug industry representatives, patient and consumer advocates, health care professionals, and other public stakeholders. These areas represent a wide range of new innovative initiatives related to virtually every aspect of the new drug life cycle. Advancing research, discussion, and understanding on these areas will benefit FDA's stakeholders, expedite drug development, and improve FDA, industry, and researchers' ability to assess drugs' safety and effectiveness. These initiatives may include, but not be limited to, the following:  Enhancing regulatory science and expediting drug development  Facilitating rare disease drug development  Advancing the use and understanding of innovative statistical methods and trial designs  Advancing development of combination product review  Enhancing the use of real world evidence for use in regulatory decision-making  Enhancing regulatory decision tools to support drug development and review  Enhancing the Incorporation of the Patient's Voice in Drug Development and Decision-making  Enhancing Benefit-Risk Assessment in Regulatory decision-making  Advancing model-informed drug development  Enhancing capacity to review complex innovative designs  Enhancing capacity to support analysis data standards for product development and Review Several key areas of research interest are described in greater detail below: Enhancing the Incorporation of the Patient's Voice in Drug Development and decision-making In PDUFA V, FDA conducted a series of Patient-Focused Drug Development (PFDD) meetings with the aim to more systematically gather patients' perspectives on their condition and available therapies to treat their condition. Under PDUFA VI, FDA proposes to build on these efforts to bridge from PFDD meetings to fit-for-purpose tools to collect meaningful patient input that can be incorporated into regulatory review and used by all stakeholders to assess drugs' benefit to patients. The field is constantly evolving and this work cannot be done in isolation. Many professional groups and research teams around the world have developed and are developing templates, checklists, and guidelines for different aspects of gathering and interpreting patient experience data, and many such documents already exist for patient reported outcomes. FDA seeks to identify the challenges and opportunities within the current collection and use of patient input. Advancing research in this area will help ensure that drug development is more responsive to patients' input and needs. Structured Approaches to Enhancing FDA’s Assessment of Benefits and Risks in Human Drugs FDA’s qualitative Benefit-Risk (B-R) Framework serves as the foundational element of CDER’s and CBER’s structured benefit-risk assessment. With implementation of the B-R Framework gaining solid ground within FDA’s drug review, there is now an opportunity to systematically explore and advance the use of more technical approaches within the qualitative framework to inform benefit-risk assessment in targeted cases. These may include structured techniques to characterize underlying judgments and uncertainties inherent in the assessment, methods to compare and/or weigh benefits and risks, and other qualitative and quantitative approaches aiming to improve the quality of the decision-making process in some cases. FDA recognizes that the agency’s efforts to develop a more structured approach to benefit-risk assessment could be complemented by further engagement of stakeholders and other parties, providing an opportunity to share challenges and lessons learned in applying a more structured approach to benefit-risk decision-making. Such research would benefit FDA, drug manufacturers, health policy analysts, and others who make decisions about the benefits and risks of drugs. APPROACH Achieving these research objectives requires bringing together input from a wide range of relevant external subject matter experts and other interested public stakeholders. In addition, this input process should be timely, well-informed, candid, thoughtful, thorough, and well-documented. FDA is therefore seeking to establish a cooperative agreement with a qualified awardee with experience in the conduct of the needed research, workshops and other meetings, and related work. The goal of this collaboration is to advance research in several key areas of interest to FDA and external stakeholders by convening stakeholders with diverse expertise, and to inform and support the advancement of regulatory science priorities, particularly those identified in PDUFA VI and the 21st Century Cures Act. Through a series of meetings, workshops, webinars, and/or workgroups, the awardee would provide effective opportunities for engagement of these stakeholders to pursue research on these topics. In addition to gathering input from selected stakeholder groups, the awardee will conduct background research prior to expert engagement, use input gathered to develop white papers and research reports, and communicate updates on the progress of its regulatory science research to broader audiences. Specific objectives of this collaboration would include: Working collaboratively with FDA to identify and prioritize pressing research issues related to the key areas of interest identified in PDUFA VI and the 21st Century Cures Act Conducting research and reviews of relevant literature to plan the focus of sessions in which experts are convened to provide critical input on regulatory science issues; Convening expert stakeholders in focused, substantive discussions of these issues, and identify and explore potential strategies for resolving them Developing reports that summarize the background research and discussion at each meeting, and posting these reports for public access to promote external interest and advance public understanding of key research issues; and Helping to make research results and reports actionable by FDA, industry, and other stakeholders. The timeframe of the project is five years. Conducting this research will require a multi-year effort, involving a variety of topics, stakeholder groups, and issue specific activities. We propose to establish a five-year collaboration, aligned with the reauthorization of the PDUFA agreement, to facilitate the successful, in-depth exploration of key research topics. Inherent in the cooperative agreement award is substantive involvement by the awarding agency. Accordingly, FDA will have substantive involvement in the programmatic activities funded by this Cooperative Agreement. Substantive involvement includes, but is not limited, to the following: FDA will provide guidance, and assistance in the identification of key research objectives.

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