Forum on Drug Discovery, Development, and Translation: 2018 Annual Report (2019)

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Reflecting Back: Forum Activities in 2018 Members of the Forum convene Forum Membership Meetings Members of the Forum convene several times each year to identify, discuss, and several times each year to act on key problems and strategies in the discovery, development, and translation identify, discuss, and act on key of drugs. In 2018, the Forum membership focused on diverse topics related to the problems and strategies in the Forum’s priorities, including issues for emerging biotech companies; transforming clinical trials through digital health; improving the drug development process through discovery, development, and examining late-stage failures; real-world evidence and medical product development; translation of drugs. harmonizing clinical trial site standards; data-sharing principles for nonprofit clinical trial funders; the science of patient input in medical product R&D; innovative models to spur translational research and drug discovery; artificial intelligence applications for drug discovery and development; and other policy updates relevant to drug discovery, development, and translation. To supplement the perspectives and expertise of its members, the Forum holds public workshops to focus attention on critical areas of drug development. Summaries of these meetings are disseminated to the public through the Forum website and published proceedings. Workshops Examining the Impact of Real-World Evidence on Medical Product Development: A Workshop Series The Forum convened a three-part workshop series, sponsored by FDA, examining how real-world evidence development and uptake could enhance medical product development and evaluation. The workshops advanced the discussions and common knowledge about complex issues relating to the generation and use of 6 

Reflecting Back: Forum Activities in 2018 Forum member Ellen Sigal shares her thoughts at the March 2018 Forum meeting. real-world evidence, including fostering the development and implementation of the science and technology of real-world evidence generation and utilization. Workshop One: Incentives (September 19–20, 2017) focused on how to align incentives to support the collection and use of real-world evidence in medical product review, payment, and delivery, including the incentives needed to address barriers impeding the uptake of real-world evidence, such as transparency. Workshop Two: Practical Approaches (March 6–7, 2018) was a “Town Hall” style meeting with in-depth audience discussion and active participation to illuminate which types of data may be appropriate for specific purposes and to discuss practical approaches for data collection and evidence use. Workshop Three: Application (July 17–18, 2018) examined approaches for operationalizing the collection and use of real-world evidence. 7 

Advancing the Science of Patient Input in Medical Product R&D: Towards a Research Agenda: A Workshop The Forum convened a 1-day workshop on May 9, 2018, focused on the science of patient input for pre-market medical product R&D (including pre-discovery, discovery, pre-clinical development, and clinical development), with consideration of downstream regulatory and post-market decision making. At this workshop, subject- matter experts representing a range of disciplines engaged in presentations and discussions to: • Examine the current state of the science, including successes and limitations of current efforts, for soliciting and incorporating patient input in pre-market R&D. • Explore gaps in the knowledge base and other barriers that impede progress, To supplement the perspectives including but not limited to: and expertise of its members, the – parameters for soliciting and incorporating different types of patient input Forum holds public workshops to along the spectrum of medical product R&D; focus attention on critical areas – availability, accessibility, and applicability of data sources for patient input; – tools and methodologies for collecting and analyzing patient input to produce of drug development. Summaries scientifically rigorous, credible evidence; and of these meetings are – understanding of the ethical and value considerations for soliciting and disseminated to the public incorporating patient input into medical product R&D decision making. through the Forum website and • Review a potential framework for and components of a research agenda for addressing the gaps or barriers to realizing a science of patient input. published proceedings. Virtual Clinical Trials: Challenges and Opportunities: A Workshop The Forum convened a 2-day workshop on November 28–29, 2018, which provided a venue to explore opportunities for a modern clinical trials enterprise capable of more efficiently answering questions about medical products by bringing participants closer to the clinical trial through the use of digital health tools. Clinical trials are a cornerstone of medical product development—supporting the evaluation of efficacy and the identification of safety issues of new drugs and devices—and a necessary regulatory requirement for bringing novel therapies to market. Since 2009, the 8 

Reflecting Back: Forum Activities in 2018 Forum member Tanisha Carino participates in a small group discussion during the plenary session of the Advancing the Science of Patient Input workshop in May 2018. Forum has been engaged in a focused effort, anchored by a multi-workshop series, to address the challenges facing the U.S. clinical trials enterprise and to engage stakeholders in an ongoing discussion of potentially transformative strategies to improve the efficiency and effectiveness of clinical trials. This workshop built on this work and examined opportunities for a modern clinical trials enterprise in light of digital health tools. Participants highlighted opportunities for systemic improvements in the clinical trials enterprise, considered novel platform designs and digital health tools to increase efficiency and enhance the interface between clinicians and participants in a clinical trial, and discussed digital platforms to facilitate recruitment and maintain diverse participation in clinical trials. 9