Forum on Drug Discovery, Development, and Translation: 2016 Annual Report (2017)

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Looking Forward Forum Activities in 2017 Forum Meetings The Forum membership will meet in March, July, and October 2017 to continue its discussions of key problems and strategies in the discovery, development, and trans- lation of drugs. Forum workshop planning committees, working groups, and Action Collaboratives will convene activities to discuss and act on priority areas identified by them, including the following: Workshops: Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development—A Workshop The Forum, in collaboration with the Roundtable on Genomics and Precision Health (the Roundtable), will convene in March 2017 a follow-up to a workshop that the two groups held in March 2016 on Deriving Drug Discovery Value from Large-Scale Genetic Bioresources. The 2017 workshop will examine later stages in the drug development pipeline, including the design and implementation of genetics-based clinical trials. The workshop will examine and discuss successes, challenges, and best practices for effec- tively utilizing genetic information in the design and implementation of clinical trials to support the development of precision medicines, including exploring the potential advan- tages and disadvantages of such trials across a variety of disease areas. The workshop will consider how clinical trials with genetically identified participants can enable more efficient and effective drug development; highlight ongoing genetics-based clinical trials across a variety of diseases to examine best practices and lessons learned; learn about the logistical challenges and successes associated with genetics-based clinical trial design; and examine possible mechanisms to engage participants and improve enrollment into clinical trials based on genetic characteristics. July 12 June 27–28 South Africa Workshop Summary Workshop: Public Engagement Release: The Emerging Threat of and Clinical Trials: New Models Multidrug-Resistant Tuberculosis: Global October 4–5 and Disruptive Technologies and Local Challenges and Solutions Forum Meeting #20 June 28–29 September 20–21 Forum Meeting #19 Workshop: Strengthening a Workforce for Innovative Regulatory Science in Therapeutics Development 

Looking Forward: Forum Activities in 2017 Examining the Impact of Real-World Evidence on Medical Product Development— A Workshop Forum member Margaret Anderson, center, discusses the launch of the The Forum held a workshop on Forum Action Collaborative on the Science of Patient Input at the July 18 Forum meeting. (Left to right) Guest speaker Kim McCleary (FasterCures), October 19, 2016, titled Real-World and Forum members Kate Knobil, Margaret Anderson, and Brian Strom. Evidence Generation and Evaluation of Therapeutics, which examined opportunities for and challenges of incorporating real- world evidence into medical product development and evaluation. This workshop built on prior work of the Forum in recent years that focused on clinical trials, data sharing, and regulatory science, and will serve as the springboard for a workshop that will more fully examine the issues highlighted at the October 2016 workshop. As part of FDA’s continued focus on building a national governance system for evidence generation, the focus of this workshop will be on the generation and utilization of real-world evidence to evaluate efficacy, effectiveness, tolerability, and safety for review of both new indications and postapproval studies. The workshop will provide a format for examining the practi- calities of collecting real-world data and deriving and applying real-world evidence for the evaluation of medical products, including drugs, biologics, and devices. Workshop presentations and discussions will include consideration of sources of real-world data; gaps in data collection activities and priority areas that real-world evidence incorpora- tion could address; standards and methodologies for collecting and analyzing real-world evidence in support of new indications or postapproval studies; applications for using real-world evidence to supplement traditional clinical trials; mechanisms for determin- ing which discrete types of real-world evidence could support regulatory decisions; and operational challenges and barriers for generating and incorporating real-world evidence in the context of a learning health system. November 7–8 June 4–5 Workshop: Envisioning a Transformed Workshop: Maximizing the Goals of the Clinical Trials Enterprise in the United March 13–14 Cures Acceleration Network to Accelerate the States: Establishing an Agenda for 2020 Forum Meeting #21 Development of New Drugs and Diagnostics 2012 November 15 June 5 Russia Workshop Summary Release: The New Profile Forum Meeting #22 of Drug-Resistant Tuberculosis: A Global and Local Perspective 

21st Century Clinical Trials—A Workshop Clinical trials are the cornerstone of medical product development—evaluating efficacy and identifying safety issues of new drugs—and a necessary requirement for bringing new therapies to patients. The cost and duration of clinical trials have escalated dramatically in the past 20 years, greatly hampering the introduction of new therapies and driving recruitment to develop- ing countries, raising questions about generalizability, data quality, and participant protections. Technological advances have opened the possibility for new trial designs that could increase par- ticipation in the United States, particularly through the engagement of community health centers or other local venues outside of traditional academic centers. Such trial designs could create the potential for more equitable and representative recruitment of participants as well as for more facile uptake of trial findings into clinical practice. Since 2009, the Forum has been engaged in a focused effort, anchored by a multi-workshop series, to address 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 will provide a venue to explore opportunities for a modern clinical trials enterprise in light of other transfor- mative changes in the drug development and health system sectors. Participants will examine the current state of the enterprise, highlight opportunities to address barriers to further improve- ments, explore incentives that could foster enhanced collaboration, and discuss mechanisms for providers and health systems to take part in and encourage patient participation in clinical trials. Action Collaboratives: Clinical Trial Site Standards Harmonization—Action Collaborative Collaborative participants are overseeing a second phase of the Collaborative activity, which includes the collection, analysis, and assessment of how clinical trial site standards currently in use by key stakeholders could be harmonized. In 2017, the Collaborative will release an NAM October 4–5 November 26–27 Workshop: Sharing Clinical Research Data (in Workshop: Large Simple Trials and Knowledge Generation collaboration with the Neuroscience Forum, National in a Learning Health System (in collaboration with the Cancer Policy Forum, and Genomics Roundtable) Roundtable on Value & Science-Driven Health Care) July 31–August 1 October 23–24 December 19 Workshop: Developing and Strengthening the Forum Meeting #23 Action Collaborative Meeting #1: Developing a National Global Supply Chain for Second-Line Drugs for Accreditation System to Improve Clinical Trial Performance Multidrug-Resistant Tuberculosis 

Looking Forward: Forum Activities in 2017 Discussion Paper highlighting its work and a summary of the consultant-generated analy- sis of a sample of stakeholder standards applied to clinical trial sites. Sharing Clinical Trial Data—Action Collaborative In 2017, the Forum will continue to serve as a sounding board for global collaborations to advance data sharing. The Forum will also support the activities of the workstream addressing Data Sharing Principles for Nonprofit Clinical Trial Funders by convening a meeting of stakeholders to review principles and share experiences and information about ongoing data sharing efforts. Science of Patient Input—Action Collaborative Though significant successes have been realized in advancing the field of patient engage- ment and the science of patient input across the biomedical enterprise, there still exists a critical need to better identify priority areas for the science of patient input; identify best practices and principles for measuring patient input; advance and coalesce devel- oped principles and taxonomies; and maintain an ongoing awareness and spirit of col- laboration among the public, private, and regulatory sectors. Under the auspices of the Forum, an Action Collaborative has been convened to identify the areas of most critical need that can be best advanced through cross-sector collaboration in the neutral venue of the National Academies. In 2017, Collaborative members will meet to review, discuss, and decide on the group’s specific next steps, including potential activities and outputs. Improving the Drug Development Process—Action Collaborative The Forum will launch, in mid-2017, an Action Collaborative that will explore avenues for improving the drug development process. The Collaborative could address, for example, the causes of late-stage failures and novel approaches that might address them. January 15 February 13–14 Workshop Summary Release: Developing and Workshop: International Regulatory Harmonization Strengthening the Global Supply Chain for Second- Amid Globalization of Biomedical Research and Line Drugs for Multidrug-Resistant Tuberculosis Medical Product Development 2013 January 16–18 February 12 June 3 China Workshop: The Global Crisis Forum Meeting #24 Forum Meeting #25 of Drug-Resistant Tuberculosis and Leadership of the BRICS Countries