Forum on Drug Discovery, Development, and Translation: 2014 Annual Report (2015)

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Looking Forward Forum Activities in 2015 Forum Meetings The Forum membership will meet in March, June, and October 2015 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 to discuss and act on the following topics: Action Collaboratives: Disruptive Innovation and the Transformation of the Drug Development and Translational Science Enterprise—Action Collaborative Many argue that the current paradigm for drug discovery and development requires disruptive innovation to break out of a crisis in research and develop- ment (R&D) productivity. Evidence suggests that industries are almost always disrupted from the outside by new technologies they were slow to embrace, new business models they wrongly dismissed, or policy changes they thought they could keep at bay. The pharmaceutical industry offers many opportunities for disruption in each of these areas. The Forum has convened an action collabora- tive that has set out to identify and highlight potentially breakthrough ideas and visionary approaches to the “drug development and translational science enter- prise of the future.” The effort has addressed new technologies (e.g., biosensors, apps and telemetry, synthetic biology, new delivery technologies); new business models (e.g., crowdsourcing platforms, drug repurposing, virtual companies, or clinical trials); and policy issues (e.g., pricing/reimbursement, patent law, data transparency). The effort has included a data gathering phase, along with a review November 3–4 June 13 Workshop: Adverse Drug Event March 28–29 Workshop: Addressing Reporting: The Roles of Consumers Forum Meeting #4 the Barriers to Pediatric and Health-Care Professionals Drug Development 2006 May 30–31 Workshop: Understanding the Benefits and Risks of Pharmaceuticals 

Looking Forward: Forum Activities in 2015 phase, and includes the preparation of a Perspective. The Forum will convene one or more workshops in 2015 to further explore issues identified through this effort, including defining a drug development paradigm for the future and considering the impact of new technologies on drug development. Mapping and Connecting the Biomedical Innovation Ecosystem—Action Collaborative The biomedical innovation ecosystem is a dynamic network of activity. Standardizing and bringing clarity to this complex process could help to set a vocabulary and allow more fluid dialogue among ecosystem participants to encourage further inno- vation. It could also facilitate ongoing discussion to help frame, map, and synergize activities across the biomedical innovation ecosystem. Defining key terms and 2007 June 27–28 October 24–25 Forum Meeting #5 Forum Meeting #6 

locating complex activities, such as translational science and regulatory science, within the biomedical innovation ecosystem landscape might help to articulate problem areas and provide opportunities to learn from local environments where the system is working more efficiently. The goal of this collaborative is to enable discussion and to identify these rate-limiting steps in order to facilitate process improvement efforts within the biomedical innovation ecosystem. Sharing Clinical Trial Data—Action Collaborative Sharing clinical trial data can facilitate more efficient and effective development of better medicines, diagnostics, and procedures for the ultimate benefit of patients. At the same time, sharing data presents risks, burdens, and challenges that need to be April 23–24 Workshop: Emerging Safety October 15–16 Science, FDA (Forum Meeting #7) Forum Meeting #8 March 12 September 14 Symposium: The Future of Drug Discussion Series: From Patient Safety: Challenges for FDA Needs to New Drug Therapies 

Looking Forward: Forum Activities in 2015 addressed by a broad set of stakeholders. These opportunities and challenges were laid out in the IOM report Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. The report calls upon stakeholders to foster a culture of shar- ing and offers a blueprint for action within and across sectors. Four IOM forums provided momentum and a framework for initiating the IOM consensus study and are working together again to form a platform that could support coordination and collaboration among stakeholders engaged in data sharing initiatives through convening and other activities. Workshops: Priorities to Advance the Field of Regulatory Science: An Update on Progress and a Forward-Looking Agenda—Workshop In the face of rapid advances in medicine and biomedical science, FDA faces pres- sure to keep pace with new technologies and develop the expertise necessary to regulate those technologies as they emerge. The Forum, in collaboration with the Burroughs Wellcome Fund, will convene a public workshop that will discuss issues related to the development of the discipline of innovative regulatory science, focus- ing on infrastructure, systems, and workforce. The workshop will explore current regulatory science priorities and strategies in federal, academic, and private sector settings; consider the current state of regulatory science as a discipline; explore the core components of a robust discipline of innovative regulatory science; consider gaps and key opportunities to address needs to support the discipline of innovative regulatory science; and examine needs and barriers to collaboration among, across, and within the public and private sectors. June 23 April 21 Workshop: Breakthrough November 30 Discussion Series: Business Models: Drug Discussion Series: A Conversation Science at FDA: Challenges Development for Rare with Tony Fauci and Opportunities and Neglected Diseases 2008 February 20–21 June 23–24 Discussion Series: Comparative Symposium: Diseases and Individualized Effectiveness (Forum Meeting #9) Therapies (Forum Meeting #10) 

In addition, the Forum will co-convene the following workshops in collaboration with other IOM Forums: Financial Incentives to Support Unmet Medical Needs for Nervous System Disorders—Workshop The global burden of nervous system disorders is projected to significantly increase over time. Although there have been recent international initiatives to better understand the human brain in order to develop new therapeutics, several large pharmaceutical companies have decreased investment or even withdrawn from their neuroscience research programs. The perceived high risk and low probability of success has made the neuroscience sector less attractive than other therapeutics areas for R&D, despite the large market potential. As a result, patients are often left with few, if any, options for treatment; thus, there is a need to consider policy options to increase private-sector investment in R&D for nervous system disorders. The Forum, in collaboration with the IOM’s Forum on Neuroscience and Nervous System Disorders, convened this public workshop, which explored opportunities to foster private-sector innovation by supporting new investments directed toward the development of novel therapeutics to meet unmet needs for nervous system disorders. Enabling Rapid Medical Countermeasure Response to Emerging Threats—Workshop With the global attention to recent large-scale outbreaks and their public health and medi- cal consequences, it has become clear that the current medical countermeasure (MCM) response system is not well adapted to rapidly respond to a large number of diverse threats through adequate development and production of vaccines, therapeutics, diagnostic tools, and other nonpharmaceutical interventions. The Forum, in collaboration with the IOM’s Forum on Medical and Public Health Preparedness for Catastrophic Events, will October 24 November 5 March 13 Workshop: Assessing and Workshop: Addressing the Threat Discussion Series: FDA Community Accelerating Development of of Drug-Resistant Tuberculosis: A Update on Personalized Medicine and Biomarkers for Drug Safety Realistic Assessment of the Challenge the Genetic Basis of Adverse Events 2009 February 23 November 3 Capitol Hill Briefing: Growing Forum Meeting #11 Threat of Drug-Resistant Tuberculosis 

Looking Forward: Forum Activities in 2015 co-­ onvene a public workshop that will examine how to better enable rapid and c nimble private-sector engagement in the discovery, development, and translation of MCMs. This meeting will explore what policies, guidance, and resources exist to guide decision making within the government and how the business and opera- tional models employed by the private sector are affected by policies and guidance and available resources set forth by the U.S. government. Further, the discussions at this meeting will be designed to identify and discuss what is needed to ensure that the private sector can respond in a rapid, nimble manner to ensure the availability of MCMs and explore advances made by the Public Health Emergency Medical Countermeasures Enterprise to improve MCM development and translation. April 27 September 2 Workshop: Streamlining Discussion Series: FDA October 15–16 Clinical Trial and Material Community Update on Forum Meeting #14 Transfer Negotiations Post-Market Drug Safety July 10 Symposium: Drug Regulation with October 7–8 April 27–28 FDA Commissioner Peggy Hamburg Workshop: Transforming Clinical Forum Meeting #12 (Forum Meeting #13) Research in the United States