Forum on Drug Discovery, Development, and Translation: 2010 Annual Report (2011)

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Forum Initiatives Addressing the Approach to Drug Development: Problems and Opportunities Recent years have seen both extraordinary opportunity and complex challenges in pharmaceutical innovation. Advances in genomic science, systems biology, and cell-based technologies have led to exciting new avenues of biomedical research and drug discov- ery and have given us glimpses of the “personalized” future of medicine. Yet despite these advances, the pathway from basic science to new therapeutics faces challenges on many fronts. The scientific challenges in finding novel drug targets are profound, and the translational divide results in only a small fraction of investigational new drugs reaching FDA approval and the patients who need them. The Forum has explored these issues from many perspectives—emerging technology platforms, regulatory efficiency, intellectual property concerns, the potential for precompetitive collaboration, and innovative business models that address the “valley of death”—and in the process has served to generate new ideas, inform policy and legislation, and provide a critical sounding board for FDA, NIH, and industry. Promoting and Enhancing the Scientific Basis for the Regulation of Drugs Over the past several years, the Forum has focused its attention on the scientific basis for the regulation of drugs. The Forum held a series of meetings on drug safety over the course of 2009, addressing topics of personalized medicine and the genetic basis of adverse events and advances in post-market drug safety since the passage of the Food and Drug Administration Amendments Act of 2007 (FDAAA), as well as Commissioner Peggy Hamburg’s vision for FDA. In February 2010, the Forum held a workshop that examined the state of the science of drug regulation and considered approaches for enhancing the scientific basis of regulatory decision making. Also, since 2007, the Forum has hosted pub- lic meetings designed to engage congressional staff and the broader policy community on current topics in drug development. October 24 November 5 March 13 Workshop: Assessing and Workshop: Addressing the Threat Discussion Series: FDA Community Accelerating Development of of Drug Resistant TB: A Realistic Update on Personalized Medicine and Biomarkers for Drug Safety 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 TB 

Forum Initiatives Transforming Research and Fostering Collaborative Research The Forum recognizes that the critical link between bench and bedside in devel- oping new diagnostics and therapeutic approaches is clinical research. The Forum tackled the issue head-on by establishing NIH’s Francis Collins with HHS Secretary Kathleen Sebelius an initiative to examine the state of clinical trials in the United States, identify areas of strength and weakness in our current clinical trial enterprise, and consider transformative strategies for enhancing the ways in which clinical research is organized and conducted. The first workshop, held in October 2009, considered case studies in four disease areas to derive lessons that can be applied throughout clinical research. The Forum held two discussion meetings in 2010 to address issues of management of conflict of interest in biomedical research and regulatory and administrative impediments to the efficient and effective conduct of clinical trials. Meetings for 2011 are under development that will address moving toward greater public engagement in and understanding of the clinical trial enterprise, and envisioning a framework for a transformed national clinical trial enterprise. Also in 2010, the Forum convened two working groups in connection with its clinical trials initiative to address major themes and problems afflicting the U.S. clinical trial enterprise: one working group considered issues pertaining to the regulation and infrastructure of clinical trials in the United States, and the other looked at ways to enhance public engagement in the clinical research enterprise. 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 US 

Developing Drugs for Rare and Neglected Diseases and Addressing Urgent Global Health Problems The Forum is sponsoring a series of workshops on the global problem of MDR TB. The Forum held a foundational workshop in Washington, DC in November 2008, for which it commissioned a Paul Farmer, Partners In Health paper from Partners In Health. Additional work- shops are under way in the four countries with the highest MDR TB burden—South Africa (held March 2010), Russia (held May 2010), India (to be held April 2011) and China (anticipated late 2011 or early 2012). Also in 2011, the Forum will convene a focused initiative addressing the global drug supply chain for quality-assured second-line drugs for tuberculosis. Promoting Public Understanding of Drug Development Successful introduction of new therapeutic entities requires testing in an informed and motivated public. The Forum has spent concerted effort to understand what limits public par- ticipation and how to enhance more widespread acceptance of the importance of advancing therapeutic development through public participation in the drug development process. Forum meetings held in the spring and fall of 2010 addressed these issues. The Forum plans to continue to work with multiple stakeholders to improve public understanding of and participation in the drug development process. February 26 March 3–4 Workshop: Building a National South Africa Workshop: The Framework for the Establishment Emerging Threat of Multidrug- August 5–6 of Regulatory Science for Resistant Tuberculosis April 29–30 Discussion Series: Drug Development Forum Meeting #15 Conflict of Interest (Forum Meeting #16) 2010 February 22–24 May 26–27 Workshop: The Public Health Emergency Medical Countermeasures Russia Workshop: The New Profile of Drug Enterprise (collaboration with the Medical Preparedness Forum) Resistant Tuberculosis 

Reports Released in 2010 Forum Initiatives The Public Health Emergency Medical Countermeasures Enterprise—Workshop Summary Released: April 8, 2010 At the request of the Secretary of the U.S. Department of Health and Human Services and the Assistant Secretary for Preparedness and Response, the IOM held a workshop February 22–24, 2010, to address challenges facing the Public Health Emergency Medical Countermeasures Enterprise. Workshop participants discussed federal policies and proce- dures affecting the research, development, and approval of medical countermeasures and explored opportunities to improve the process and protect Americans’ safety and health. The Forum on Medical and Public Health Preparedness for Catastrophic Events and the Forum on Drug Discovery, Development, and Translation collaborated on this workshop and summary. This document summarizes the workshop. Transforming Clinical Research in the United States: Challenges and Opportunities—Workshop Summary Released: August 2, 2010 The IOM held a public workshop October 7–8, 2009, to evaluate the state of clinical research in the United States, and to identify strategies for improving clinical trials’ efficiency and effectiveness. Clinical trial experts discussed their successes, failures, and challenges in conducting clinical research. This document summarizes the workshop. Building a National Framework for the Establishment of Regulatory Science for Drug Development—Workshop Summary Released: October 8, 2010 The IOM held a public workshop on February 26, 2010, to examine the state of regulatory science and to consider approaches for enhancing it. The workshop provided an opportunity for stakeholders to clarify and explore the concept of regulatory science, examine how it can be used to improve regulatory decision making, consider its funding needs, and contemplate alternative mechanisms and institutional frameworks for its development and application. This document summarizes the workshop. 2011 October 29 Discussion Series: Administrative and Regulatory Inefficiencies in Clinical Trials (Forum Meeting #17)