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Suggested Citation:"Forum Initiatives." Institute of Medicine. 2012. Forum on Drug Discovery, Development, and Translation: 2011 Annual Report. Washington, DC: The National Academies Press. doi: 10.17226/26112.
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Suggested Citation:"Forum Initiatives." Institute of Medicine. 2012. Forum on Drug Discovery, Development, and Translation: 2011 Annual Report. Washington, DC: The National Academies Press. doi: 10.17226/26112.
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Suggested Citation:"Forum Initiatives." Institute of Medicine. 2012. Forum on Drug Discovery, Development, and Translation: 2011 Annual Report. Washington, DC: The National Academies Press. doi: 10.17226/26112.
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Suggested Citation:"Forum Initiatives." Institute of Medicine. 2012. Forum on Drug Discovery, Development, and Translation: 2011 Annual Report. Washington, DC: The National Academies Press. doi: 10.17226/26112.
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Page 17

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Forum Initiatives Addressing the Approach to Drug Development Despite exciting scientific advances, the pathway from basic science to new therapeutics faces challenges on many fronts. New paradigms for discovering and developing drugs are being sought to bridge the ever-widening gap between scientific discoveries and transla- tion of those discoveries into life-changing medications. The Forum has explored these issues from many perspectives—emerging technology platforms, regulatory efficiency, intellectual property concerns, the potential for precompetitive collaboration, and innova- tive business models that address the “valley of death.” Strengthening the Scientific Basis of Drug Regulation There has been considerable and increasing attention to the need for robust science to underlay and serve as the basis for regulatory decision making. “Regulatory science” involves the development and application of scientific tools and methodologies to improve the development, review, and oversight of new therapeutics. Recent rapid advances in innovative drug development science present an opportunity for revolutionary developments of new scientific techniques, therapeutic products, and applications. For example, advances in genomic science, systems biology, and cell-based technolo- gies have led to exciting new avenues of biomedical research and drug discovery and have given us glimpses of the “per- Regulatory Science Workforce Workshop panelists Steven Galson and Clifford Lane sonalized” future of medicine. As FDA July 12 South Africa Workshop Summary November 7–8 Report Release: The Emerging Threat of Workshop: Envisioning a Transformed Multidrug-Resistant Tuberculosis: Global Clinical Trials Enterprise in the United June 28–29 October 4–5 States: Establishing an Agenda for 2020 and Local Challenges and Solutions Forum Meeting #19 Forum Meeting #20 2012 June 27–28 September 20–21 Workshop: Public Engagement and Clinical Workshop: Strengthening a Workforce for Innovative Trials: New Models and Disruptive Technologies Regulatory Science in Therapeutics Development

Forum Initiatives has recognized, both science and infrastructure are key components in ensuring that the highest-quality regulatory decisions are made to ensure the safety and public health of the U.S. population while fostering, not inhibiting, innovation in an increasingly challenging and globalized business environment. The Forum has focused considerable attention on the need to develop, support, and enhance a discipline of regulatory science as an essential component of the drug discovery enterprise and translational sciences. Activities under- taken by the Forum, including symposia and workshops, have contributed to the defin- ing and establishment of regulatory science, and have considered in-depth issues such as cutting-edge drug safety science, development of biomarkers for safety, personalized medicine, adverse event reporting and post-marketing safety surveillance, infrastructure and workforce needs, and collaborative approaches to support and sustain regulatory sci- ence and drug development. Transforming Clinical Research and Fostering Collaborative Research Clinical research is the critical link between bench and bedside in developing new therapeutics. Significant infrastructural, cultural, and regula- tory impediments challenge efforts to integrate clinical trials into the health care delivery system. Collaborative, cross-sector approaches can help articulate and address these key challenges and foster systemic responses, such as a comprehen- sive clinical trials infrastructure and harmoniza- tion of regulatory standards and institutional Forum member James Doroshow June 4-5 Workshop: Maximizing the Goals of the March 13–14 Cures Acceleration Network to Accelerate Forum Meeting #21 the Development of New Drugs and October 23–24 Diagnostics (Forum Meeting #22) Forum Meeting #23 November 15 July 31–August 1 Russia Workshop Summary Report Release: The New Profile Workshop: Developing and Strengthening the Global Supply Chain of Drug-Resistant Tuberculosis: A Global and Local Perspective for Second-Line Drugs for Multidrug-Resistant Tuberculosis

processes. The Forum has established an initiative to examine the state of clinical trials in the United States, identify areas of strength and weakness in our current clinical trials enterprise, and consider transformative strategies for enhancing the ways in which clinical trials are orga- nized and conducted. Ongoing efforts will examine issues with the clinical and translational research workforce, including academic career paths. Developing Drugs for Rare and Neglected Diseases and Addressing Urgent Global Health Problems There are a number of diseases for which there is extensive unmet medical need and a lack of therapeutics, where the population affected by the disease is very small (rare/orphan diseases) or the market for the therapeutic product is underdeveloped or inaccessible (neglected diseases). For these diseases, economic markets alone are insufficient to incentivize product development, and financial and regulatory incentives have been sought to stimulate therapeutics development and access to products. The Forum has recognized that the emergence of DR TB is a global threat and warrants focused attention to address the multitude of complex problems in drug develop- ment and access to therapeutics for patients affected by this neglected disease. The Forum is sponsoring a series of international collaborative workshops on DR TB, with a focus on drug delivery issues in the four countries with the highest burden of DR TB. The Forum has also held focused meetings and public workshops considering particular issues impeding the development of orphan drug products and other areas of unmet medical need, such as MCMs. Promoting Public Understanding of Drug Development In these increasingly resource-constrained times, it is essential that the public understand both the complexity of and the need for the drug development process. Collaborative efforts to de- risk drug development are reported to be limited in some cases by a lack of public understanding of the need to support innovation and collaboration among the federal government, academia, industry, and other entities such as voluntary health organizations. In addition, 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 engagement and how to enhance more widespread acceptance of the importance of advancing therapeutic development through public participation in and support for the drug development process.

Reports Released in 2011 Forum Initiatives The Emerging Threat of Drug-Resistant Forum on Drug Discovery, Development, and Translation Tuberculosis in Southern Africa: Global and the Forum on Medical and Public Health Preparedness and Local Challenges and Solutions: for Catastrophic Events held a workshop to examine meth- Summary of a Joint Workshop ods to improve the development, evaluation, approval, and Released: March 24, 2011 regulation of MCMs. TB kills approximately 4,500 people Public Engagement and Clinical worldwide every day. While most cases of Trials: New Models and Disruptive TB can be treated with antibiotics, some Technologies: Workshop Summary strains have developed drug resistance that Released: October 14, 2011 makes their treatment more expensive, more toxic, and less effective for the patient. The IOM Forum on Drug Discovery, Clinical trials provide essential infor- Development, and Translation and the Academy of Science mation needed to turn basic medical of South Africa held a workshop to discuss ways to fight the research findings into patient treatments. growing threat of DR TB. New treatments must be studied in large numbers of humans to find out whether The New Profile of Drug-Resistant they are effective and to assess any harm that may arise Tuberculosis in Russia: A Global And from treatment. There is growing recognition among many Local Perspective: Summary of a Joint stakeholders that the U.S. clinical trials enterprise is unable Workshop to keep pace with the national demand for research results. The IOM Forum on Drug Discovery, Development, and Released: April 8, 2011 Translation, along with the Mount Sinai School of Medicine, DR TB presents a number of signifi- held a workshop to engage stakeholders and experts in cant challenges in terms of controlling a discussion about possible solutions to improve public its spread, diagnosing patients quickly engagement in clinical trials. and accurately, and using drugs to treat patients effectively. In Russia in recent decades, the rise of Strengthening a Workforce for these strains of TB, resistant to standard antibiotic treatment, Innovative Regulatory Science in has been exacerbated by the occurrence of social, political, Therapeutics Development: Workshop and economic upheavals. The IOM Forum on Drug Discovery, Summary Development, and Translation, in conjunction with the Released: December 21, 2011 Russian Academy of Medical Sciences, held a workshop to discuss ways to fight the growing threat of DR TB. The development and application of regu- latory science—which FDA has defined Advancing Regulatory Science as the science of developing new tools, for Medical Countermeasure standards, and approaches to assess the Development: Workshop Summary safety, efficacy, quality, and performance of FDA-regulated Released: June 15, 2011 products—calls for a well-trained, scientifically engaged, and motivated workforce. FDA faces challenges in retaining regu- Whether or not the United States has latory scientists and providing them with opportunities for safe and effective MCMs—such as vac- professional development. In the private sector, advancement cines, drugs, and diagnostic tools— of innovative regulatory science in drug development has available for use during a disaster can not always been clearly defined, well-coordinated, or con- mean the difference between life and nected to the needs of the agency. As a follow-up to a 2010 death for many Americans. FDA and the scientific com- workshop, the IOM Forum on Drug Discovery, Development, munity at large could benefit from improved scientific and Translation held a workshop to provide a format for tools and analytic techniques to undertake the complex establishing a specific agenda to implement the vision and scientific evaluation and decision making needed to make principles relating to a regulatory science workforce and dis- essential MCMs available. At the request of FDA, the IOM ciplinary infrastructure as discussed in the 2010 workshop.

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In 2011, there was a need for innovation across the biomedical science, policy, and business sectors. The National Academies’ Forum on Drug Discovery, Development, and Translation (the Forum) contributed to the conversation by hosting public workshops on envisioning a framework for a transformed clinical trials enterprise, and advancing the discipline of regulatory science. The Forum membership dedicated activities to global health concerns and neglected diseases, such as multidrug-resistant tuberculosis, and undertook efforts to better engage the public in the drug discovery and development enterprise.

For more information, please see https://www.nationalacademies.org/our-work/forum-on-drug-discovery-development-and-translation.

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