Report
Note: Workshop Summaries contain the opinion of the presenters, but do NOT reflect the conclusions of the IOM. Learn more about the differences between Workshop Summaries and Consensus Reports.
The process for developing new drug and biologic products is extraordinarily expensive and time-consuming - many consider the traditional model to be unsustainable. Although large pharmaceutical companies may be able to invest in the development of blockbuster drugs because they can expect a large return on their investment, these same organizations, when developing drugs to treat rare and neglected diseases, are unable to rely on such returns.
Recognizing the lack of available treatments for rare and neglected diseases and the difficulty of finding companies to develop them, Congress passed the Orphan Drug Act in 1983 to provide financial incentives to companies to develop such drugs. Despite passage of this bill, many rare and neglected diseases still do not have effective treatment options.
On June 23, 2008, the Institute of Medicine's Forum on Drug Discovery, Development, and Translation held a public workshop, "Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies", which sought to explore new and innovative strategies for developing drugs for rare and neglected diseases. Presentations and discussions considered recent progress and continuing obstacles in the development pipelines for these drugs, and considered a range of innovative strategies that are currently being pursued, including financial mechanisms for de-risking the drug development process, encouraging the sharing of data, and engaging patients in the clinical trial process to enhance participation.
Other Reports by this Activity
Displaying: 3 of 14 Reports
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Strengthening a Workforce for Innovative Regulatory Science in Therapeutics Development - Workshop Summary
The development and application of regulatory science – which FDA has defined as the science of developing new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of FDA-regulated products – calls for a well-trained, scientifically engaged, and motivated workforce. FDA faces challenges in retaining regulatory scientists and providing them with opportunities for professional development. In the private sector, advancement of innovative regulatory science in drug development has not always been clearly defined, well coordinated, or connected to the needs of the agency. As a follow-up to a 2010 workshop, the IOM held a workshop on September 20-21, 2011, to provide a format for establishing a specific agenda to implement the vision and principles relating to a regulatory science workforce and disciplinary infrastructure as discussed in the 2010 workshop.
Released: December 21, 2011
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Public Engagement and Clinical Trials: New Models and Disruptive Technologies - Workshop Summary
Clinical trials provide essential information needed to turn basic medical research findings into patient treatments. New treatments must be studied in large numbers of humans to find out whether they are effective and to assess any harm that may arise from treatment. There is growing recognition among many stakeholders that the U.S. clinical trials enterprise is unable to keep pace with the national demand for research results. The IOM, along with the Mount Sinai School of Medicine, held a workshop June 27-28, 2011, to engage stakeholders and experts in a discussion about possible solutions to improve public engagement in clinical trials.
Released: October 14, 2011
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Advancing Regulatory Science for Medical Countermeasure Development - Workshop Summary
Whether or not the United States has safe and effective medical countermeasures—such as vaccines, drugs, and diagnostic tools—available for use during a disaster can mean the difference between life and death for many Americans. The Food and Drug Administration (FDA) and the scientific community at large could benefit from improved scientific tools and analytic techniques to undertake the complex scientific evaluation and decision making needed to make essential medical countermeasures available. At the request of FDA, the IOM held a workshop to examine methods to improve the development, evaluation, approval, and regulation of medical countermeasures.
Released: June 15, 2011
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