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 7 Reports
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Addressing the Threat of Drug-Resistant Tuberculosis: A Realistic Assessment of the Challenge. Workshop Summary
Tuberculosis is one of the leading causes of death in the world today, with 4,500 people dying from the disease every day. Many cases of TB can be cured by available antibiotics, but some TB is resistant to multiple drugs--a major and growing threat worldwide. The Institute of Medicine’s Forum on Drug Discovery, Development, and Translation hosted a workshop on November 5, 2008, to address the mounting concern of drug-resistant TB. The session brought together a wide range of international experts to discuss what is known and not known about this growing threat, and to explore possible solutions.
Released: August 26, 2009
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Accelerating the Development of Biomarkers for Drug Safety. Workshop Summary
Biomarkers can be defined as indicators of any biologic state, and they are central to the future of medicine. As the cost of developing drugs has risen in recent years, reducing the number of new drugs approved for use, biomarker development may be a way to cut costs, enhance safety, and provide a more focused and rational pathway to drug development. On October 24, 2008, the IOM’s Forum on Drug Discovery, Development, and Translation held “Assessing and Accelerating Development of Biomarkers for Drug Safety,” a one-day workshop on the value of biomarkers in helping to determine drug safety during development.
Released: June 19, 2009
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Addressing the Barriers to Pediatric Drug Development. Workshop Summary
The majority of drugs prescribed for children—50 to 75 percent—have not been tested in pediatric populations. Addressing the Barriers to Pediatric Drug Development is the summary of a workshop that was organized to identify barriers to the development and testing of drugs for pediatric populations, as well as ways in which the system can be improved to facilitate better treatments for children.
Released: August 22, 2008
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