On this page you can find important Policy Reports from the nation’s leading thought leaders in the Rare Disease Community.


New Thinking, New Competencies, New Results Driving New Paradigms in Cancer Research
December 2010

Executive Summary
A recent town hall meeting offered an opportunity to explore how government, nonprofit organizations, and academic institutions can define new models of working with the private sector to enhance drug development efforts and bring safer, more effective drugs to the market more efficiently. While the challenge to innovative drug development can be great, our investments in biomedical research are providing promising opportunities to capitalize on emerging science. The following recommendations are based on a series of expert panel discussions, and can ensure that the promise of scientific research translates into reality, benefiting the health of the nation.



IOM: Rare Diseases and Orphan Products: Accelerating Research and Development 2010

by the Institute of Medicine, Oct 2010

Rare diseases collectively affect millions of Americans of all ages. They often are serious and life-altering; many are life-threatening or fatal. But because each rare disease affects a relatively small population, it can be challenging to develop drugs and medical devices to prevent, diagnose, and treat these conditions. We still lack even a basic understanding of the cause or underlying molecular mechanisms of many rare diseases. To help in accelerating rare diseases research and product development, the National Institutes of Health (NIH), with support from the Food and Drug Administration (FDA), asked the IOM to examine the opportunities for and obstacles in developing drugs and medical devices for treating rare diseases.

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FDA Science and Mission at Risk

Report of the Food & Drug Administration’s Subcommittee on Science and Technology

PREPARED FOR FDA Science Board, November 2007   
FDA Mission Statement
“The FDA is responsible for protecting the public health by assuring the safety, efficacy, and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation. The FDA is also responsible for advancing the public health by helping to speed innovations that make medicines and foods more effective, safer, and more affordable; and helping the public get the accurate, science-based information they need to use medicines and foods to improve their health.”


Department of Health and Human Services, OFFICE OF INSPECTOR GENERAL  MAY 2001

PURPOSE:  The purpose of this inspection was to assess the implementation of the Orphan Drug Act of 1983 and its impact on industry and patients.

Congress passed the Orphan Drug Act of 1983 to stimulate the development of drugs for rare diseases. A rare disease is defined as a disease that affects fewer than 200,000 people in the United States. Prior to passage of this historic legislation, private industry had little incentive to invest money in the development of treatments for small patient populations, because the drugs were expected to be unprofitable. The law provides 7-year marketing exclusivity to sponsors of approved orphan products, a tax credit of 50 percent of the cost of conducting human clinical testing, and research grants for clinical testing of new therapies to treat orphan diseases. Exclusive marketing rights limit competition by preventing other companies from marketing the same version of the drug, unless they can prove clinical superiority.

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Orphan Drug Act: Background and Proposed Legislation in the 107th Congress

Congressional Research Service (CRS) Report for Congress, July 2001

The Orphan Drug Act (P.L. 97-414) was signed into law on January 4, 1983. The Act provides incentives for pharmaceutical manufacturers to develop drugs, biotechnology products, and medical devices for the treatment of rare diseases and conditions. These products are commonly referred to as orphan products. Incentives for orphan product development include marketing exclusivity for orphan drug sponsors, tax incentives, and research grants. Since the Act was passed in 1983, the Food and Drug Administration (FDA) has approved 183 new orphan products. Critics of the Act argue that, because the Act relies on market-oriented strategies to promote orphan drug development, overpricing of drugs can limit patient access to orphan drug treatment, especially among those who lack health insurance. Others argue that the Act has been very successful in finding new treatments for rare diseases and conditions, and that any changes to the incentives provided in the law would suppress research and development.  Legislation has been introduced in the 107th Congress to modify marketing exclusivity provisions, and to accelerate and expand tax benefits for orphan drug manufacturers.


Report of the National Commission on Orphan Diseases, 1989

By the US Department of Health and Human Services, February 1989

Read the report at:  http://rarediseases.info.nih.gov/files/Report_of_the_National_Commission_on_Orphan_Diseases_February_1989.pdf