FDA Commemorates 30th Anniversary of the Orphan Drug Act

By: Gayatri R. Rao, M.D., J.D.

Gayatri R. Rao, M.D., J.D., is Director of FDA's Office of Orphan Products Development

When President Reagan signed the Orphan Drug Act 30 years ago, he enacted a critically important piece of health care legislation. The passage of this Act on January 4, 1983, was monumental because it created—for the first time—incentives to develop desperately needed medical products for Americans suffering with rare diseases. Until that point, development of such products was very limited. For instance, in the decade leading up to the passage of the Orphan Drug Act, only 10 industry-supported products for rare diseases were brought to market.

The Office of Orphan Products Development (OOPD) was formed at FDA more than 30 years ago, prior to the passage of the Orphan Drug Act, because FDA recognized that rare diseases, when taken together, posed a significant national public health issue. Once the Orphan Drug Act was passed, OOPD became responsible for administering the incentive programs created to spur the development of medical products for rare diseases, namely the Orphan Drug Designation Program and the Orphan Products Grants Program. These products include drugs, biologics, medical devices, and medical foods for the treatment of rare diseases.

As FDA commemorates the passage of this important legislation, we look back over the last 30 years with pride. Since its passage, over 2700 products in development have been designated as orphan drugs through the Orphan Drug Designation Program and over $290 million has been awarded to clinical studies through the Orphan Products Grants Program.  These programs, along with the critical, collective efforts of the Center for Drug Evaluation and Research’s (CDER) Rare Diseases Program, and those of many individuals across FDA, have helped to bring over 400 orphan products for rare diseases to the market.

We also commemorate the more than 30 years of dedicated service from every member of the rare disease community:

  • the patient advocates, who spurred national awareness about the challenges that people with rare diseases face and who continue to support families, educate the community, and drive research into their diseases;
  • the legislators who heard the voices of rare disease advocates and worked to champion the passage of the Orphan Drug Act;
  • the research community, which continues to leverage resources and foster collaborations among academia and industry stakeholders;
  • the clinicians, who support the medical needs of families with rare diseases and work to advocate on behalf of the community;
  • and industry, including pharmaceutical and biotech companies, angel investors, and venture capitalists who, in the spirit of the Orphan Drug Act, have come together to develop products for rare diseases.

Our many successes give us a reason to celebrate 30 years of hard work to provide diagnostic or treatment options to those with rare diseases. But we are keenly aware that there is still a challenging road ahead. We at FDA remain firmly committed to working with the rare disease community to tackle those challenges and to find new diagnostic tools and treatments for the millions of patients with rare diseases.

Gayatri R. Rao, M.D., J.D., is Director for The Office of Orphan Products Development

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