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

Advocating for Patients with Rare Diseases

By: Debra Y. Lewis, OD MBA

This week we commemorate the fifth annual Rare Disease Day, a global campaign to raise awareness of the millions of people worldwide who suffer from rare diseases. In the United States, about 30 million Americans have rare diseases, so in reality, rare diseases are not so rare when viewed together as a group.  Because of the recognition of the need for therapies for rare diseases, today FDA held our first-ever Rare Disease Patient Advocacy Day.  

Rare Disease Patient Advocacy Day at FDA March 1, 2012 Stick figures of familes with the in front of the earthFDA designed Patient Advocacy Day to help patients and caregivers engage with us on issues related to drug and medical device development for rare diseases and conditions. Today gives rare disease patient advocates the opportunity to meet with FDA staff and learn more about how FDA works. And, as we come together with colleagues, families, patients and advocacy groups, it gives FDA a moment to reflect on recent news in helping people with rare diseases. 

We often talk about how important the Orphan Drug Act has been in bringing treatments to people with rare diseases – notably children with rare diseases. And we can see its impact. A new study published this week in Pediatrics highlights the progress we’ve made over the past 10 years in bringing treatments to children with rare diseases. The study reports that from 2000 to 2009, 1138 “orphan” drugs were designated and 148 received FDA approval, of which 38 were for pediatric diseases. The proportion of approvals for pediatric products increased from 17.5% in the first half of the decade, to 30.8% in the second. Orphan Drug Act incentives have led to increased product availability for rare diseases overall, with an increasing number of marketing approvals for children in the past decade.

And in 2011, FDA approved 26 drugs and biological products for rare diseases.  About one-third of all new molecular entities approved by the agency were for rare diseases. FDA also approved six medical devices for rare conditions under the Humanitarian Device Exemption program in 2011. We are very proud of the collaborations between industry, patient advocates and FDA that have made this a reality.  

There are over 400 drugs and devices approved for rare diseases and conditions, and millions of lives have been saved or improved with these new products. However, we still have much to do to meet the rare diseases challenge – there are 7000 rare diseases and most are without a current treatment available. Meeting this challenge is the focus of FDA’s Patient Advocacy Day today and will continue to be in the coming years as we strive to improve the lives of all Americans impacted by a rare disease.

Debra Y. Lewis, OD MBA, is Deputy Director of FDA’s Office of Orphan Products Development