Congratulations to Sharon Terry and All the Rare Disease Heroes!

On January 7, 2013 Sharon Terry was recognized by FDA as one of 30 rare disease heroes. Sharon has been a leader in the rare disease advocacy community and beyond since her children were diagnosed with pseudoxanthoma elasticum (PXE) in 1994. She led the way in novel and transformational activities as the first lay person to: establish a registry and biobank, discover and patent the gene associated with the disease (ABCC6), and develop and commercialize a diagnostic test. In addition, she created and deployed a natural history study and advanced clinical trials. Not satistifed that this should be done for one disease, and realize that is a waste of resources, she generalized each of her solutions to extend to all diseases and established the Genetic Alliance Registry and BioBank and Registries for ALL. Her work continues today throughout the 10,000 organizations in the Genetic Alliance network.

FDA also commemorated more than 30 years of dedicated service by all stakeholders in 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.

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. 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.