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The Orphan Drug Act Celebrates 35 Years of Innovation and Impact on the Rare Disease Community

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By: Erin Reese

Rare and orphan diseases are uncommon, happen infrequently, and affect small numbers of people. Individually, occurrence is relatively uncommon – depending on the disease, affecting perhaps 1 in 20,000 live births. As a whole, the numbers are much more staggering. In the US, upwards of 30 million individuals are living with one of more than 7,000 identified rare and orphan diseases. Of those 7,000+ known diseases, only about 500 currently have some identified form of treatment. While it seems small, those 500 treatments are considered a victory, won with the help of legislation signed into law 35 years ago called the Orphan Drug Act.

Drug discovery and development is a long and expensive process. Due to the time and capital required to discover, develop, and market a drug, pharmaceutical companies had little incentive to focus on treatments for diseases that affected extremely small populations. Individuals with rare and orphan disease had few or no options, answers, or hope for treatments. This changed in 1983, when the Orphan Drug Act was signed into law, becoming a turning point for the rare and orphan disease community.

According to data from the Rare Disease Report, prior to the Orphan Drug Act, there were only 34 approved orphan drugs. Following approval, more than 400 additional treatments have been approved.

One disease population that greatly benefited from the incentives developed by the Orphan Drug Act are patients with cystic fibrosis (CF). Prior to the passage of the orphan drug act, there were no therapies approved to treat CF and getting companies on the phone to even discuss the possibility of working on CF drugs was unthinkable. While the Cystic Fibrosis Foundation’s venture philanthropy model and the discovery of the CFTR gene in 1989 were critical in changing this landscape, passage of the Orphan Drug Act was pivotal, as it provided incentives for companies to invest in therapies for CF and other rare diseases. As a result, by 2004, there were at least 24 drugs in development for the treatment of CF. In 2012, Kalydeco, a new drug that that treats the underlying cause of disease for some CF patients, was approved by the FDA.

“In many ways, the rare disease landscape has changed dramatically over the last 30 years because of the Orphan Drug Act,” said Ted Torphy, PhD, Chief Scientific Officer at BioMotiv and Board Chairman, Cystic Fibrosis Therapeutics, Inc. “Hundreds of groundbreaking therapies are now available to treat serious illnesses, like CF, that may otherwise have been ignored. The Orphan Drug Act remains a powerful and important tool to fuel continued scientific innovation for the patients who need it most.”  

While the Orphan Drug Act has certainly had a measurable impact, the valley of death continues to challenge drug development for rare diseases as well as diseases for larger patient populations.  There is a need for additional, varied structures and approaches, including accelerators and impact investors, to ensure continued advancement of potential treatments and cures for orphan diseases. In the coming months, we will continue to delve into novel solutions, including the Orphan Disease Megafund concept proposed by Andrew Lo, Roger Stein, and colleagues.

The Orphan Drug Act (ODA)

The ODA provided drug manufacturers with three primary incentives to develop treatments for patients with rare and orphan disease:

·        federal funding of grants and contracts to perform clinical trials of orphan products;

·        a tax credit of 50 percent of clinical testing costs; and

·        an exclusive right to market the orphan drug for seven years from the date of marketing approval.

Additional benefits available to sponsors of orphan-designated products include close coordination with the Food and Drug Administration (FDA) throughout the drug's development, priority FDA review, and a waiver of drug application fees.

Erin Reese