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Study highlights impact of current legislation on orphan products to treat rare diseases

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Between 1983 and July 2020, the U.S. Food and Drug Administration approved 599 orphan products, of which 552 are currently on the market, according to a new study by the National Organization for Rare Disorders (NORD) to determine if laws and regulations regarding orphan products, generics, and biosimilars have helped bring new products to market.

Prior to 1983 when the Orphan Drug Act became law, there were only 38 orphan products.

Of the 552 drugs or biologics available with an orphan designation, 394 had a form of patent or orphan exclusivity protection. Of the 158 products that had no protection, generics were available for 51%. The patent life exceeds the term of orphan drug exclusivity for 125 orphan products.

Overall, 37% of orphan products were originally approved for a common condition and earned an orphan indication later, while 154 were first FDA-approved for a rare disease and eventually earned ≥1 orphan indication. Only 64 have ≥3 orphan indications.

The majority (75%) of orphan products have only 1 orphan indication.

“More than 9 out of 10 orphan products on the market today would never have been developed without the Orphan Drug Act,” said Peter L. Saltonstall, President and CEO of NORD, in a press release. “The vast majority of people with rare diseases still have no treatment, and we need government to provide a framework that helps patients by encouraging the development of innovative therapies, and spurs competition after a reasonable amount of time.”

National Organization for Rare Disorders. Orphan Drugs in the United States: An examination of patents and orphan drug exclusivity. 2021. Available at: rarediseases.org/rareinsights.