Report Finds Most Rare Disease Drugs Protected by Patents Rather than Exclusivity

Rare Daily Staff

The Orphan Drug Act is widely recognized for driving the development of therapies to treat rare conditions, but critics have complained of leveraging the act to reap rewards from it in ways not envisioned by its creators.

That’s because a number of drugs with orphan designations have won approval for broader indications, giving the drug company access to sizable markets while at the same time conferring incentives such as tax breaks, extended periods of exclusivity to guard against competition, and fee waivers.

Now, a new report the National Organization for Rare Disorders commissioned from Avalere finds drug patents and not extended exclusivity has much more to do with a lack of generics and biosimilars to treat rare diseases.

According to the report, the U.S. Food and Drug Administration (FDA) approved 599 orphan products to treat rare diseases between 1983 and July 2020, 552 of which were on the market at the time of the study. Before the Orphan Drug Act became law in 1983, only 38 orphan products existed.

The majority (75 percent) of FDA-approved orphan products treat one rare disease and have no other use.

While more than 90 percent of rare diseases have no FDA-approved treatment, some have multiple treatments available due to the presence of generics and biosimilars. 

At the time of the study, 158 orphan products were eligible for generic/biosimilar competition due to expired patents and orphan drug exclusivity; generics/biosimilars were available for 81 (51 percent) of these products. 

The researchers examined why the majority (394) of orphan products were not eligible for generic or biosimilar competition and found that 80 percent of orphan products were protected from competition due to the patent life of the product, and 20 percent were protected because of orphan drug exclusivity. Notably, 22 percent of all orphan products had patent protections lasting more than 20 years. 

“More than 9 out of 10 orphan products on the market today would never have been developed without the Orphan Drug Act,” said Peter Saltonstall, president and CEO of NORD. “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.” 

Photo: Peter Saltonstall, president and CEO of NORD