Approvals for Rare Diseases Reached Historic High in 2018


A review of novel drug approvals in 2018 found more than half of all novel approvals were for rare diseases and that 2018 also saw more novel drug approvals for rare disease than any other year.


While the rare disease community is aware of the recent efforts from the US Food and Drug Administration(FDA) to create new therapies and devices for the treatment of rare diseases, a new study from the 2019 National Organization for Rare Diseases (NORD) and Orphan Products Breakthrough Summit is demonstrating just how impactful these efforts have been.

Presented by investigators from the FDA, results of the study found changes in policy and a greater emphasis on rare disease has resulted in more than half of novel drug approvals in 2018 were for rare or orphan diseases.

In an effort to highlight the FDA’s Center for Drug Evaluation and Research(CDER) novel drug approvals for therapies to treat rare diseases, investigators conducted a review of approvals from 2018. Results of the review highlighted more than half(34) of the CDER’s novel drug approvals in 2018 were for rare or orphan diseases. Of these 34, 33 were approved under the orphan drug designation.

Investigators pointed out that 34 approvals is the highest number since the passage of the Orphan Drug Act in 1984. Investigators from the FDA also highlighted several orphan drug approvals as part of their presentation. These included migalastat(Galafold) for a rare inherited form of rickets, the first orally-administered drug for Fabry disease, tecovirimat(TPOXX) for treatment of smallpox, and cannabidiol(Epidiolex) for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome.

Additional analyses performed by investigators also revealed the impact different designations from the FDA during the review process can have on potential therapies for rare diseases. Of the 34 novel approvals, 18(53%) had previously received a Fast Track designation.

Breakthrough, Priority Review, and Accelerated Approval designations were also highlighted. The CDER granted Breakthrough status to 13(38%) of the 34 novel drugs approved in 2018 and Accelerated Approval was awarded to 4(12%) novel drugs. Priority Review was the most common for novel drugs to receive prior to approval, with 29(85%) of the 34 drugs approved for rare diseases receiving a Priority Review designation.

In an interview with MD Magazine® at NORD Summit, study investigator Lucas Kempf, MD, associate director of Rare Diseases Program, remarked on the profound impact FDA programs have had on the treatments available for many rare diseases.

“It's fundamentally transformative to a rare disease population when they do first get a drug on the market,” Kempf said.

This study, “2018: Advancing Health Through Innovation FDA and Novel Rare Disease Drug Approvals,” was presented at NORD Summit 2019.

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