3 charts that show why 2014 was a record year for US orphan drugs
According to a new year-by-year analysis of orphan drug designation requests and approvals since 1983 by food and drug law firm Hyman, Phelps & McNamara (h/t to the WSJ's Ed Silverman for flagging the study), 2014 was a groundbreaking year for orphan drugs the U.S. in several key metrics.
Kurt Karst of Hyman, Phelps & McNamara notes in a post at FDA Law Blog that 2014 saw the highest number of orphan drug designation requests, the highest number of orphan drug designations, and the highest number of orphan product approvals ever. The firm combed through data from the FDA’s Office of Orphan Products Development (OOPD) database and found, respectively, 35%, 13%, and 53% increases in orphan drug designation requests, designation approvals, and products approvals compared to 2013.
The increase in designation requests is particularly interesting, suggesting that more pharma companies are getting interested in pursuing novel and rare disease markets (you can click on the charts to expand the images):
The orphan drug market is tricky. First, conducting clinical trials can be difficult considering that the available patient pools are so small, and R&D expenditures tend to be huge. But in many cases, so are margins—and as the FDA and Congress show increased interest in easing the regulatory pathway through initiatives like 21st Century Cures, which would extend rare disease drugs' exclusivity, pharma companies might be taking notice.
As this next chart shows, the number of granted designations had a relatively modest increase over 2013. But that's only because 2013 itself was a blockbuster year for orphan drug designations:
Finally, orphan product approvals were up a staggering 53%, falling just shy of 50 orphan approvals in 2014:
As BioPharma Dive has previously reported, 2014 was an impressive year for FDA drug approvals all around, with every single New Molecular Entity (NME) and Biologics License Application (BLA) NDA being accepted by the agency.
For all of the challenges that orphan drug development poses, it appears that many industry players are looking towards the rare disease space as an area ripe for innovation. And companies will be sure to keep a close eye on 21st Century Cures' progress—especially at the fate of its provisions on licensing exclusivity, which have already drawn criticism from organizations like the Generic Pharmaceutical Association (GPhA).