Orphan drug boom comes with a steady stream of outsourcing

17-Feb-2015 - Last updated on 16-Nov-2023 at 10:32 GMT

CROs have a hand in developing an increasing number of orphan drugs

With the FDA approving a record number of orphan drugs in 2014, sponsors weren’t the only ones to reap profits from their development.

CROs (contract research organizations), some of which specifically cater to orphan drug development, saw a rising share of the trials devoted to developing drugs for these rare and orphan indications, according to a new report from ISR Research. The report -- which is based on the interviews with about 100 pharma, biotech and medical device execs -- found that over 60% of respondents outsource at least half of their orphan drug/rare disease clinical studies.

And nearly a quarter of respondents said they outsource between 91% and all of their orphan drug/rare disease trials. In addition, 440 orphan trials are still ongoing, and the vast majority of these trials is either past Phase I or not classified as being within a particular phase.

The growing share of CRO involvement seems to indicate that CROs are becoming more advanced at overcoming what many consider to be difficulties associated with orphan drug development, which includes recruiting difficult-to-find patients and identifying and setting up investigative sites for studies.

Statistical Shakedown

The shakedown in the orphan space is as clear as the latest numbers offered by FDA’s Office of Orphan Products Development, which in 2014, received the highest ever number of orphan drug designation requests, granted the most orphan drug designations, and approved the highest ever number of orphan drugs.

In 2014, according to the FDA Law Blog, “records were shattered…with an astounding 467 designation requests (a nearly 35% increase over 2013), an astonishing 293 orphan drug designations granted (a nearly 13% increase over 2013), and a whopping 49 orphan drug approvals (a 53% increase over 2013).”

What’s more, a new bill introduced in the US House of Representatives would make it even more lucrative for firms to develop orphan drugs. The bill would grant existing drugs -- initially not approved as an orphan drug product -- an additional six months of marketing exclusivity if a company is able to demonstrate the product is able to treat or prevent a rare disease or condition, according to RAPS (Regulatory Affairs Professionals Society).