- Applications from drug companies seeking orphan drug designations have increased sharply over the past several years, straining the ability of the Food and Drug Administration to review them all quickly, wrote Gayatri Rao, director of the Office of Orphan Products development, in a blog post.
- Last year the FDA received 472 orphan drug designation applications, breaking a record set the year before. And this year appears to be on track to set another record, with 30% more requests received so far in 2016 compared to the same time in 2015.
- In response to the higher demand, Rao said the FDA has been forced to shift its internal goal for turnaround time on orphan applications. The regulator now aims to review 75% of designation requests within 120 days of receipt, 30 days longer than the previous standard.
Pharmaceutical companies have been drawn to orphan drug development not only by the statutory incentives under federal law but also by a greater ability to price drugs higher. Orphan diseases rarely have many drugs approved for treatment, meaning a developer of a new drug often has less competition than typically seen in more common therapeutic areas.
And the numbers bear this out. Evaluate Pharma, an analysis firm, projects the global orphan drug market will grow to $178 billion in 2020 sales, nearly double the $97 million in sales hit in 2014. Evaluate's analysis also found the average annual cost of an orphan drug to be $111,820 per year (median cost was $66,057), compared with $23,331 for non-orphan drugs.
The FDA granted 354 of the 472 orphan drug designations requested in 2015, and 21 of the 45 new molecular entities approved last year had received an orphan drug designation. So far this year five of the 14 new drugs have been tagged as orphans.
"The number of requests for orphan drug designation received by FDA’s Office of Orphan Products Development (OOPD) has grown dramatically in recent years and is prompting FDA to adjust its timeframes for reviewing orphan drug designations in order to meet the demand," Rao said in her blog post.
Previously, the FDA has aimed to review 75% of designations in 90 days. Now, however, the FDA pushed back its (internal) deadline to 120 days.
"The sustained increase in designation requests over the last three years, coupled with the increasing number of incentive programs and competing workload priorities, have forced us to reconsider our internal review target," Rao said.
Congress enacted the Orphan Drug Act to spur drug development for rare diseases. Companies that receive an orphan drug designation are rewarded with& several benefits, including increased support from the FDA during the review process, seven years of marketing exclusivity and a tax credit on R&D costs.
The FDA's recently updated PDUFA guidance also increases support for rare disease drug development, including allowing companies to be more flexible when designing clinical trials for orphan drug candidates.
But Rao stressed in her blog post that companies have a part to play too, and should ensure their applications are complete and address all requirements.