The FDA recently released the Orphan Drug Modernization Plan designed to shorten the time required to secure an orphan disease drug designation. This is part of its continued effort to expedite the process to bring products to market. The orphan designation approval process is overloaded – the FDA currently has a backlog of 200 requests, which the agency aims to clear by mid-September.   Its plan is simple – form a team of reviewers experienced in reviewing orphan products to focus on clearing the backlog. 

Orphan diseases have become more of a focus for pharma companies.  They can command significant prices for these products, driving strong revenue potential even with small patient populations.  This had previously been something to dissuade development in this area, but the high prices make it too attractive to the manufacturers. 

Requests for the designation are growing – doubling since 2012, to a high of 568 requests in 2016.  The FDA has a plan to address these concerns by establishing a new Orphan Products Council and reorganizing other staff to handle these requests with greater efficiency.  It has also committed to ensuring all reviews within 90 days.  There’s no real downside. Manufacturers receive an additional incentive to pursue orphan drug development knowing they have a higher likelihood to receive the accelerated approval cycle and additional exclusivity that comes with the designation.  Patients also benefit. There will be more drugs to treat diseases, and life-saving therapies will make it to market where they might not have otherwise.

This is one area the FDA is saying the right things.  "Congress gave us tools to incentivize the development of novel therapies for rare diseases and we intend to use these resources to their fullest extent,” Commissioner Scott Gottlieb said. 

However, the key here is in execution.  Not only must the FDA clear the backlog, it needs to do it in a way that provides the designation to the right products.  Should it approach the 200 pending applications with a higher than usual rate of approvals simply to clear the backlog by September, there may be implications for the future unseen today. 

Manufacturers, seeing an easier path to approval, might pursue development or even just an orphan designation where they might have not otherwise. At the same time, the exclusivity provided by the designation might otherwise deter development of promising therapies, especially from smaller organizations, unwilling or unable to take on the risk.  This has a low likelihood but something the FDA panel should consider nonetheless.

Of all the efforts put forth by the FDA this one seems the most promising.  Orphan diseases face challenges from being what they are – small patient populations with hard-to-treat disease.  The corresponding therapies are highly valuable to a manufacturer – these therapies often require extensive long-term treatment. With the high prices they command the chance to get to market that much quicker, manufacturers now have the right set of incentives to pursue development and commercialization. 

Should the FDA get this right--first clearing the backlog and then maintaining the approval process in such a way that maintains the right drugs receive the approvals--patients and manufacturers will benefit from being able to rapidly bring potentially life-saving therapies to market.