The WSJ Health blog reports on a new paper that advocates that the FDA should offer specific guidelines to help companies seeking to develop drugs for rare diseases to better utilize the accelerated approval apparatus.
The paper explains that the FDA has established mechanisms -- in the wake of HIV/AIDs advocacy in the 1990's - to approve drugs more rapidly by using surrogate endpoints. In return, companies need to keep testing and reporting data to see if the benefits of the drug hold up in the treatment of patients. The agency says it is crucial to establish a surrogate endpoint is a valid indicator of a drug's efficacy. The Catch-22 though is that it is hard for companies to know what data the FDA will require to establish the surrogate endpoints so that they are "reasonably likely" to predict good patient outcomes.
The paper's authors plan to hold a workshop aimed at getting the FDA to write down guidelines to meet the "reasonably likely" standard. They would also like the FDA to consider whether the criteria used should be different for at least some rare diseases. They believe several drugs that have languished in development for years could benefit rare disease patients now if the accelerated pathway were open to them.
Several large pharma organizations, in addition to certain strong niche players, have indicated a desire to work more in the rare and orphan drug arenas. I would expect that they would be very interested in pathways that could accelerate their progress to approval.
Posted by Bruce Lehr July 6th 2011.