Arithmetic is being able to count up to twenty without taking off your shoes.
-- Mickey Mouse
To help reviewers better understand patient perspectives, FDA will conduct four public meetings per year on different disease areas during the five-year PDUFA cycle. That equals 20 meetings on 20 different disease areas. The information gleaned from these sessions is expected to help reviewers better understand patient needs and risk tolerance and to help spur further research.
(FDA agreed as part of the reauthorization to better incorporate patient opinions about risk tolerance and other drug development issues into its approval decisions.)
But, under the headline of “no good deed goes unpunished,” FDA is working to allay advocates’ fears that the 20 disease areas selected for public meetings will be the agency’s sole focus over the next five years.
FDA has developed a list of 39 candidate topics for the meetings, about one-third of them rare diseases – but, according reportage in the Pink Sheet, “it drew the ire of some patient groups.” Diane Dorman, NORD’s vice president of public policy said that the agency’s list has pitted disease organizations against each other.
But according to the always thoughtful Theresa Mullin (CDER’s Director of Planning and Informatics), the disease manifest “is not is any sort of priority list for FDA. According to Mullin,the proposed diseases were chosen in part because existing measures are inadequate.
“This is not to try to address all the important diseases, just ones where, in fact, we don’t have very good clinical measures, we don’t have good objective measures now, and we need to develop the best measures we can to encourage drug development,” she said.
Mullin mentioned chronic fatigue syndrome as an example because it does not have a definitive set of symptoms and obtaining more patient information would help develop more drugs for the treatment armamentarium.
The FDA is also convening several “consultation meetings” with patient stakeholders to talk about drug development issues and help formulate the direction of the disease-specific public meetings. Topics will include: resolving conflicting opinions among stakeholders, balancing the priorities of different disease areas within FDA’s limited resources, and ensuring groups outside the Washington D.C. area gain equal access to the talks.
Talk about 20/20 vision!
