FDA stirred up a firestorm recently with its publication of a list of 39 diseases or disease areas … ultimately to be shortened to 20 … to be the focus of public meetings over the next 5 years in the new Patient-Focused Drug Development Initiative.
Patients and patient organizations whose diseases are not on the list protested their exclusion at two public meetings to discuss the initiative in October. And word has it that FDA has been swamped with written responses to its invitation to provide input on this topic.
In FDA’s defense, the agency points out that the diseases on the list are intended to serve as models and that the list was never meant to be all-inconclusive. This is perfectly understandable and would be a reasonable approach in most circumstances.
However, in this case the patient reaction is also understandable because those who have been most vocal in their opposition to the list are from rare disease organizations. These are patient advocates who represent, in many cases, patients with no FDA-approved therapy and who feel that, historically, they have been forgotten by the nation’s medical research and regulatory establishments.
NORD has proposed a solution in written comments submitted to FDA. Our suggestion is to create a list of disease areas that meet FDA’s criteria for the Patient-Focused Initiative that are based on body systems, similar to those employed by the Social Security Administration for disability decisions.
Some of our members and advocacy partners also have submitted written comments suggesting other ways of categorizing the disease areas.
We understand the agency’s position and we agree with FDA that it would be a mistake to assume the list of diseases represents the only opportunity patients will have to provide input for the Patient-Focused Initiative.
However, given the fact that this is one of the first tangible results to come out of this year’s PDUFA reauthorization, and that rare disease patients for so long have felt left out of the process, we understand the response of the patients.
We encourage FDA to find a way to broaden its list of diseases to send a clear message to all that everyone will be included in this Patient-Focused process.
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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