The Firestorm Over FDA’s List of Diseases

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Nov. 05, 2012

TOPIC: Advocacy, Featured News, Industry, Patients & Members

The Firestorm Over FDA’s List of Diseases

Posted at November 5, 2012 2:58 pm by Mary Dunkle

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.

TAGS: FDA, Mary Dunkle, Patient-Focused Drug Development

2 Responses to The Firestorm Over FDA’s List of Diseases

Dean Suhr says:

November 6, 2012 at 2:43 pm

Greetings Mary. Thanks for keeping the community updated on this very important topic. I attended the meeting at the FDA, testified orally, and submitted written comments as well. I also called for some careful thought as to how the 20 diseases are selected.

But I respectfully disagree about the “firestorm” label. The oral comments were very reasonable with some specific disease lobbying, but I felt the overall attitude was quite respectful. There was no bickering or hostility – just a good group of advocates sharing perspective. We all know that every one of the 7,000+ rare disease cannot fit into a 20 meeting schedule and were trying to make sure that above all, rare disease was represented to address the unique issues of research with very small disease knowledge and small patient communities.

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Mary Dunkle says:

November 7, 2012 at 3:18 pm

Thanks for your thoughts, Dean. Our concern was that patients were not given a chance to provide input into the creation of the original list of diseases. The conversation at the Oct. 25 public meeting was reasonable, which is important and good. But several patient representatives have expressed concern about the list and the fact that it focuses on some specific diseases along with other much more general disease areas.

We understand that FDA has been given a huge challenge in trying to assure that all voices are heard, and that there will be many more opportunities for patient input through the Rare Disease Initiative. We pledge our support to the agency in this process. But we also feel it’s important to send a strong message of inclusion early in this process, and we hope FDA will consider alternatives to the current list such as those suggested by NORD and other patient organizations in their written comments.

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