ADVOCACY UPDATE! FDA Announces Disease Areas It Will Be Focusing On During The First Three Years of Patient-Focused Drug Development
By Natacha T. Pires, MBBS, Director, Medical and Public Affairs

April 15th, 2013

On April 11th, the Food and Drug Administration (FDA) announced via a Federal Register notice a list of 16 diseases areas--several with clear links to neuropathy--it would focus on for the first three years of its five-year Patient-Focused Drug Development Program.

This 5-year initiative is being conducted to fulfill FDA's performance commitments made as part of the fifth authorization of the Prescription Drug User Fee Act (or PDUFA V). It provides a more systematic approach for the FDA to obtain patients' input on specific disease areas, including their perspectives on their condition, its impact on daily life, and available therapies. The FDA selected these disease areas based on a set of selection criteria, the perspectives of the reviewing divisions at FDA, and the public input received on a preliminary set of disease areas published in the Federal Register on September 24, 2012.

FDA's Decision Criteria

The FDA indicated that the final list of diseases selected were based on the following criteria: 

- Disease areas that are chronic, symptomatic, or affect functioning and activities of daily living;

- Disease areas for which aspects of the disease are not formally captured in clinical trials; and

- Disease areas for which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels or functions.

Almost 4,500 comments addressing over 90 disease areas–more than half concerned lung cancer, narcolepsy, and interstitial lung disease (pulmonary fibrosis was not on the FDA's preliminary list of 39 diseases)--were submitted by patients, patient advocates and advocacy groups, caregivers, healthcare providers, professional societies, scientific and academic experts, pharmaceutical companies, and others. The majority of comments were submitted by individual patients. 

Neuropathy Was On The FDA’s Preliminary List of 39 Diseases Being Considered

Neuropathy was included in the FDA's September 2012 Federal Register preliminary list of 39 diseases areas being considered. This--in itself--was very encouraging for our neuropathy community: it validated the FDA’s awareness of the unmet needs of over 20,000,000 people living with neuropathy, 6 million of whom also battle chronic neuropathic pain.

With the FDA requesting public comment on potential disease areas to be addressed throughout PDUFA V, The Neuropathy Association immediately informed its members—patients, family members, and health care professionals—about this time-sensitive opportunity to submit public comments and encouraged members to write in. The Association also submitted a formal letter—on behalf of the entire neuropathy community—to the FDA applauding the effort, reminding them that the few therapies we currently have in our neuropathy arsenal only reached our community within the past eight years…and that these therapies marginally address only a third of our patients’ needs.

The Neuropathy Community’s Next Step

While neuropathy did not make the list of disease areas being considered for the first three years of the FDA’s five-year Patient-Focused Drug Development Program, the Association is optimistic neuropathy will make the list during the next round. The FDA will be initiating a second public process to determine the list of disease areas for FY 2016-2017. And, as a community, every single one of us must commit to submitting public comments to ensure neuropathy has a better chance of making the list in 2016-2017.

In the interim, the Association is proactively seeking other opportunities to voice and address the unmet needs of the over 20,000,000 living with neuropathy.


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