VERY IMPORTANT (FDA INITIATIVE)By
Below is information about an FDA initiative. Thirty-nine diseases are in consideration for priority and 20 will be selected. This has a Nov. 1 deadline!
As part of its commitment under the recently approved Prescription Drug User Fee Act (PDUFA V), the FDA will be conducting an initiative, called the patient-focused drug development initiative, to provide for a more systematic approach to obtain the patient’s perspective on the disease severity and the currently available treatments. The intent is to ensure a thorough understanding of the severity of the treated condition and the adequacy of the existing treatment options.
Dercum’s disease is NOT on this list!!
The FDA is also interested in public comment on disease areas that are not represented on this preliminary list. The Agency used several criteria to develop the preliminary list of potential disease areas. FDA requests that when proposing additional disease areas for consideration, please describe how you applied the identified criteria in making recommendations for additional disease areas to consider.
These criteria include the following:
Disease areas that are chronic, symptomatic, or affect functioning and activities of daily living;
Disease areas that reflect a range of severity;
Disease areas for which aspects of the disease are not formally captured in clinical trials;
Disease areas that have a severe impact on identifiable subpopulations (such as children or the elderly);
Disease areas that represent a broad range in terms of size of the affected population; or
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, functions, or survives.
FDA will consider the public comments received at the public meeting and through the docket and post the set of disease areas for FY 2013-2015 on the FDA Web site. By the end of FY 2015, FDA will initiate a public process for determining the list of disease areas for FY 2016-2017.
This is an excellent opportunity to help the FDA better understand how fat disorders affect patients. Your support is essential to ensure that your fat disorder is considered in the future as a selected disease.
Please send your comments in by November 1 to ensure that the FDA understands why your fat disorder should be selected. As you write your own letter, make points that reflect how your fat disorder meets the criteria above.
Comments should be submitted electronically at:
Select ‘Individual Consumer’ for the ‘Category’ and ‘None’ for ‘Organization’ if no other choice is appropria te . Written comments can be submitted to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
Further information on the patient focused drug development initiative and the list of 39 diseases initially nominated can be found here: https://www.federalregister.gov/articles/2012/09/24/2012-23454/prescription-drug-user-fee-act-patient-focused-drug-development-public-meeting-and-request-for
Sample Letter – limited to 2000 characters
To: FDA Patient Focused Drug Development Initiative
From: <include your name here>
I am writing to request that Dercum’s disease (also called adiposis dolorosa) be included as one of the diseases in the patient focused drug development initiative.
Dercum’s disease is a complex, chronic pain disorder that was initially identified in 1888 as a lipomatous disorder of unknown etiology. Dercum’s disease can affect people of all ages with an average age of onset of 35 years. People have similar signs and symptoms of fibromyalgia but have exquisitely tender fat nodules that cause them to take pain medications including opiates and intravenous lidocaine. It affects over 300,000 children, men and women in the US of all races and income levels. Over 60% of people with Dercum’s disease become disabled losing their jobs, spouses and friends. Dercum’s disease is inherited in an autosomal dominant disorder according to a paper in the New England Journal of Medicine.
What is tragic is that over 100 years, there have been no clinical trials for drugs to treat Dercum’s disease and there are still NO approved treatments and NO biomarkers or outcome measures and NO identified gene.
<Include personal story here>
By ensuring a thorough understanding of the severity of Dercum’s disease from a patient perspective, especially given the lack of any viable treatments to change the course of the disease, the patient focused drug development initiative could make a tremendous difference in the lives of a huge number of people.
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