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.
This initiative will be conducted for each of 20 different disease areas over a period of 5 years. The FDA has nominated an initial list of 39 diseases, including ME/CFS, using the following criteria:
- 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
- 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.
The next step in the process is for the FDA to gather public input between now and November 1st on which of disease areas should be selected for inclusion in this initiative. Additional disease areas may also be nominated during this time.
Note that initiative is in addition to the ME/CFS FDA stakeholder meeting that Dr. Woodcock, Director of the Center for Drug Evaluation and Research at the FDA, has already committed to.
How can you help: For ME/CFS, this is an excellent opportunity to help the FDA better understand how ME/CFS affects the patients. Your support is essential to ensure that ME/CFS is one of the 20 selected diseases.
Please send your comments in by November 1 to ensure that the FDA understands why ME/CFS should be selected as one of the 20 diseases.
Comments can be submitted electronically, or by regular mail to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. Make sure you include the docket number (FDA-2012-N-0967).
Further information on the patient focused drug development initiative and the list of 39 diseases initially nominated can be found here.