FDA Opens Dialogue

FDA-teleconference-300x278Ten representatives of the Food and Drug Administration (FDA) participated in a teleconference with ME/CFS stakeholders to provide a brief update on the regulatory agency’s role and activities to facilitate drug approvals for ME and CFS. The 90-minute call was announced last month and registration was open to 50 pre-registrants. The stated purpose of the call was to “discuss issues of mutual interest and concern including the lack of approved treatment options available for ME/CFS and how treatment development might be facilitated.”

Sandra Kweder, M.D., deputy director of FDA’s Office of New Drugs, led the call for the agency. She provided a brief overview of the agency’s role in the drug approval process and addressed several issues of particular concern and importance to ME/CFS stakeholders. The majority of the time was spent hearing from and responding to questions and concerns raised by members of the public, most of whom were individuals with ME and/or CFS (by their own designation) who had been ill for 10, 20, 25 years or more. The Solve ME/CFS Initiative’s CEO, Kim McCleary, and scientific director, Dr. Suzanne Vernon, participated in the call.

These announcements were among the statements provided in the overview and in response to participants’ questions:

  • The FDA considers ME/CFS to be a “serious and life-threatening” condition, making applications for marketing approvals to treat ME/CFS eligible for expedited review and other incentives.
  • FDA uses the term ME/CFS to indicate inclusiveness of the conditions defined by ME and CFS criteria. As stated in background information for the call, “Drug development focuses on quantitative measures of benefit (e.g., symptom improvement), not on the name of the disease. Therefore, for the purpose of drug development, the reference of ME/CFS does not mean that FDA views the two diseases or syndromes as the same.”
  • There are presently 8 open investigational new drug (IND) applications for indications for ME and/or CFS. Most are preliminary, for small numbers of individuals and the products being tested are mostly nutrutional supplements.
  • FDA will provide an overview of the drug approval process at the next meeting of the federal CFS Advisory Committee on Oct. 3-4.
  • FDA will host a webinar in mid-November on “excellence in advocacy” to advise groups and individuals about common themes that may help them be more effective in working across sectors to accelerate the drug discovery, development and approval process for ME/CFS.
  • On Dec. 20, 2012, an FDA advisory committee will hold an open meeting to discuss the application for new drug approval for Ampligen submitted by Hemispherx Biopharma. Under current FDA regulations, a decision on the application will be made by Feb. 2, 2013.
  • FDA will convene a scientific stakeholder meeting, open to the public, in spring 2013 to identify measurable outcomes that can be used in clinical trials to evaluate the effectiveness of therapies. FDA will involve academic researchers, clinicians with long-term clinical experience in ME/CFS, patients and advocates in this meeting.
  • Forthcoming FDA guidelines on “enrichment strategies,” where a subgroup of patients within a diagnostic classification may demonstrate enhanced benefits of a treatment, may be useful to advance treatment of ME/CFS.

The FDA has established a new web page to house information about drug development for ME/CFS: http://www.fda.gov/Drugs/NewsEvents/ucm319188.htm

“The Pink Sheet Daily,” a publication from Elsevier for the pharmceutical industry, reported on the FDA call in its Sept. 13 edition. Here’s a link to that report (posted by Hemispherx Biopharma): http://www.hemispherx.net/ThePinkSheet_CFS_Drug_Development.pdf

A transcript of the teleconference has been posted by FDA here:

FDA asks that correspondence about ME/CFS be directed to the following email address: ME-CFS-Meeting@fda.hhs.gov

September 13, 2012