FDA Stakeholders Meeting for Chronic Fatigue Syndrome: Goals and Objectives



To accelerate treatments and access to treatments for ME/CFS patients (in the absence of a wholly agreed upon definition, a diagnostic biomarker* and defined subset populations).


1.   Expediting treatments to patients – distinguish the various accelerated pathways including and not limited to fast tracking, accelerated approvals, post-marketing trials, and other regulatory mechanisms

Discussion Considerations:

  • Range of treatment trial strategies (combinations of drug treatments and/or broad-spectrum of      patients)
  • Parallel tracking
  • Expanded population trials (increasing population size as shows benefit)
  • Priority reviews
  • Use of cross functional review teams, etc.

2.  Examine and define benefit risk and quality of life for chronic illnesses; specifically looking at ME/CFS

Discussion Considerations:

  • How does FDA currently evaluate chronic illnesses
  • Patient and clinician considerations on risk and benefit
  • Safety versus risk
  • Severity/morbidity
  • Persons freedom to make health choices
  • Promote and protect public health
  • Other chronic illnesses

3.   Combine the considerations and conclusions from objectives #1 & 2 and re-examine through benefit risk lenses treatments currently in the pipeline and those being used off-label to determine the path forward.

Discussion Considerations

  • Healthcare access/Trial access
  • Availability of product
  • Insurance, Medicare, Medicaid
  • Costs of individual healthcare and total impact to nation
  • Long term care, home health, etc.
  • Timing versus Patient’s Health

4. To educate and engage a broader healthcare-wide community on the challenges associated with ME/CFS. In addition to broadly educate the community at-large the regarding the implications of this disease, treatments and access within all the layers of the healthcare system.

A survey to assist us in this process will ensue shortly.

*Please note for the initial stakeholders meeting we do not want to include the discussion of biomarkers. We anticipate there will be follow on meetings and biomarkers should be included at that time along with a number of other topics.

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