Regulatory Focus™ > News Articles > Public Meeting to Focus on Regulatory Issues Associated with Chronic Fatigue Syndrome

Public Meeting to Focus on Regulatory Issues Associated with Chronic Fatigue Syndrome

Posted 11 March 2013 | By Alexander Gaffney, RAC

The US Food and Drug Administration (FDA) is set to hold a workshop on the development of therapies for Chronic Fatigue Syndrome (CFS) (also known as Myalgic Encephalomyelitis), a poorly-understood and often controversial syndrome identified by both the agency and patient advocates as an area in significant need of treatment options.


CFS affects patients by causing persistent fatigue. It is unknown if it is a single condition or a group of them, as there are no tests to determine its cause. Despite some evidence that CFS could be caused by xenotropic murine leukemia virus-related virus (XMRV), that theory has since been debunked by subsequent scientific inquiries.

That leaves patients in something of a bind: the effects CFS are real, but without an understanding of its underlying causes, researchers and manufacturers are unable to target potential or existing therapies to treat it.

That bind was put on display and amplified in February 2013, when FDA-following the advice of one of its advisory committee panels-did not recommend Hemispherx's Amligen (rintalolimod), saying it needed additional clinical evidence and data before it could consider approving the drug.

FDA's decision leaves patients without a treatment and, worse, with one less promising prospect to buoy their hopes.

A Focus on Patients

But patients may have one shred of hope: FDA has heard their concerns, and is interested in discussing development issues with the public, and in particular patients and members of industry, in a public forum.

Under the Food and Drug Administration Safety and Innovation Act of 2012, FDA was charged with setting up a "Patient-Focused Drug Development Initiative," which is set to seek out opinions from patients in 20 different disease areas over the next five years.

"The human drug and biologic review process could benefit from a more systematic and expansive approach to obtaining input from patients who experience a particular disease or condition," FDA explained in a Federal Register posting announcing the program in September 2012.

Practically, the program means FDA will start to assess the benefit:risk paradigms that each patient group is willing to accept in return for a treatment. A patient group suffering from an under-treated but otherwise medically benign condition-trichotillomania, for example-might be less willing to accept high levels of risk in return for an effective treatment. Conversely, those suffering from CFS might be more willing than most to accept a drug that might either be less effective or more dangerous that FDA's benefit:risk paradigm ordinarily would permit.

CFS Meeting to Address Concerns

A preliminary meeting in October 2012 got the process started for FDA and patients alike, and now the agency says it wants to sit down for a meeting specifically on CFS. That meeting will be held on 25 April-26 April 2013.

The first day will be dedicated to discussions with patients, who are invited to give feedback on how CFS impacts their lives and quality of life. The second day will bring in academic and government experts, patient advocates, patients and clinicians to assess "how to identify sound, quantitative outcomes measures that can be used in clinical trials to determine whether disease symptoms improve with specific drug interventions.

FDA also said it wishes to focus discussion on a number of questions:

  • What are the most significant symptoms that you experience resulting from your condition?
  • What are the most negative impacts on your daily life that result from your condition and its symptoms? (Examples may include difficulty with specific activities, such as sleeping through the night.)
  • How does the condition affect your daily life on the best days and worst days?
  • What changes have you had to make in your life because of your condition?
  • What treatments are you currently using to help treat your condition or its symptoms?
  • What specific symptoms do your treatments address?
  • How has your treatment regimen changed over time and why?
  • How well does your current treatment regimen treat the most significant symptoms of your disease?
  • Have these treatments improved your daily life (for example, improving your ability to do specific activities)?
  • How well have these treatments worked for you as your condition has changed over time?
  • What are the most significant downsides of these treatments (for example, specific side effects)?

Registration for the meeting must be sent to FDA by 8 April 2013. Details are available in the Federal Registerannouncement. 

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