Thursday, June 6, 2013

FDA Drug Development Meeting Transcripts and Summary

Note: All of the following and more are located on the following page-

Transcript: Thursday, April 25, 2013 (PDF - 2MB)

Transcript: Friday, April 26, 2013 (PDF - 1.88MB)

Drug Development Meeting for Chronic Fatigue Syndrome and Myalgic
Encephalomyelitis Meeting Summary

On April 25 and 26, 2013, FDA held a public meeting with patients,
patient representatives and scientific, industry and regulatory
experts to discuss important issues regarding the development of safe
and effective drugs for the treatment of patients with chronic fatigue
syndrome (CFS) and myalgic encephalomyelitis (ME). CFS and ME are
serious conditions with no approved drug therapy currently available.
Below summarizes these two days of meetings:

Day One (April 25): This portion of the meeting focused exclusively on
gathering patients' perspective. FDA heard directly from patients
about their experiences with this debilitating condition. Discussion
focused on two key topics: 1) disease symptoms and daily impacts that
matter most to patients, and 2) the patient perspective on treatment
of this condition. Participants were also able to provide input,
through an open public comment session on additional issues with
regard to drug development.

This facilitated discussion with patients with CFS and ME provided a
particularly valuable opportunity for FDA to hear in patients' own
words about how they experience their disease. For instance, one
patient, when discussing a clinical term associated with the disease,
"post-exertional malaise," noted that she considered this aspect of
ME-CFS to be more like "post-exertional collapse," with exacerbation
of multiple disease symptoms. Many patients shared stories of having
had successful and productive lives prior to contracting CFS and ME
but now struggle with even the simplest tasks of daily life. They also
shared stories of debilitating episodes of CFS and ME that last days,
weeks, months, and even years, before abating.

Day Two (April 26): The second day expanded the discussion to include
input from the broader expert community, including health care
professionals, academics, researchers, pharmaceutical manufacturer
representatives, and FDA staff. Main discussion areas included 1) an
FDA review of the tools used by the agency to expedite the development
and approval of drug therapies for serious diseases such as CFS and
ME, 2) current treatments used to help patients with CFS and ME, 3)
challenges involved in conducting research that can help lead to the
development of effective drug therapies, and 4) creating a path
forward to achieve success in finding effective drug treatments for
patients with CFS and ME.

Among other things, this day's discussion underscored how widely
diverse each patient's symptoms of CFS and ME can be, and how these
disparities create challenges in designing useful studies that can
demonstrate the effectiveness of a medication being tested as a
potential treatment for the disease. Despite these challenges, a
number of outcome measures and trial designs exist that could be used
in clinical trials supporting drug development.

Day One was conducted in support of FDA's Patient-Focused Drug
Development initiative, an FDA commitment under the fifth
authorization of the Prescription Drug User Fee Act (PDUFA V) to more
systematically gather patients' perspective on their condition and
available therapies to treat their condition. As part of this
commitment, FDA is convening at least 20 public meetings over the next
five years, each focused on a specific disease area. Building on the
perspectives expressed on Day One, Day Two was a scientific workshop
to discuss how best to facilitate and expedite drug development for
CFS and ME.

To view a complete video of the meetings, visit A complete
transcript of the meeting will be posted at!documentDetail;D=FDA-2012-N-0962-0004.
Members of the public unable to attend either meeting can contribute
their comments via the meetings' docket:!documentDetail;D=FDA-2012-N-0962-0004
until August 2, 2013.

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