A new initiative launched by the Food & Drug Administration (FDA) will bring patients and other stakeholders together with agency officials to better understand patient experiences and how those experiences should influence decisions the agency makes to regulate availability of new treatments. Named the “Patient-Focused Drug Development Initiative,” this set of meetings is mandated under a law, PDUFA-V (see inset), passed on July 9, 2012. Twenty conditions will be chosen for this focused attention and meetings will be held between 2013-2017. CFS is one of 39 conditions the FDA included on a preliminary list of topics being considered for this meeting series. This article in the online magazine PharmaTimes describes the new initiative.
A public meeting was held on October 25, 2012 at the FDA to obtain public input on the conditions being considered as well as others that may not have been on the preliminary list. The three-hour meeting attracted about 100 participants representing a wide spectrum of diseases. Four individuals, including Solve ME/CFS Initiative Board chairman Amy Squires, spoke in support of keeping CFS on the final list of 20 conditions. Jennie Spotila posted a summary about the meeting on her blog, Occupy CFS. The meeting was webcast, although this fact was not included in the public announcement or other meeting notices.
FDA has also invited brief written comments through its online docket system. The deadline for submitting comments on this topic is November 1, 2012 at 11:59 p.m. (Eastern time). Comments are limited to 2000 characters, inclusive of spaces.
The Solve ME/CFS Initiative submitted this statement for the written record and Ms. Squires’ comments made at the meeting were similar:
The Solve ME/CFS Initiative applauds FDA’s affirmation that CFS is a serious and life-threatening condition, as stated during a 9-13-12 stakeholder conference call and on 10-3-12 at the DHHS CFS Advisory Committee meeting. This is an important beacon of hope to people with CFS and a critical signal of opportunity to industry and academia. With at least 1 million Americans affected, CFS represents a vast unmet need and large potential market.
We applaud inclusion of CFS on the preliminary topic list for the patient-centered drug development meetings and urge FDA to keep it on the final list.
There has never been a systematic effort to identify effective treatment for CFS and there remains no single or polypharmacy regimen shown to treat the symptoms or modify disease. In the void patients and physicians use a wide range of off-label therapies. On PatientsLikeMe.com people with CFS report using 800+ different treatments. That stat reflects a lot of trial and error with uncertain results, risk with possibly no benefit and great expense to the individual and health care system.
CFS meets all published criteria for this series of meetings. A focused session on CFS would allow for productive exchange with people with CFS about 4 key topics: condition severity; relative risks/benefits which should be viewed as tolerable in regulatory decisions; greater understanding of measures of benefit that matter most to patients; and innovative ways to measure and monitor dynamic symptoms and their impact on daily living.
We also encourage FDA to also undertake a meeting on chronic comorbid pain conditions, of which CFS is one. Exploring comorbid conditions as a group (in addition to individually) would generate new insights across conditions and reveal how therapies for one might benefit others.
The last FDA-hosted meeting on CFS was held in 1993. The agency’s attention to CFS is overdue; including it in this meeting series will advance treatment-focused research.
Please consider submitting a comment to FDA to support inclusion of CFS on the final list of conditions for the Patient-Centered Drug Development Initiative.
- Here is the URL for submitting your comments: http://www.regulations.gov/#!docketDetail;D=FDA-2012-N-0967
- Here are some tips for effective comments (from the regulations.gov site): http://www.regulations.gov/docs/Tips_For_Submitting_Effective_Comments.pdf
The FDA has scheduled a webinar titled, “Excellence in Advocacy,” for Nov. 15, 2012 at 2:00 p.m. (Eastern time). Watch the FDA’s page for Drug Development for ME/CFS and Research1st for details on how to connect to this webinar.October 30, 2012