On Wednesday, April 23, 2014 FDA held a webinar to discuss the draft Guidance for Industry CFS/ME: Developing Drug Products for Treatment. We were on the webinar and are happy to provide this summary. While there is still much work to be done, we are greatly encouraged that ME/CFS is receiving this kind of attention on a federal level.
The draft guidance is intended to assist sponsors in the development of drug products for the treatment of Chronic Fatigue Syndrome/myalgic encephalomyelitis (CFS/ME). The guidance focuses on specific drug development and trial design issues that are unique to the study of CFS/ME and on the FDA’s current thinking on how effective treatments can be developed for CFS/ME. The points discussed in this guidance may not be applicable to all drug products. The FDA encourages sponsors to design clinical programs that fit their particular needs and to discuss their planned approach with the Division of Pulmonary, Allergy, and Rheumatology Products (DPARP).
The webinar began with a brief review of the contents of the draft guidance. This document offers industry input on trial design and duration, potential efficacy endpoints, the need for patient reported outcomes (PRO’s) and safety considerations.
During the webinar it was stated by the presenters that “because CFS is so widely diverse and symptoms are subjective and individualized by patients, patient reported outcomes (PRO’s) are extremely important to the process.” This further acknowledges the patient’s value and import in the process of solving ME/CFS.
They went on to note that drug development requires multiple partners. The FDA’s role is to advise on the regulatory standards for product approval – the draft guidance articulates the expectations for drug approval. In essence, letting industry know what is necessary and expected in order to successfully bring a therapy to market. The intent of the guidance is to encourage pharma to more deeply engage in the process
When asked if they understood the severity of the disease and its dramatic impact on patient’s lives, the responded by saying “FDA recognizes the extreme unmet need.” Panelists went on to acknowledging the importance for the drug development groups to find ways to include the severely ill. Stating that the draft guidance was created and the webinar held because “We want patients to be served by companies that can develop therapies for the disease”
Currently the draft guidance is just that – a draft. We are currently in a period of open feedback with all comments to be submitted by May 12, 2014. The Solve ME/CFS Initiative has reviewed the document with its Research Advisory Counsel and will be submitting our review prior to the deadline. Once this review and comment compilation is complete, we will share our response in a subsequent blog post.
If you are interested in hearing the webinar in its entirety, it was recorded and will be available on the FDA website HERE after April 24th.
To review the draft guidance document, you can access it HERE:
Should you choose to submit your own comments, please be aware of these requirements:
- Include docket number with all comments: FDA–2014–D–0264
- Submit electronic comments to http://www.regulations.gov
- Submit written comments to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852
- For questions regarding this draft document contact Dr. Janet W. Maynard at 301-796-2300