On September 13, a group of 50 FDA officials, pharmaceutical developers, and ME/CFS patients & advocates met via a 90-minute telephone conference. It was a big step in the FDA’s plan for hastening development, testing and approval of drugs to treat ME/CFS – a serious disease with ‘unmet medical need’.(1)
The FDA.gov page dedicated to sharing information about these activities has now posted the transcript of this discussion (www.fda.gov/downloads/Drugs/NewsEvents/UCM320310.pdf). It includes input from many stakeholders, and is well worth reading.
The meeting was led by Dr. Sandra Kweder, MD, deputy director of the FDA/CDER’s Office of New Drugs. Just a few of the key points she covered in this very interesting meeting are as follows:
• Importantly, Dr. Kweder established that the FDA considers ME/CFS to be in the category of “serious or life threatening diseases.” And that the FDA is committed to making promising drugs available to individuals with such diseases for which there are as yet no approved drugs as quickly as possible via “the rapid development and review of these types of therapy” – a process called ‘the accelerated approval process.’
• Second, Dr. Kweder emphasized it is very important to identify factors that if measured can be considered trial outcome measures. Using HIV just as an example, she noted, what really revolutionized the study of drugs for HIV/AIDS treatment “was finding something to measure in the clinical trials. In that case they had the… fortune or misfortune to be able to identify some marker in the blood that was predictive of the clinical course of the disease. In the beginning it was a type of white blood cell, CDR lymphocytes.”
• As the drug developers further explained, they have learned from experience, “you’ve got to be able to define the condition well… and know where are the rules about studying it. What am I going to have to show in order to get a drug approved.”
• So, respecting the need for measures and rules, said Dr. Kweder, “We are planning to hold a scientific workshop that explores how to identify valid, reliable, quantitative outcome measures to determine if disease symptoms do improve with specific interventions.”
• The workshop, which CDER (Center for Drug Evaluation and Research) plans to host in spring, 2013, will be open to the public and will “explore a multitude of scientific issues about identifying valid, reliable and measurable outcomes to determine if disease symptoms improve with intervention.” As part of this discussion, says Dr. Kweder, “we want to hear from academic experts, from clinical practitioners who are taking care of patients with these conditions, and patients themselves.”
• In the meantime, the FDA will describe the drug review process in detail for the benefit of all viewing the CFSAC meeting in Washington Oct 3 & 4.
• And in mid-November (date to be determined) the FDA will host an Excellence in Advocacy webinar that will “advise patient advocates or advocacy groups on how to come together and develop common themes to… further your mutual goals” with regard to the process of getting drug therapies for ME/CFS approved.
1. See a brief report circulated after the Sep 13 telephone conference by Rivka, a patient activist who participated in the meeting. “FDA Pronounces ME/CFS ‘Serious and Life Threatening Disease in Sep 13 Meet.”