Yesterday, the FDA hosted a webinar for the ME/CFS community on “Working Together for Change.” Their stated goal was to show what has been successful for patient groups in the past in working with FDA and other entities. The slides and recording of the meeting will be available on the FDA website, and I will update this post with that link when it becomes available.
Dr. Sandra Kweder, Deputy Director of the Office of New Drugs (Center for Drug Evaluation and Research, FDA) opened the meeting with a presentation similar to the one she gave at the CFS Advisory Committee meeting on October 3, 2012. She reviewed the history of the FDA and the role of CDER in ensuring that only safe and effective drugs are marketed to the public. Dr. Kweder explained the review process and the unique challenges of evaluating drugs for conditions that do not have objective measures of disease state or signs. She suggested that ME/CFS advocates could learn from the process other disease groups have followed in addressing those challenges, such as irritable bowel syndrome, functional dyspepsia, depression, and fibromyalgia. Dr. Kweder’s advice included: partnering with organizations and centers equipped to conduct trials; recognize that a research definition is for research; filling in the gaps and remember that the science matters; and to be creative in learning from other groups.
I saw a lot of chatter during and after the meeting expressing frustration that this presentation was so similar to the CFSAC presentation. Neither slide deck is available, but the presentations did seem very similar to me. However, there were almost certainly participants in yesterday’s webinar who did not see the CFSAC meeting. Dr. Kweder provided essential background on what FDA does and does not do, and it is crucial for all advocates to understand this. I think it is reasonable to spend some time at these meetings making sure everyone has some shared basic knowledge.
Richard Klein from FDA’s Office of Special Health Issues provided an overview of the ways FDA seeks inputs from patients. He reviewed the history of how this process has evolved, particularly since the HIV/AIDS crisis of the 1980s. FDA uses several mechanisms in this process, including the Patient Representative Program which places patient representatives on advisory committees reviewing drug applications. Mr. Klein emphasized several things advocates should understand about FDA:
- FDA works within a tight regulatory framework that restricts what the agency can and cannot do
- FDA can guide sponsors of drug development but cannot direct drug research and development
- FDA provides assistance but has no direct control over drug development
- FDA must follow strict confidentiality rules
- FDA is only one piece of a larger puzzle that includes researchers, industry, and Congress
- FDA has a strict oversight role and that is its only role
Mary Dwight, Vice President of Government Affairs with the Cystic Fibrosis Foundation gave a presentation on the path that the Foundation pioneered in working with FDA. Cystic fibrosis is a rare genetic disease affecting approximately 30,000 patients. In 1955, the life expectancy for a child with cystic fibrosis was 5 years; today the life expectancy is to the 40s and beyond. This drastic change is the result of a successful drug development process, and the Foundation has played a critical role in that process. Ms. Dwight explained how the Foundation achieved this remarkable success:
- Good science about the disease was essential, including the discovery of the cystic fibrosis gene by Dr. Francis Collins (now Director of NIH) in 1989.
- Drug developers were reluctant to invest, so the Foundation provided venture philanthropy to spur investment. Drug developers also needed data and expertise, so the Foundation stepped up there as well.
- They created a patient registry and Foundation-accredited care centers. This enabled them to connect the drug developers with the patients for clinical trials.
- They created a national therapeutic network, coordinated from one central location, to provide best practices in study design, standardized research procedures and expert advice on correct outcome measures. They also assemble scientific consortia to foster communication and collaboration among researchers.
- The Foundation sees FDA as a partner to talk about patient data, but that it is the patients’ job to bring the data to the table. FDA only evaluates data, it does not collect the data.
Ms. Dwight also said that as science and treatments have evolved, their approach has evolved too. She recommended that if the ME/CFS community does nothing else, it should collect natural history and group data for the patient population.
Pat Furlong of the Parent Project Muscular Dystrophy started her presentation by saying that ten years ago the Duchenne community was where the CFS community is now. Duchenne is a progressive and fatal genetic disorder affecting boys, with approximately 20,000 new cases per year. She said that stakeholders (patients, researchers, regulators, etc) interact with non-living parts of the system (regulations, etc) in an ecosystem of drug development. Ms. Furlong said that they modeled their process on the work of the Cystic Fibrosis Foundation, and started with a patient registry. While there is still no treatment for Duchenne, there are now drugs in the pipeline. She categorized the elements of a successful program:
- Clinical infrastructure – patient registry; identify endpoints; research networks
- Advocacy – increased money from NIH, DOD and CDC; direct engagement of regulators
- Education – patients; drug developers; clinical trials; management of expectations (because this is a lengthy process)
Ms. Furlong said that her organization’s Board created a policy statement on its goals for FDA advocacy. They’ve held two meetings with CDER to discuss the policy and what data FDA needs. They are working with a consulting firm to develop and publish a risk-benefit framework for rare diseases, and are developing a parent survey on risk tolerance.
Participants had an opportunity to ask questions of the panel. Patricia Carter, myself, Marly Silverman, Courtney Miller, and Hillary Johnson made statements or asked questions. Steve Morin of the Office of Special Health Issues also invited people to submit questions since we ran out of time.
Overall, I found this webinar to be very informative, and not a little daunting. The CFS community is a long way from accomplishing what the Cystic Fibrosis and Duchenne communities have done. However, there are steps being made in the right direction. FDA will hold a meeting next year to address the issues of endpoints and outcome measures in CFS. The CFIDS Association is competing for prize money to fund patient registry projects. Several organizations, including the Chronic Fatigue Initiative and the CFIDS Association have established biobanks. CFI’s Bank will house 200 patient and 200 control samples; the Association’s Biobank has enrolled more than 500 patients and controls. One of the Association’s grants this year was to a pharmaceutical company to screen approved drugs for applicability in CFS. Finally, on December 20th, the FDA will hold an advisory committee meeting on Ampligen, the only new drug currently in the pipeline for CFS.
The main takeaway from the webinar was this: patients, advocacy groups, researchers, industry, NIH, CDC, FDA, legislators and policymakers must all cooperate to help make treatment for CFS a reality. As Mary Dwight said, forced collaboration among the players is essential but the patient is the only player who participates in every step of the process and is the only one with a holistic view of the disease. We must be at the table at each stage in the process.