What FDA Wants

I think it’s clear what ME/CFS patients want to come out of the FDA’s Drug Development Workshop for CFS and ME. We want short, straight lines through the drug development landscape to FDA-approved treatments for our disease. But what does FDA want to come out of the meeting? To understand that, we need to understand the Patient Focused Drug Development (PFDD) Initiative.

The PFDD is part of the latest set of commitments between pharmaceutical manufacturers and FDA over the next five years (you’ll hear this described as “PDUFA Five”).  The FDA has committed to holding a minimum of 20 public meetings, and each meeting will focus on a specific disease area. Day One of the ME/CFS Drug Development Workshop is the inaugural disease specific meeting of the PFDD Initiative.

For the most part, patient perspectives inform FDA’s review of specific products. Just as we saw at the December advisory committee on Ampligen, patients offer public comment on a product and, perhaps, the need for approved treatments. FDA recognized the value in having a more broad and systematic way to collect patient points of view on their diseases, not limited to a specific product.

FDA reviewers need to evaluate a specific product within the broader context of the disease the product treats. The only way to adequately assess whether a product is safe and effective enough for people with a particular disease is to understand the impact of that disease. FDA needs to understand the severity of a condition, the most significant impacts on patients, and the range and effectiveness of current treatment options. The idea behind PFDD is to develop that contextual understanding with the direct input of the patients themselves. In addition, the patient input has to be collected in a way that will be useful to reviewers in the future. FDA’s goal for the PFDD is to explore and develop the process for collecting this patient input.

Patient perspectives on these issues will not only inform FDA’s internal review process, but can also help inform the drug development process for industry. It’s easy to see how this could happen in ME/CFS. If we asked a pharmaceutical company what symptom mattered most to ME/CFS patients, what would that company say? Probably that fatigue was the worst symptom, and that a successful treatment would reduce that feeling of fatigue. But I suspect that at the April meeting we will hear that post-exertional relapse is one of the most troubling symptoms, and a successful treatment should improve exercise testing results. Another likely symptom target is cognitive dysfunction, including impairment in focus, concentration, processing speed, and word-finding. If we are successful in communicating our input at the meeting, perhaps we will see a shift in the way treatments are developed for our disease, as well as how FDA reviews those treatments.

Through the PFDD, patients have the opportunity to educate FDA and industry about their diseases. The questions FDA has posed to the ME/CFS patient community (pdf link) are designed to elicit the input that FDA believes will be helpful. What are the most significant symptoms and how has the disease impacted your life? What treatments have you tried and what impact have the treatments had on you? This is the kind of input FDA believes it needs to create that contextual understanding of the severity of the disease and the available treatments.

After each PFDD meeting, FDA will produce a report of the meeting, which will be available to the public (including researchers and industry). The input patients provide in person at the meeting and through comments submitted to the meeting docket will form the backbone of that report. Hopefully, drug development companies will rely upon that report in targeting new compounds and designing clinical trials. Our input could help shape the drug targets and the ways trials measure the impact of the drugs on those targets.

Everyone – patients, FDA, drug developers – want to identify treatments that will successfully target the worst aspects of ME/CFS and that will move quickly through the FDA review process. Patients want treatments that are more than palliative. FDA wants to improve its assessment of product safety and efficacy by understanding the context of our disease. And drug developers want to make money by creating FDA approved products. PFDD meetings are an attempt by FDA to make progress toward those goals. The April meeting will be the first test of whether FDA has designed a process of collecting patient input that will accomplish it.