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.
I Heard the News Today, Oh Boy
What is there to say about something like the Boston Marathon bombing? There is no sane way to reconcile the gruesome images, the suffering and destruction with our need to believe that we are safe. We are privileged enough in this country to think that this sort of thing does not happen here. Once I confirmed that everyone I love in Boston was ok, my real-world connection to the event faded. But this morning, my mind and heart are still bound up in the news.
Being housebound makes me vulnerable in an odd way. On the one hand, I am remarkably safe in my home and neighborhood. I rarely go in to the city or to places with large crowds. But on the other hand, I am an automatic audience for big news events. I don’t have a meeting to go to, or kids to pick up from school, or any other demands on my attention. Most of the time, it’s just me, the tv, Facebook and Twitter. If I choose to tune in, I end up vicariously experiencing these events.
I watched the Columbine shootings unfold. For a week after 9/11, I only turned off the tv to sleep. Six years ago today, I followed the Virginia Tech shootings on CNN, and I held my breath between the phone calls from my brother who was there. It has gotten to the point where I start to cry as soon as I learn about an incident like these. Sometimes I have the self-discipline to avoid the media if I think the news will be too upsetting. I stayed off Twitter and Facebook for days after the Newtown shootings, and I still have not seen any footage from that day. I long ago decided that tv news is shallow, and frequently borderline moronic, so it’s easy to avoid that. But if I pay attention – and Twitter makes that incredibly easy – I get sucked in to following every update and rumor.
Emotional shock and distress quickly induces a cascade of physical exhaustion, pain, and brain fog. The more I watch, the worse I feel. Compounding the simple stress is an overwhelming feeling of powerlessness. I am trapped in my home, alone with the images of people who need help. There is nothing I can do. I remember the despair I felt after 9/11 because I couldn’t simply drive to New York and pitch in. After the Virginia Tech shootings, my brother started volunteering for an emergency services provider. I am limited to donating to the Red Cross or knitting afghan squares. This is not a substitute for directly helping people face to face. Watching people suffer, and being unable to do anything about it, feels like sandpaper against my heart.
This morning, I’m thinking about the people who will now join the ranks of the disabled. There are reports of people who lost legs or feet. Sure, they’ll get prosthetic limbs and rehab and lots of attention. But their lives will never be the same. Perhaps there will be people disfigured by the blasts, or people who develop post-traumatic stress disorder. Some of them may have great family support and health insurance, but others may not. In the moment of the explosions, everything changed for these people. They have to run a different marathon now. This is the marathon of doctors and procedures and medication and paperwork and learning to live with a changed body. This is the marathon of people helping in the immediate aftermath and then fading away and going back to their lives. This is the marathon of answering the “how are you?” and “it could have been worse” comments. This is the marathon of the sick and injured.
I don’t know what it’s like to be the victim of any crime, let alone a crime like this. But I do know about suffering and endurance and navigating a changed life. The real crisis is not the moment of explosion. It’s everything that comes after. I know a little bit about that marathon, and I wish I could help other people on that path. But my own marathon keeps me imprisoned, acutely aware that others are suffering and completely unable to help them.