I’ve been very critical of HHS and how they have handled communications and engagement with the ME/CFS patient community, and the fiasco surrounding the Institute of Medicine study is a sinkhole of terrible engagement. But if HHS and/or the IOM want to improve upon past mistakes, I offer Exhibit A: the FDA.
Listen First, Listen Fully
Coincidentally, the Institute of Medicine recently hosted a workshop on Partnering with Patients to improve healthcare. One of the key messages from the workshop was the importance of listening to patients first, and listening to them fully. This is precisely what FDA has done in the last year or so.
First we have the Patient Focused Drug Development meeting on ME/CFS last April. Patients were very specific in their description of symptoms and treatments, but what blew me away was how carefully the FDA employees were listening to us. They asked excellent clarifying questions, took extensive notes, and several commented on how much they learned.
I know some patients were frustrated that this meeting seemed to be such a revelation to the FDA, saying that patient comments at CFS Advisory Committee meetings should have taught them about this disease. But a static five minute slot at a CFSAC meeting is barely adequate to convey one piece of information, let alone the multiple messages we frequently need to communicate. The FDA meeting was interactive, accommodating questions from FDA but also clarifications from the patients and caregivers. The format and the purpose of the meeting facilitated more detailed communication from patients, and the FDA was there to listen. There is simply no comparison between a CFSAC meeting and the PFDD meeting.
How well FDA listened is captured in the first report on the meeting, The Voice of the Patient Report: Chronic Fatigue Syndrome and Myalgic Encephalomyelitis. This report summarizes the comments we made at the meeting, as well as the comments received through the docket. Data from the two surveys conducted prior to the meeting were also considered.
As many patients commented online and to me personally, this report describes our disease. They got it. The report emphasizes the importance of post-exertional malaise, recognizes the seriousness of the disease and its impacts on our lives, and describes the breadth and severity of our cognitive dysfunction. I can’t think of another document produced by the government that does that. If the Institute of Medicine study creates a clinical case definition for ME/CFS that matches this report, then we might be on the right track.
This report will also serve an internal purpose at FDA. On the teleconference FDA held with the ME/CFS community on October 16th, Sara Eggers said that they have already received positive feedback from colleagues at FDA about how helpful the report is to them. During the discussion period, Donna Pearson made the brilliant suggestion that all members of advisory committees considering drugs for ME/CFS receive the report as part of the meeting materials, and Dr. Sandra Kweder immediately acknowledged that as a good suggestion.
But a single report will not stimulate drug development on its own, and FDA does not appear to be sitting still. We learned on the October 16th call that the FDA is drafting a Guidance to Industry document specifically for ME/CFS. Guidance documents capture FDA’s current thinking on a topic, identify specific endpoints or outcome measures that FDA finds valuable for a specific disease, and send a strong signal to industry about the worthiness of a topic or disease. Dr. Janet Maynard (who is replacing Dr. Theresa Michele as FDA ex officio on the CFSAC) said that FDA hopes to publish the draft guidance in spring 2014. This is another good outcome: the public is able to comment on draft guidance documents, and we will have to take full advantage of that opportunity in the spring.
By listening to ME/CFS patients first, and listening fully as demonstrated in the Voices report, FDA sent our community a powerful message: we hear you, we know you are seriously ill, and we want to help. We have responded with respect, while not giving up on the tough questions as demonstrated by multiple advocates on yesterday’s call.
A Thought Experiment
Imagine how different our interactions with the Office of Women’s Health or the other agencies would be if they modeled the FDA’s engagement with us. For example:
- What if the CDC PCOCA calls answered all questions in real time, rather than selecting from advance submissions through email?
- What if the NIH held teleconferences with groups and individuals about the planning of the Evidence Based Methodology Workshop, just as FDA did prior to the April meeting?
- What if the Office of the Assistant Secretary would pay us the courtesy of responding to our multiple inquiries about the allegations of intimidation?
- What if, instead of pursuing the IOM study in secret, the Office of Women’s Health had considered ideas and concerns from the public on how to address the case definition issue?
FDA is not just paying lip service to listening and then chucking our feedback in the trash. They have stated on multiple occasions (including yesterday) that our feedback has shaped their efforts going forward. We don’t like everything FDA does, and we have no guarantees about what the guidance document will say. But they have demonstrated through their behavior that they are willing to listen, to hear, and to treat us with respect. Imagine if we got that same message from CDC or NIH or the Office of Women’s Health. What if we could actually believe – based on their actions – that they were willing to listen and to take what we say and apply it going forward? Can you imagine?
If HHS wants to improve its relationship with the ME/CFS community, they would do well to see FDA’s engagement as a positive case study. If the Institute of Medicine wants to avoid ending up distrusted and reviled like the Office of Women’s Health, they should look to FDA as one possible path forward. Or the IOM could simply take its own advice and listen first, listen fully.