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.
Thank you Jennie for this report and account of the FDA teleconference. I agree with you that the FDA both at the April meetings and yesterday were very respectful and seem genuine in their desire to listen and learn from us.
There is no doubt that they are seeking our input and that they seem to get it as evidenced by their “The Voice of the Patient” report which was excellent.
I just hope that all these words will translate in to action when needed.
I listened in to the call but wasn’t feeling well. One concerning comment to me was in reply to a question about the FDA’s response to Ampligen and the fact that the panel members did not seem too educated about ME/CFS. The response was about the unique constraints of panel selection. They seemed to say that they are not allowed to pick anyone with experience in research of the disease, if I understood correctly.
This is pretty disturbing both because of future FDA panels for drug approval and for the fear that this might hold true with the selection of the IOM panel for redefining ME/CFS.
That’s not how I understood it. FDA has extremely strict conflict of interest rules for advisory committees. If a potential member has a financial connection to the drug company, obviously they are excluded. But a member can also be excluded for a financial relationship with a competitor. If they have a demonstrable bias for or against the product (or a competitor”s product), they are excluded. And it’s not just direct financial relationships but it includes things like stock ownership. At the training I attended, we were told it takes four months to go through conflicts clearance for an advisory committee meeting, and based on the forms I had to fill out to join the program they look at EVERYTHING.
Given how few experts we have in our field, and given how interrelated everyone’s work is, I can see it being a challenge to find enough experts without bias or conflicts of interest.
Here, here!
Good news with the teleconference–thank you!
Thanks as always for the valuable information!
I’ve been raking through various documents. Elizabeth Unger seems to be a dedicated medical researcher when it comes to HHV, but the impression one gets is that she’s been listening to people who promote the ‘exercise’ model of ‘curing’ ME/CFS. In a letter quoted on Phoenix Rising, Peter White has written to Swiss Re that exercising seems to treat the cause of ME. (Based on the PACE Trial). Elizabeth Unger is quoted as having said that ‘exercise is not negotiable’.
It seems that if these quotes are accurate, it may have already been decided that exercise will cure us, and therefore there is a danger that both the definition arrived at, and the research which is proposed to be done (in which a 2-day exercise test has already been eliminated, but prior research indicates that it is the 2-day test which will show post-exertional deterioration) will be shaped towards supporting this conclusion and recommendation. It is completely unethical that an exercise which is supposed to be trying to arrive at an accurate definition, and research which is purported to about finding something out are instead intended to be shaped to a pre-decided conclusion.
Alarm about this seems to me entirely justified.
One question I would like to know the answer to, and maybe someone out there has the answer – how did ME/CFS get labelled an issue of ‘Women’s Health’? Uterine cancer, breast cancer, other gynecological illness are by definition concerned with ‘Women’s Health’, being necessarily gender-linked. The gender balance in ME/CFS is tipped towards the female, as is the case in a number of other auto-immune disorders, but that does not mean that they are issues of ‘Women’s Health’. There are many men who suffer from ME/CFS, and occasional historical outbreaks have occurred in all-male settings.
This seems to me possibly to go right back to McEvedy and Beard’s (unfortunately successful) effort to get ME labelled ‘mass hysteria’, comparing it to an outbreak of what they also dismissed as mass hysteria in an isolated boarding school for girls.
Looking at the fact that in the Royal Free Outbreak, only 12 inpatients became ill, a larger number of doctors, and the largest number of nurses: suppose we make the connection not of gender, but of level of prior muscular exertion involved in the activities of those who succumbed? The patients were presumably already resting in bed. The doctors, depending on their speciality, will have had a varying range of physical exertion in the course of their work. The nursing profession involves a very high level of physical exertion, probably more so in 1955, before hospitals will have been equipped with modern equipment for lifting patients. No link to gender, rather a link to the mostly culturally unacknowledged fact that women’s work is often extremely physically demanding.
This is quite hypothetical, but an illness to which both men and woman can succumb is clearly not an issue of ‘women’s health’ – so how did ME/CFS get so labelled in the first place? One gets a sense that this label has contributed to the disrespect for patients which seems to have been an ongoing aspect of dealings concerning this illness. Please tell me if I’m wrong!
Your point is well taken, Nancy, about someone’s tactics of slotting ME/CFS under women’s health– ME/CFS is NOT gender specific–nor is it ethnicity specific — nor age specific–AND it IS GLOBAL and it IS spreading!!! Seems like a purposeful disreputable ploy (another one)!!
To the pacing–that is a ridiculous nonsensical dangerous methodology of purporting to cure ME/CFS.
In fact, it is a no-brainer that that is how we have learned ‘ourselves’ to ‘exist’. In other words–we cannot carry on a normal life with NO ENERGY, etc., etc. with ME/CFS–and if we try to do something we must do it in very small increments and then rest–soooo, someone’s study labelled this PACING ??? OK.
Larger increments of exertion–NOT just exercise–equals PEM or CRASH. Building up does NOT happen with ‘barely exisiting’ and ‘pacing’–more pushing worsens the symptoms and can worsen the disease itself. We need a scientific FIX that gets to the root of the disease; not this nonsense–that merely describes a ‘strategy’ to exist.
I have read several books on ME/CFS–only to find that the info presented was ‘gathered’ from ‘other sources’–and this equals a lot of MISinformation–written as FACT!!!
The EXPERTS that, in fact are experts, know that forcing exercise/exertion makes us worse. Those with an opposite opinion are WRONG and dangerously wrong for us!
Because the male anatomy and physiology is so different from the female, I believe it is a legal violation of the 14th amendment on discrimination and equal protection under the law and therefore should be taken out of the Office of Womens Health as it has an inherrent prejudicie for men receiving adequate research under Federal Law and Statute It is a discriminatory practice favoring or giving preferential treatment to one class of gender over another.