This is not my written testimony. That is here.
This post contains my predictions, which I posted elsewhere before the meeting, based on their misplaced focus on symptoms.
UPDATE: the FDA's "Meeting Summary", April 26, 2014, makes it clear that all of the concerns in this post have been borne out. The FDA is not serious.
I am still having problems getting that testimony submitted. They have not posted it to the docket, even though I was told long ago that it was hand-carried.
At this point, I would submit this post as a second written testimony if I could.1
I am grateful that the FDA has published its draft agenda. I am grateful that the people who matter — the sufferers — are being included.
However, the focus on symptoms is problematic.
Here is the accompanying blurb:
The discussion on April 25 is dedicated to engaging patients, as part of a participatory process, in dialogue on the most significant symptoms and negative impacts experienced in their daily lives as a result of their condition.
I do not believe that this is the best way to include sufferers.
To me it is a problem that the draft is focused on symptoms.
This is not a side point
The best way to show respect to sufferers is to get the fundamental nature of the disease correct in the first place.
Many people have died from the disease. The disease is serious.
It is better to ask sufferers what is needed instead of guiding their answers with lifestyle-based questions.
Those questions steer away from progress on the disease.
This is not a side point. It affects the types of drugs being developed.
We do not need the development of more stimulant drugs. We do not need antidepressants.
Instead we need sincere research into drugs (and non-patentable substances) that seek to address, at the very least, the underlying PATHOPHYSIOLOGY of the disease.
There are some that exist (for example, immune modulators and methylation supplements), but they are under-studied and we need far better ones.
We also need, for example, clinical trials on antiretrovirals.
This concern is not theoretical
As we speak, an amphetamine is being "developed", meaning, as far as I can tell, that somebody wants to label the existing molecule for sale for the disease.
Given the history of this disease, it is possible that this meeting was, in fact, specifically designed to push such drugs, or other evasions from the pathophysiology.
That is how such meetings sometimes run: the outcome is determined before the meeting even begins.
Including sufferers is good.
However, the FDA's choice of questions about lifestyle and symptoms instead of root causes and pathophysiology suggests a possible attempt to get them to answer the way they want.
This is because making the questions be about symptoms makes the meeting be about symptoms. This in turn promotes symptom-based drugs.
Panel 1 Questions:
- What are the most significant symptoms that you experience resulting from your condition? (Examples may include prolonged exhaustion, confusion, muscle pain, heat or cold intolerance, etc.)
- What are the most negative impacts on your daily life that result from your condition and its symptoms? (Examples may include difficulty with specific activities, sleeping through the night, etc.)
a. How does the condition affect your daily life on the best days and worst days?
b. What changes have you had to make in your life because of your condition?
— Draft agenda
Questions about symptoms and lifestyle constitute an invitation to promote purely symptom-based drugs.
I have pointed out before the selection bias inherent in the meeting: only people who are relatively well will attend.2
Not a single hospitalized, tube-fed, bedridden, or housebound sufferer will physically attend the meeting.
To my knowledge, the bereaved of sufferers who died will not attend the meeting.
This will bias the answers regarding lifestyle and symptoms.
If the FDA is insincere, it will help pharmaceutical companies push more lifestyle drugs and palliative symptom drugs on sufferers.
Stimulants, antidepressants, and Lyrica can be harmful. Existing ones are already usable off-label should there be a need for them. This is the wrong direction to go in.
In a previous FDA meeting, there was an endpoint called the "salad endpoint": if you can make a salad, then you are better.
This was completely well-intentioned in the context of NK cell counts (as it was first stated). But it is a problem if it is misunderstood.
It fits perfectly with the widespread, misopathic myths about the disease being a "condition" involving effort (as opposed to attacking almost every system in the body with 50 symptoms not being unusual).
The FDA liaison praised the salad endpoint. Perhaps that was with good intentions, but we have no evidence of that from the FDA as a whole so far.
One can imagine the advertisement now:
If you can't make a salad, try Uppita stimulant! Ask your doctor now! [May cause serious worsening of the disease it is ostensibly targeted at. May cause death.]
Endpoints the FDA needs to consider
If symptom-based endpoints are necessary, I propose more like these:
- the "did not die and can leave the hospital" endpoint
- the "is not in extreme pain" endpoint
- the "does not need a feeding tube" endpoint
- the "is no longer bedridden" endpoint
- the "is no longer housebound" endpoint
- the "can enter a moldy room" endpoint
Is the FDA serious?
I expect a serious agency to acknowledge the most fundamental fact about the disease: the fact that it is a serious disease.
While I am grateful for the outward appearance of taking the disease seriously, there is no evidence that the FDA is sufficiently serious about the disease.
While we will always welcome sincere efforts at progress, given the history and the above, it is not unreasonable for sufferers to conclude that this is more evasion.
The only way to convince us otherwise is to make tangible progress immediately.
I believe that this meeting is at least in part designed to pacify advocates and help out a few drug companies who want to sell palliatives like stimulants.
I will be happy to be corrected, but I do not welcome apologia from those who profit (or believe that they do) from the status quo.
I implore all people considering attending the meeting to:
- Please attend the meeting
- Please make it plain that we are talking about a DISEASE
- Please include the ENTIRE range of severity in everything related to the disease
I added two sentences ("Many people have died from the disease. The disease is serious.") and a note in a footnote.
I submitted testimony about the selection bias inherent to the the official meeting. Mary Gross at the FDA graciously promised to include it. Her email to me was dated Wed, Mar 20, 2013. Randi at the FDA later confirmed and Mary Gross again confirmed on Apr 1, 2013.
(Note onas far as I know the testimony has not been added to the docket. Thus the following is possibly out of date.)
I want to sincerely thank both of them. It is greatly appreciated, especially as I was having difficulty using the web software due to accessibility reasons. Their assistance, to me, demonstrates good intentions.
Physical mail is a hardship for severe sufferers, which is a point that I believe the FDA was unaware of.
It is critical that the FDA allow email (NOT only website) submissions in the future to avoid accessibility issues with websites.
Selection bias due to channels of communication is a problem.