The Voice of the patient - FDA report ME & CFS 2013

I found this report from 2013 which I think is pretty good and accurate (I'm only skimmed thro it though). It is more than the title would suggest.

It's a shame that it would seem nothing resulted from it.

https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM368806.pdf

In addition to the report there were webcasts:
FDA: Development of Safe and Effective Drug Therapies for CFS and ME

http://www.tvworldwide.com/events/fda/130425/

the videos all have big subtitles.

unfortunately the transcript link no longer works.

"
• Many patients can pinpoint a specific time in their life when they contracted the disease. Prior to contracting CFS and ME, many patients were highly productive and successful professionalsor students. They now struggle with even the simplest aspects of day-to-day living.

• Patients struggle daily with their symptoms. Of over 50 symptoms identified, the most frequently mentioned included severe fatigue or exhaustion, impairments in cognitive functioning (e.g., concentrating or processing information), chronic pain, sleep difficulties, blood pressure drops and dizziness, sensitivity to light, sound and temperature and susceptibility to infection. The type, nature and severity of symptoms can vary from patient to patient.

• Post-exertional malaise or PEM (which participants believe is more aptly termed a “crash” or “collapse”) is a severe exacerbation of those cognitive and physical symptoms. A crash can result from even minimal mental or physical exertion, can happen without warning, and can last for days, weeks, months, or even years.

• Patients use or have tried a complex regimen of drug and non-drug therapies to treat their disease and manage their symptoms. Appendix 4 lists the therapies mentioned during this meeting or in comments submitted to the docket. These treatments have been met with varying degrees of effectiveness, and for some, none is effective. These treatments are often associated with bothersome side effects, which can, for example, exacerbate other symptoms of their
disease (e.g., by causing drowsiness).

• CFS and ME takes a devastating toll on the lives of many patients and their families, including loss of careers, decreased quality of family life, social isolation, and feelings of hopelessness.

• Patients are desperate for research and development of treatments that can: (a) better relieve their most significant symptoms and (b) address the underlying cause(s) of their disease."

"
Cognitive symptoms
The most frequently described symptoms were a range of chronic impairments to cognitive functioning.
By a show of hands, many participants appeared to share the perspective expressed by a participant that “[the] most debilitating symptoms fall under the rubric of neurological and cognitive dysfunction.”

Participants described their overall general cognitive dysfunction using terms such as “brain fog,” “impaired executive function,” and “neurocognitive problems.”

They described specific manifestations of their cognitive dysfunction including “confusion,” “disorientation,” “hard to concentrate, can’t focus,” “inability to process information,” “can’t find the right words,” “inability to multi-task,” “slowed processing speed,” “impaired working memory,” “stuttering,” “slow reaction times,” “short-term memory loss,” “expressive dysphagia,” “dyslexia,” “inverting words and numbers when speaking or reading,” and “problems with decision-making.”

A webcast participant described “’mapping dyslexia,’where I lose all sense of how to get from place to place, even with familiar locations.”

"
• Some participants described orthostatic intolerance and symptoms related to blood pressure drops, including dizziness, spatial disorientation, losing balance, fainting, and falling.

• Some participants reported a variety of gastrointestinal symptoms, including irritable bowel syndrome

• A few participants described depression, fear, and anxiety, not as a symptom, but as a consequence of the devastating impact of the disease and its symptoms. As one web participant
stated, “anxiety is NOT a symptom – anxiety is a byproduct [of this disease].”

• Other symptoms mentioned by web and in-person participants included muscle weakness, increased heart rate, immune problems, blurred vision and eyesight problems, seizures, ataxia, fevers and chills, weight loss, tinnitus, ulcers, and rashes."

"Post-exertional malaise or crashes:

"....
it was their collective experience with acute, debilitating PEM, which participants called a “crash” or “collapse,” that received the most attention at the meeting. As one participant said, “The term, ‘malaise’ to the lay-person is a misnomer, it is much more like a collapse.” Participants described a crash as an exacerbation of all symptoms to extreme levels that generally lead to complete incapacitation.

Participants described their complete exhaustion, inability to get out of bed to eat,
intense physical pain (including muscle soreness), incoherency, blacking out and memory loss, and flu-like symptoms (i.e., sore throat, congestive cough, and others).

Participants described how the sudden onset of crashes has put them in dangerous situations, including falling, driving in the wrong direction, unknowingly wandering across a busy street, other instances of extreme confusion in public places, and blacking out."

well worth a read.

 

@Sly Saint -a few things that arose from the publication of the Voice of the Patient
this FDA drug guidance for industry references it https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM388568.pdf
CDC now links to the Voice of the Patient report https://www.cdc.gov/me-cfs/index.html
NIH links to it (from the trans-NIH Working Group site) https://www.nih.gov/mecfs/mecfs-resources
NIH P2P report links to it https://prevention.nih.gov/docs/programs/p2p/mecfs-federal-partners-report.pdf

 

Looking back, perhaps we could have done more to make use of this. I think that at the time many of us failed to realise the potential value of these sots of reports.

 

To my mind this is the sort of information that NICE, the NHS and the DWP should know and make available to medical staff and benefit assessors.

It would also be of use to people newly diagnosed or still looking for a diagnosis.

I have not seen such a detailed and accurate list of symptoms and the effects they have on day to day living and quality of life.

It's not too late to use the info now..............in fact it might be just the right time to put this sort of stuff out there to show that if they had only listened to patients..............

 

That's true. Now to find time to re-read it!

 

this is a table of drug therapies from the report:

 

Interesting drug table.

Several items on that list I would like to try, but insurance won't pay for it, e.g. Valcyte, IVIG, IV saline, Rituximab.

Gotta be careful with the stimulants and antidepressants; they can make things worse. The CDC website now warns:

• "doctors should use caution in prescribing these (antidepressant) medications. Some drugs used to treat depression have other effects that might worsen other ME/CFS symptoms"
• "While stimulants might help improve concentration for some patients with ME/CFS, they might lead to the ‘push-and-crash’ cycle and worsen symptoms."

Interesting that staph vaccine and Ampligen are on the list.

Never heard of angina as a ME/CFS symptom before.

 

the whole series of FDA webinars and reports available thro me-pedia
http://me-pedia.org/wiki/U.S._Food_and_Drug_Administration