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FDA Workshop on Drug Development for CFS and ME

Ember

Senior Member
Messages
2,115
2/20/2013 Update
The public workshop will be held on the afternoon of April 25 and all day, April 26, at the Bethesda Marriott, 5151 Pooks Hill Road, Bethesda, MD 20814, Phone 301-897-9400.

Beginning at 1:00 pm on April 25, FDA will gather patients’ perspectives on CFS and ME through a facilitated group discussion focusing on two main topics:
(1) disease symptoms and daily impacts that matter most to patients; and
(2) patients’ perspectives on current approaches to treating CFS and ME.

The second day will include a scientific discussion with patients, clinicians, researchers and government experts focusing on how to identify quantitative outcome measures to determine if disease symptoms improve with specific drug interventions.

Additional information will be posted soon (see link below), including the Federal Register Notice that will describe the meeting format and provide details about how to register to attend the meeting.
 

Ember

Senior Member
Messages
2,115
The FDA “Alternative Approval Pathway...Intended to Address Unmet Medical Need” proposes that a drug's “safety and effectiveness would be studied in a smaller subpopulation of patients with more serious manifestations of a condition. Such a pathway could involve smaller and more rapid clinical trials than would occur if the drug were studied in a broader group of patients with a wide range of clinical manifestations.”

How will the FDA distinguish in this workshop between CFS and the smaller subpopulation of ME patients with more serious manifestations of the condition?
 

SOC

Senior Member
Messages
7,849
How will the FDA distinguish in this workshop between CFS and the smaller subpopulation of ME patients with more serious manifestations of the condition?

Unfortunately, there is unlikely to be any distinction because ME is not a recognized illness at all in the US medical system. It simply doesn't exist. In the US, for anything official, you have CFS. Period. Annoying as hell, but that's where it is right now.
 

Ember

Senior Member
Messages
2,115
Unfortunately, there is unlikely to be any distinction because ME is not a recognized illness at all in the US medical system. It simply doesn't exist. In the US, for anything official, you have CFS. Period. Annoying as hell, but that's where it is right now.
Clearly the FDA is aware of the lumping or splitting issue, though, as is shown by the use of both CFS and ME in the workshop title and description. During the Teleconference between FDA and Patients/Patient Advocates on September 13, Dr. Sandra Kweder said:
I want to mention that we have heard from some patients who object to us using the term ME/CFS or CFS/ME in the screening of these. I want to make it clear for our purposes relating to drug development, drugs are developed based on quantitative measures that improve symptoms.

We believe that the term CFS/ME is a frame of reference that is intended to be inclusive, but doesn't make any judgment on the cause of different symptom complexes and whether or not the symptoms are best lumped into one or split into different diseases.

The FDA knows, of course, that an inclusive frame of reference doesn't work if you're trying to identify a smaller subpopulation of patients with more serious manifestations of a condition.
 

SOC

Senior Member
Messages
7,849
Clearly the FDA is aware of the lumping or splitting issue, though, as is shown by the use of both CFS and ME in the workshop title and description. During the Teleconference between FDA and Patients/Patient Advocates on September 13, Dr. Sandra Kweder said:


The FDA knows, of course, that an inclusive frame of reference doesn't work if you're trying to identify a smaller subpopulation of patients with more serious manifestations of a condition.

Well, I certainly won't argue that there's a massive amount of ignorance and lack of basic common sense in the way the medical authorities address ME/CFS in the US. :)
 

Ember

Senior Member
Messages
2,115
Well, I certainly won't argue that there's a massive amount of ignorance and lack of basic common sense in the way the medical authorities address ME/CFS in the US. :)
In How to Survive a Plague, AIDS activist Peter Staley is shown having to take the research initiative:
I'd just like to say that the reasons that I can't get any of these drugs is 'cause for nine years now the leadership of this country has failed to come up with a plan of action. They've failed to come up with a plan of research, a national research agenda. People with AIDS and their advocates have finally done this for them. This is it: (A National AIDS Treatment Research Agenda). This is the plan we're presenting. We need the government to read this plan. We need them to work with us. If they want to change it a little, we'll talk to them. But I want them to adopt it. I want them to get started on it. I want them to save our lives. Thank you.
When drugs weren't approved by the FDA, public opinion changed:
The idea that there were people who wanted drugs and the FDA was refusing to give them these drugs was startling. It was something that people could identify with and did identify with and began this really turning of the ship of public opinion about what AIDS activism was and what the government's response should be to them.

The parallels with our situation are striking, and the “inside/outside” strategy makes a lot of sense.
 

Shell

Senior Member
Messages
477
Location
England
I still think the biggest barrier to us getting attention is that 'They' insist we aren't dying of this. The wazzak (I refuse to call him a doctor) who dx me went on and on about how no one dies of ME - they die of something else.
It took my brain a while to unwrap that.
People don't die of AIDS or Flu either. Nearly every person who died in the 1918 fle pandemic died of pneumonia, and AIDS patients die of cancer or other opportunistic diseases.
Sadly I think we need to highlight those who have died of opportunistic infection or heart disease caused by ME.

We somehow need to show that like flu or HIV whatever causes ME is a door opener to all the other crap we are forced to live with or die of.

The other major disadvantage that we have over those who are hiv+ is that we are ill from the get-go and many of us are very ill indeed. HIV+ people tend to be quite well for the beginning and therefore have the energy for a fight.
 

Purple

Bundle of purpliness
Messages
489
The other major disadvantage that we have over those who are hiv+ is that we are ill from the get-go and many of us are very ill indeed. HIV+ people tend to be quite well for the beginning and therefore have the energy for a fight.

This point - that we can become severely ill virtually overnight and stay that way for many years or even decades - is in my opinion something to be highlighted in advocacy.
 

taniaaust1

Senior Member
Messages
13,054
Location
Sth Australia
I hope the illest of our communities can get representives to go to this meeting in support of them and their needs.

I too think OI drugs is very important to have something approved for ME/CFS patients. A few of the prescription drugs which really have helped me re the ME are Clonazepam, Temazapam for sleep (not taken at same time as other benzo), Clonidine (can help multiple ME symptoms) and Florinef.
 

Seven7

Seven
Messages
3,444
Location
USA
Drugs that have helped me the most: Midodrine, florinef, inosine (equivelant to imunovir that is not FDA approved) Sleep Meds: in my case trazadone.

I have an extremely good testimony how I went from bed ridden to work on 3 days. If somebody is going to the meeting and want to read my statement I am willing to write it. Just PM me.

(And for those wondering I DO HAVE ME/CFS, been tested by Klimas group, have extremely low NK cell, Low T cells, High B cells and inflammations cytokines. parvo reactivation, casaxie and hh6v). AT is 115bpm.
 

Ember

Senior Member
Messages
2,115
I hope the illest of our communities can get representives to go to this meeting in support of them and their needs.
For me, the ME symptom and daily impact that matters most is post-exertional neuroimmune exhaustion (PENE), “a pathological inability to produce sufficient energy on demand with prominent symptoms primarily in the neuroimmune regions.” Pacing helps; GET/CBT doesn't.

I would welcome any drug that helps to restore my rest/activity balance, such that one hour of rest would reliably support two hours of normal activity. So far, the test-retest protocol provides our best quantitative outcome measure.
 

Jarod

Senior Member
Messages
784
Location
planet earth
Since when did the FDA get interested in developing drugs for CFS? Do they even know what it is? Are they doing the same for autism and other similar diseases I wonder? Do they have an increased budget for such efforts and will there be any project management charts to track results/accountability?

Do they realize that the best pharmaceutical treatments for CFS have gone up by 500-1000% in some cases? For instance: Stromectol and Invermectin which are anti-parasitic medicines have increased. Invermectin has gone from like $150 for a couple week dose to $1400 suddenly.

Doxycycline price has shot through the roof and is hard to get. Online price has tripled, but in the US pharmacies has gone up much more and is available in limited quanities.

Injectable Hydroxyb12 and methyl B12 are extremely hard to get in the US. The price has increased significantly. I couldn't get methyl b12 injectable at all. Hydroxy b12 has gotten expensive and is sold in useless quanities in US pharmacies.

------------
Online pharmacies can be an option for aquiring affordable meds, but the shipping companies seem to be under attack for dealing with online pharmacies. My linked article says for pain killers, but then goes on to say.......
UPS said it was cooperating with a DEA investigation involving the "transportation of packages on behalf of online pharmacies that may have operated illegally."

Not sure if that bodes well for online prescriptions.
 

Jarod

Senior Member
Messages
784
Location
planet earth
Lately the FDA seems to be interested in global regulation activies.

http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm339630.htm

In order to meet the challenges of today’s global marketplace, the FDA is transforming from a predominantly domestically focused agency to one that is fully prepared to help ensure product safety and quality within a globalized world. In this context, many of the IOM recommendations support actions and efforts already underway at the FDA, including advancing technology, strengthening global regulatory capacity, strengthening surveillance, developing science-based standards and engaging in global dialogue
 

Ember

Senior Member
Messages
2,115
Unfortunately, there is unlikely to be any distinction because ME is not a recognized illness at all in the US medical system. It simply doesn't exist. In the US, for anything official, you have CFS. Period. Annoying as hell, but that's where it is right now.
ME is listed in the ICD-10-CM and it's part of the FDA lexicon. So it must, then, have been deemed to exist. Could it be considered an orphan condition? On the ME and CFS Stakeholder Teleconference in September, Dr. Sandra Kweder told us that the FDA has an “office of orphan products:”
There is absolutely a trend in the industry to look at smaller populations of patients with orphan conditions before drugs are approved. Again whether or not it applies in these conditions you'd have to be able to articulate what that sub-population is. But I know that the agency, you know, our office of orphan products is open-minded and willing to have those conversations with groups that are interested in doing that.
 

SOC

Senior Member
Messages
7,849
ME is listed in the ICD-10-CM and it's part of the FDA lexicon. So it must, then, have been deemed to exist. Could it be considered an orphan condition? On the ME and CFS Stakeholder Teleconference in September, Dr. Sandra Kweder told us that the FDA has an “office of orphan products:”

ICD-10-CM is not currently valid and is still subject to change. We're still operating under ICD-9-CM which doesn't have a code for myalgic encephalomyelitis.
Although this release of ICD-10-CM is now available for -public viewing, the codes in ICD-10-CM are not currently valid for any purpose or use. As noted above, the effective implementation date for ICD-10-CM (and ICD-10-PCS) is October 1, 2014. Updates to this version of ICD-10-CM are anticipated prior to its implementation.

We can hope for future distinctions, but at present they do not exist in the US. At best we get ME/CFS as the broad definition of a single illness, not the distinct ME entity that exists in the WHO classification. Even in the ICD-10-CM ME is coded under Postviral Fatigue Syndrome (not neurological disorders as it is in the WHO-generated ICD documents) and is given as equivalent to CFS.
The etiology of CFS may be viral or immunologic. Neurasthenia and fibromyalgia may represent related disorders. Also known as myalgic encephalomyelitis.