Posts Tagged 'FDA'

link to Miriam Tucker’s article Chronic Fatigue Syndrome: Wrong Name, Real Illness
“Chronic Fatigue Syndrome: Wrong Name, Real Illness
Miriam E. Tucker
January 08, 2015


Sufferers of what has been called chronic fatigue syndrome (CFS) are challenging patients, presenting with complaints of postexertional
malaise, persistent flulike symptoms, unrefreshing sleep, “brain fog,” and often a long list of other symptoms that don’t seem to fit any
recognizable pattern. Some appear ill, but many don’t. And the routine laboratory tests typically come back negative. ….”


FDA Webinar, chance for Q&A and comments April 23, 2014

FDA is hosting a webinar about the draft guidance document “Guidance for Industry Chronic Fatigue Syndrome/ Myalgic Encephalomyelitis: Developing Drug Products for Treatment”.

Note: the webinar will be close-captioned and there questions/comments can be submitted in real-time using a chat window. (It is safe to assume that personal information should not be submitted as this would then be available for all to see onscreen.)

Public comment on the draft guidance should also be submitted to the docket DOCKET NUMBER: FDA–2014–D–0264!docketDetail;D=FDA-2014-D-0264 – as it then becomes part of the public record.

FDA’s Guidance Webinar series aims to foster collaboration and transparency in the development of guidance documents through direct outreach to affected stakeholders.” (

FDA Webinar: Guidance for Industry Chronic Fatigue Syndrome/ Myalgic Encephalomyelitis: Developing Drug Products for Treatment – April 23, 2014

The Office of Medical Policy (OMP) in CDER presents another in a series of webinars on 60-day guidances for industry on Wednesday, April 23, 2014 from 1PM – 2PM EDT. The topic is “CHRONIC FATIGUE SYNDROME”.

Guidance for Industry Chronic Fatigue Syndrome/ Myalgic Encephalomyelitis: Developing Drug Products for Treatment



COMMENTS [DUE] DATE:  05/12/2014

DOCKET NUMBER: FDA–2014–D–0264!docketDetail;D=FDA-2014-D-0264


Janet Maynard, MD and others

Medical Officer

Division of Pulmonary, Allergy, and Rheumatology Products

Office of New Drugs


SUMMARY: This guidance is intended to assist sponsors in the development of drug products for the treatment of chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME). This guidance focuses on specific drug development and trial design issues that are unique to the study of CFS/ME and on the FDA’s current thinking on how effective treatments can be developed for CFS/ME. The points discussed in this guidance may not be applicable to all drug products. The FDA encourages sponsors to design clinical programs that fit their particular needs and to discuss their planned approach with the Division of Pulmonary, Allergy, and Rheumatology Products (DPARP).

For questions concerning the webinar, please contact Marsha Holloman (301-796-0731)

Webinar information on FDA’s Web site:


Guidance Webinar Online-Access Instructions:

To access this webinar, follow the link provided below. Audio will broadcast from your computer speakers.

After following the link, enter as a guest and provide your FULL NAME and organization (i.e. “John Smith – FDA/CBER”). The host will then allow you to enter. If you experience technical difficulties email for assistance. Closed captioning will be provided. Questions/Comments can be submitted live via a Q/A chat window.

Access link:

Public comment 11 March 2014

It often feels as though we (patient community) speak a different language than Health and Human Services (HHS) agencies and that this results in ongoing communication problems between us. This is complicated by the ongoing lack of openness and transparency by HHS.

At the FDA meeting on April 25th 2013, (, time was spent querying participants and restating things to make sure listeners understood the intent of the speakers. As a result, in its Voice of the Patient report (, FDA was able to characterize the patient experience in a way that no other government agency has done before.

After that meeting, at least one noted clinician/researcher restructured the questions posed to patients and is looking for additional ways to ensure more accurate understanding of patient experiences, symptoms, etc.

It seems that Dr. Unger has begun to understand how threatened patients feel by the term “malaise”. To laypeople the connotation is “feeling out of sorts”. This means that the phrase “post-exertional malaise” gives very little indication of the intensity, severity and extent of the serious problems that post-exertional malaise produces. How did Dr. Unger begin to understand how seriously and negatively the term “malaise” impacts us? It started during a conversation between advocates and Dr. Unger. During that conversation, there were opportunities to clarify points and answer questions.

During public comment we have the opportunity to provide input but there is no way to know if you have understood what we say. And during CFSAC meetings, we have no way of asking clarifying questions to ensure that we understood what you have said. Simply put, as currently structured, there is no opportunity on our part or yours for clarification to ensure that we understand each other. And given that some agencies do not respond to questions submitted by advocates between meetings, or instead respond by reiterating things we are already well aware of, we have no opportunities outside of CFSAC to get answers.

We endure this illness, minute by minute, week by week, decade by decade and as a result, we have a wealth of knowledge and experience.

If you are truly interested in this illness and in helping us, surely there are questions you have, that we can help provide answers to. But we currently have no way of doing so.

We must find ways to ensure substantive discussion and accurate understanding on everyone’s part.

Matthew Lopez-Majano


Goal 4: Increase Efficiency, Transparency, and Accountability of HHS Programs


HHS’s Open Government efforts will break new ground in enabling the public to give feedback to HHS programs. HHS can help stakeholders contribute knowledge and experience to help it do jobs better, and HHS can support new kinds of collaborative teamwork that will deliver better results for our citizens.  HHS will move forward toward new strategies, new tools, and a new culture of public participation and collaboration in its affairs. “ (emphasis added)

Summary of April 26, 2013 FDA meeting (day 2)

Recently FDA released a summary ( of the April 26, 2013 FDA Workshop on Drug Development for Chronic Fatigue Syndrome (CFS) and Myalgic Encephalomyelitis (ME). You may recall that this was the second day of a 1.5 day meeting and on this day, there were discussions about identifying quantitative outcome measures and determining which treatments produce improvements.

Remember that FDA is currently producing a guidance document for CFS and ME drugs that will provide advice for pharmaceutical companies and industry to expedite research for treatments. Our input will be needed on this. Note – typically when a draft guidance is released there is a 60 day comment period.

Additional information about the meeting and links to transcripts etc. can be found here:

FDA CFS and ME Teleconference – October 16, 2013


In case you have not seen it, FDA has produced a report based on the April 25th meeting. You can download it or read it here Based on the input received at the meeting and from material submitted to the docket, their characterization of ME is quite good.

As a followup to the April FDA meeting, FDA is hosting a teleconference on Wednesday Oct 16th fro 1:30-3pm (eastern time). Details below.

On October 16 from 1:30 PM – 3:00 PM EST, FDA will hold a teleconference that is open to the public. FDA will provide introductory and background remarks then the teleconference will be open for public comment on the following three topics:

1.            Feedback from the CFS and ME public workshop.

2.            Current projects related to drug development in CFS and ME.

3.            Potential methods to catalyze CFS and ME drug development.

Registration (Closes October 11, 2013):

FDA CFS and ME Activities Website:

Please contact Randi Clark ( or Mary Gross ( if you have any questions.

There’s still time to submit comments to FDA docket on Drug Development for ME and CFS

Did you know, as of July 17th 2013, only 193 comments have been submitted to the FDA docket on Drug Development for ME and CFS?

Comments (from any country) can still be submitted (!submitComment;D=FDA-2012-N-0962-0004 ) until August 2, 2013.

Remember you can attach a document if your comments are more than 2000 characters long and/or you can provide your comment via video with a YouTube link.

FDA suggested two topics for comment. Consider including medicines you have tried – even if off-label – and their effects (positive, negative, no effect,side effects, intolerable side effects, etc.).


FDA Suggested Topic 1: Disease Symptoms and Daily Impacts That Matter Most to Patients

1. 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.)

2. 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, such as sleeping through the night.)

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?

FDA Suggested Topic 2: Patients’ Perspectives on Current Approaches To Treating CFS and ME

1. What treatments are you currently using to help treat your condition or its symptoms? (Examples may include FDA-approved medicines, over-the-counter products, and other therapies, including non-drug therapies such as activity limitations.)

a. What specific symptoms do your treatments address?

b. How has your treatment regimen changed over time and why?

2. How well does your current treatment regimen treat the most significant symptoms of your disease?

a. Have these treatments improved your daily life (for example, improving your ability to do specific activities)? Please explain.

b. How well have these treatments worked for you as your condition has changed over time?

c. What are the most significant downsides of these treatments (for example, specific side effects)?

Transcript Day I April 25, 2013

Transcript Day II April 26, 2013


CFSAC May 2013 videos have all been posted now

Twenty-six days elapsed between the end of the meeting and the posting of these videos. (Typically the time frame has been 4-6 weeks….)

Day 1 May 22, 2013

Welcome & Call to Order – Day 1 (CFSAC Spring 2013)

May 22, 2013, 9:00 – 10:30 AM.

Public Comment on Day 1 (CFSAC Spring 2013)

Chronic Fatigue Syndrome Advisory Committee (CFSAC) Meeting, Day 1, May 22, 2013, 11:45 AM – 1:00 PM. (I think the time is incorrect here. I think the start time should be 10:45AM)

Approve Prioritized Recommendations List – Day 1 (CFSAC Spring 2013)

May 22, 2013, 1:00 – 1:30 PM.

Determination of Medicare Coverage of Test and Treatments – Day 1 (CFSAC Spring 2013)

May 22, 2013, 1:30 – 2:30 PM.

Public Question & Answer on Day 1 (CFSAC Spring 2013)

2:45 – 3:15 PM. Public Q&A.

Committee Discussion and Plans for Day 2 (CFSAC Spring 2013)

3:15 — 5 PM.

Day 2 May 23, 2013

Opening Remarks on Day 2 (CFSAC Spring 2013)

9:00 – 10:15 AM.

Health Insurance Marketplace – Day 2 (CFSAC Spring 2013)

10:15 – 11:00 AM

Public Comment on Day 2 (CFSAC Spring 2013)

11:15 AM – 12:15 PM

Public Question and Answer on Day 2 (CFSAC Spring 2013)

1:30 – 3:00 PM.

Committee Discussion on Day 2 (CFSAC Spring 2013)

3:00 – 3:30 PM.




Committee Discussion (Part #2) on Day 2 (CFSAC Spring 2013)

3:30 – 5:00 PM