Posts Tagged 'Stakeholders Meeting'

Deadline fast approaching!

Drug Development for CFS and ME: Public Workshop to be held April 25/26, 2013

 

The registration deadline is fast approaching. Monday April 8th, 2013 (5pm eastern time) is the deadline for:

registration to attend,

registering to view the webcast,

consideration for participation on Day 1 panels,

public comment summaries.

Register here: http://mecfsmeeting.eventbrite.com/#

You can request to be part of a Day 1 panel discussion (topics below)

Suggestions for those providing comments: For those who wish to provide comments, either as part of the panel or as part of the ‘open discussion’, the following information will be useful:

    • The suggested topics include:
      • Topic 1: Disease Symptoms and Daily Impacts That Matter Most to Patients
      • Topic 2: Patients’ Perspectives on Current Approaches To Treating CFS and M

The questions that the FDA is asking on each of these topics are listed below. You are also given the option to suggest a topic when you register.

    • In addition to the questions that the FDA asked, please provide comments about your lab/test abnormalities – linked to symptoms and dysfunction where possible – and indicate how those abnormalities changed as a result of treatment if known.

If you wish to be considered to present comments part of initial panel discussions, you will need to indicate the topic that you wish to address when you register. Then, you will need to send a brief summary of your response to the topic(s) you selected to ME-CFS-Meeting@fda.hhs.gov. The deadline to send the summary of your comments is April 8.  You will be notified before the meeting if you will be on a panel.

You can request to provide comments on other topics as part of the open comment period at the end of the day

If you want to do this, you will need to register as above. In addition, you will need to send a brief summary of your comments for the open public comment session to ME-CFS-Meeting@fda.hhs.gov. The deadline to submit the summary of your comments is April 8.

Previously we had heard that public comment by proxy would be allowed. This is no longer the case as apparently there are so many community members who want to provide comment in person that FDA can only schedule those providing comment in person. BUT everyone can submit comments to the docket.

Provide comments directly to the FDA now

  • Go here and submit comments now by clicking on the ‘Comment Now!” box in the upper right corner of the page.
  • You can also submit written comments to Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. Include the docket number, which is “FDA-2012-N-0962-0004”.
  • The Docket is open until August 2, 2013.
  • The FDA is encouraging patients to provide answers to the questions asked in the Federal Register. But again, don’t forget to also include information about the biological abnormalities – linked to symptoms where possible – and indicate how those abnormalities changed as a result of treatment if known.

Participate in the FDA meeting remotely

Remember to respond to two different surveys that are collecting information for the meeting.

  • Both surveys are collecting information a) disease symptoms and daily impacts that matter most to patients and b) patient perspectives on current approaches to treating ME/CFS.
  • Dr. Leonard Jason and his team and Dr. Lily Chu have created an online survey that uses checkboxes to collect information on symptoms, impact on daily life, treatments being used and the effect of treatments. For those who live outside the U.S., ignore questions about the U.S. region. The link to Dr. Jason’s and Dr. Chu’s survey is here.
  • The CFIDS Association of America (CAA) has taken the questions from the FDA workshop agenda and turned them into an online questionnaire where you can type answers into text boxes. The link to the survey is here.
  • Both surveys are open to everyone no matter where you live. Your responses will be anonymous and confidential.
  • Please try to answer both questionnaires.
  • Although the surveys may not ask for it specifically, don’t forget to also include comments as above about your lab abnormalities – linked to symptoms and disease dysfunction where possible – and indicate how those abnormalities changed as a result of treatment if known. Examples are given above in #1.

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updated post

 

Please note that OccupyCFS has some clarifying info about public comment http://www.occupycfs.com/2013/03/28/clarity-on-comment/ for the FDA April 25/26th meeting as well as lots of background info about the meeting.

 

The following has been slightly revised to reflect information FDA provided as answers to some questions.

On April 25-26, 2013, the FDA is holding a workshop to discuss how best to facilitate and expedite the development of safe and effective drug therapies to treat signs and symptoms related to CFS and ME. This meeting is expected to attract not only patients and ME/CFS expert clinicians, but also other groups including pharmaceutical companies and the FDA. This is an unprecedented opportunity for us to get the attention and interest of the pharmaceutical industry and to bring a patient perspective to the development and review of drugs for ME/CFS.

 

Day 1 of the workshop will give patients the opportunity to share the symptoms and signs that they experience, their experience with treatments and how this disease affects their quality of life. This is being conducted as part of the Patient-Focused Drug Development Initiative, an FDA effort to incorporate the patient experience into the drug approval process by better understanding the patients’ perspective on the severity of their disease and the effectiveness of available treatments. ME/CFS has been chosen as one of the 20 diseases for which such a workshop will be conducted and will be the first of the 20 to go! For a disease like ME/CFS whose impact is poorly understood by those outside the community, this first day will provide the critical insights needed for discussions on the second day of the FDA workshop but just as importantly will also ensure that the drug approval process better incorporates the patient perspective going forward.

 

Day 2 of the workshop will include a scientific workshop on how to best facilitate and expedite the development of safe and effective drug therapies for the signs and symptoms related to ME/CFS. Presentations will include:

  • Lessons learned from previous studies
  • The role of drug repurposing
  • Pathways to expediting drug therapies
  • Appropriate clinical trial design in CFS and ME
  • Outcome measures to assess efficacy
  • Potential valid endpoint measurements of symptom improvement.

 

The Importance of both Symptoms and Biological Abnormalities

Obviously, it is critical that FDA and the pharmaceutical companies understand the symptoms you experience and the impact of those symptoms on your quality of life. But it is just as essential that they also understand the biological abnormalities that are associated with ME/CFS – biological abnormalities that are already being measured through lab tests and other testing. Some examples include:

  • Neurological impairment seen in symptoms like sleep dysfunction, headaches and sensory disturbances and that can be measured in lab tests like SPECT scans or MRI.
  • Cognitive dysfunction seen in symptoms like impaired memory and processing speed and that can be measured through various cognitive performance tests.
  • Immunological dysfunction seen in cytokines abnormalities, high viral titers, RNASE-L abnormality and low natural killer cells.
  • Autonomic dysfunction and orthostatic intolerance causes symptoms like dizziness, racing heart and feeling sick when stand for long periods and that can be measured by tests like tilt table tests, heart rate and blood pressure monitoring and blood volume.
  • Post exertional malaise and low anaerobic threshold that cause a relapse after mental or physical exertion and documented by cardiopulmonary exercise testing.

By understanding both the symptoms and the related biological pathologies and associated abnormalities, pharmaceutical companies will be in a better position to identify opportunities to subgroup patients and potentially repurpose existing drugs that might treat ME/CFS. This is especially critical in a disease like ME/CFS which is too often described simply in terms of “fatigue not improved by rest”, is assumed to be due to depression, deconditioning or is thought to be simply another name for chronic fatigue.

 

To ensure that the pharmaceutical companies understand this biology, a group of ME/CFS organizations and advocates submitted a joint letter to the FDA to ask for assurance that a brief overview of the biology and abnormalities of ME/CFS will be summarized right at the beginning of the meeting. The letter that was sent can be found athttps://dl.dropbox.com/u/89158245/LetterFDAWorkshopMar18.pdf

 

Take Action

So what can you do to make sure the FDA and the pharmaceutical companies understand how ME/CFS has affected you and what you have experienced with treatments? There are a number of opportunities for you to participate and to provide comments.

 

  1. Register to attend the meeting and optionally provide comments

Here’s what you need to do:

  • To attend in person, register first-come, first-serve at http://mecfsmeeting.eventbrite.comby April 8. You will be asked to provide your name and business, organization, or personal affiliation as applies (e.g., industry, government, patient). When you register, you will need to indicate that you wish to provide comments as outlined next.

 

  • There are three opportunities to present comments at the meeting:
    • You can request to be part of an initial panel discussion on the topics listed below.

If you wish to be considered to present comments part of initial panel discussions, you will need to indicate the topic that you wish to address when you register. Then, you will need to send a brief summary of your response to the topic(s) you selected to ME-CFS-Meeting@fda.hhs.gov. The deadline to send the summary of your comments is April 8.  You will be notified before the meeting if you will be on a panel.

 

    • You can participate in open moderated discussion following the panel discussions on the topics listed below.

You do not need to register to provide comments or have prepared testimony for this part of the discussion. A moderator will recognize participants to comment when they raise their hands.

 

    • You can request to provide comments on other topics as part of the open comment period at the end of the day

If you want to do this, you will need to register as above. In addition, you will need to send a brief summary of your comments for the open public comment session to ME-CFS-Meeting@fda.hhs.gov. The deadline to submit the summary of your comments is April 8.

 

  • Suggestions for those providing comments: For those who wish to provide comments, either as part of the panel or as part of the ‘open discussion’, the following information will be useful:
    • The suggested topics include:
      • Topic 1: Disease Symptoms and Daily Impacts That Matter Most to Patients
      • Topic 2: Patients’ Perspectives on Current Approaches To Treating CFS and M

The questions that the FDA is asking on each of these topics are listed below. You are also given the option to suggest a topic when you register.

    • In addition to the questions that the FDA asked, please provide comments about your lab/test abnormalities – linked to symptoms and dysfunction where possible – and indicate how those abnormalities changed as a result of treatment if known. Examples include:
      • Abnormal VO2Max as a result of PENE/PEM
      • Abnormal tilt test as indicative of orthostatic intolerance
      • High inflammatory cytokines indicative of immunological issues
      • Cognitive issues seen in impaired working memory and slow processing speed tested by cognitive performance testing
    • Other suggestions:
      • Comments should be concise and to the point. Avoid jargon. Speak about your personal experience and how this disease has affected you.
      • For some people, the term biomarker may imply that the biomarker has been “validated”. It is better to use the term abnormalities or test abnormalities to avoid this confusion.
      • Given that the time for comments may need to be shortened, it would be good for you to have both a 1-2 minute version and a 3-4 minute version.
  • Provide comments by proxy: If you cannot attend but wish to provide comments, you can have a proxy provide them for you. You will need to register to do this.

 

  1. Participate in the FDA meeting remotely
  1. Respond to two different surveys that are collecting information for the meeting.
  • Both surveys are collecting information a) disease symptoms and daily impacts that matter most to patients and b) patient perspectives on current approaches to treating ME/CFS.
  • Dr. Leonard Jason and his team and Dr. Lily Chu have created an online survey that uses checkboxes to collect information on symptoms, impact on daily life, treatments being used and the effect of treatments. For those who live outside the U.S., ignore questions about the U.S. region. The link to Dr. Jason’s and Dr. Chu’s survey is here.
  • The CFIDS Association of America (CAA) has taken the questions from the FDA workshop agenda and turned them into an online questionnaire where you can type answers into text boxes. The link to the survey is here.
  • Both surveys are open to everyone no matter where you live. Your responses will be anonymous and confidential.
  • Please try to answer both questionnaires.
  • Although the surveys may not ask for it specifically, don’t forget to also include comments as above about your lab abnormalities – linked to symptoms and disease dysfunction where possible – and indicate how those abnormalities changed as a result of treatment if known. Examples are given above in #1.

 

  1. Provide comments directly to the FDA now
  • Go here and submit comments now by clicking on the ‘Comment Now!” box in the upper right corner of the page.
  • You can also submit written comments to Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. Include the docket number, which is FDA-2012-N-0962-0004”.
  • The Docket is open until August 2, 2013.
  • The FDA is encouraging patients to provide answers to the questions asked in the Federal Register. But again, don’t forget to also include information about the biological abnormalities – linked to symptoms where possible – and indicate how those abnormalities changed as a result of treatment if known. Examples are given in above.

 

Get more information

You can get additional information from the following sources:

  • The FDA maintains this webpage that lists information on its efforts regarding ME/CFS.
  • The Federal Register Notice for the April 25-26 FDA workshop can be found here.
  • The FDA has also provided a set of FAQs here about the meeting, which provide additional information.
  • A description of the Patient-Focused Drug Development Initiative can be found here.

 

 

 

Details on the questions that the FDA would like asked on the two topics.

Remember to also provide information on the biological abnormalities associated with these symptoms and the known neurological, immunological and energy production dysfunction and how treatments have affected these biological abnormalities. Examples are given above in #1.

 

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.)
    1. How does the condition affect your daily life on the best days and worst days?
    2. What changes have you had to make in your life because of your condition?

 

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.)
    1. What specific symptoms do your treatments address?
    2. 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?
    1. Have these treatments improved your daily life (for example, improving your ability to do specific activities)? Please explain.
    2. How well have these treatments worked for you as your condition has changed over time?
    3. What are the most significant downsides of these treatments (for example, specific side effects)?

 

The FDA Drug Development for CFS and ME public workshop April 25 and 26, 2013

A Federal Register notice about the meeting will be posted on Monday March 11 (links at bottom of post). Here are some of the details.

 

To attend in person go to http://mecfsmeeting.eventbrite.com to sign up. You must sign up by 5pm EST Monday, 8 April 2013.

Required information

your name (first and last)

email address

affiliation (patient, advocate, name of organization, etc.).

 

The next part of the form asks if you (are a patient/patient representative and)want to be considered to present comments as part of the April 25th panel discussion on specified meeting topics.

 

If you indicated that you wanted to be considered, you are then asked which of the two discussion topics you want to address.

The topics are:

Disease Symptoms and Daily Impacts That Matter Most to Patients

Patients’ Perspectives on Current Approaches To Treating CFS and ME

 

Each topic will start with a panel discussion of patients and then there will be a facilitated discussion that all participants will be invited to take part in.

 

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?

 

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)?

 

It seems there will be additional public comment time (this will depend on how many requests there are). Probably the most helpful comments will be related to facilitation of and expediting development of safe and efficacious drug therapies for ME and “how to identify sound, quantitative outcome measures that can be used in clinical trials to determine whether disease symptoms improve with specific drug interventions”.

 

For those who cannot attend in person – the meeting will be webcast ( http://mecfsmeeting.eventbrite.com) and you can submit comment via www.regulations.gov (as of 9 March 2013 the docket for this meeting does not seem to be open yet )

 

In addition to the confirmation page on eventbrite, you will receive a confirmation email from United States Food and Drug Administration via <orders@eventbrite.com>. – Your confirmation email will include an order number (also on your confirmation page).

 

(The agenda isn’t out yet…)

 

Have you signed up to attend?

Which patient discussion panel are you interested in being on? Why?

Are you providing public comment?

Which topic will provide comment on? Why?

Will you watch the webcast?

Will you submit comment to the docket?

Which topic will provide comment on? Why?

 

Here is the Federal Register notice for “Drug Development for Chronic Fatigue Syndrome and Myalgic Encephalomyelitis; Public Workshop” the FDA meeting that will be held 25/26 April 2013

https://www.federalregister.gov/articles/2013/03/11/2013-05562/drug-development-for-chronic-fatigue-syndrome-and-myalgic-encephalomyelitis-public-workshop

this is the link to the pdf of the notice:

http://www.gpo.gov/fdsys/pkg/FR-2013-03-11/pdf/2013-05562.pdf

 

 

Public Comments due Thursday, December 6th 2012 for FDA Advisory Committee on Ampligen CALL TO ACTION (updated)

Thursday, 6 December, 2012 is the deadline to submit public comment for the 20 Dec 2012 Arthritis Advisory Committee Meeting!

(2 Dec 2012 – CORRECT EMAIL ADDRESS:   AAC@fda.hhs.gov)

Please spread the word about this – there are only a few days until the deadline. Anyone, anywhere can participate!

If anyone has trouble submitting their comments please email us (see below) – we will do our best to help.

Why is it important for you to send your comments?

For the first time ever, ME/CFS has the chance to get a treatment approved.  Whether you have been on Ampligen or not, whether you wanted to try it or not – once one drug gets approved other drugs will come along and so will additional research dollars.  It will also legitimize the disease in the minds of the medical community and the world.

Why is this public comment just about Ampligen?

Because this particular meeting is part of the process to approve a drug. 

Send in your comments to let the advisory committee know what it is like to have ME/CFS and to tell them that treatment is needed NOW.  

Your comments do not need to be lengthy. Feel free to use the template below. (The template uses CFS because that is how the FDA website http://www.fda.gov/AdvisoryCommittees/Calendar/ucm324972.htm refers to this wretched illness. Using CFS will probably make it easier for those on the committee to follow.)

(The advocacy community will continue to update you about additional significant meetings coming up – including the Stakeholder meeting in the spring, as well as about issues dealing with CDC and NIH…) 

Right now we need to let the Advisory Committee hear us on this issue.

Send in your public comment by THURSDAY DECEMBER 6th, 2012

Send your comments to: AAC@fda.hhs.gov   (this is the correct email address)

Subject line:  Treatment for Chronic Fatigue Syndrome- Ampligen

To The Advisory Committee Reviewing Ampligen:

My name is ___________________…. .

I have had CFS for more than ___ years. 

Before I became ill, I ______________________……. 

My life because of CFS _______________________________________________…….. 

The reality is that CFS is a horribly devastating illness that effects every moment of my life. 

CFS patients need treatments.

We have seen patients do well on Ampligen. We have seen patients’ quality of life improve as a result of Ampligen.

Help us have access to treatments.

Please give the CFS community hope of getting our lives back.

Sincerely,

…………………………

send a copy of your email to:  (SpeakUpAboutMe at gmail dot com)

Remember to send in your public comment by THURSDAY DECEMBER 6th, 2012

Transcript of FDA teleconference now posted

The transcript of the teleconference call (Sept 13th) is here

http://www.fda.gov/downloads/Drugs/NewsEvents/UCM320310.pdf

Please remember that FDA is accepting questions and comments in the docket http://www.regulations.gov/#!searchResults;rpp=25;po=0;s=FDA-2012-N-0962

Questions and comment for this docket must be submitted before Sept 30th, 2012.

 

 

 

FDA ME/CFS page:

http://www.fda.gov/Drugs/NewsEvents/ucm319188.htm

Reminder

Remember the email campaign to the FDA begun last spring?

And later, we sent more emails to the FDA…

And we reminded you again to send emails and to have friends and family send emails also….

And in early July we were really glad to let everyone know that our efforts had been successful and that the FDA had agreed to a Stakeholder Meeting with the ME/CFS Community.

 

Well, soon, we start meeting with the FDA.

The first meeting is a teleconference on September 13th, 2012 when the FDA will host a 90 minute teleconference to plan the ME/CFS Community and FDA Stakeholder Meeting.(The call is limited to 50 phone lines.)

This call is a planning meeting for the FDA Stakeholder Meeting that Dr. Janet Woodcock has agreed to.

(The date and location of the actual ME/CFS and FDA Stakeholder Meeting are not yet known.)

The goal of this actual FDA Stakeholder meeting is the acceleration of treatment and expanded access to treatments.

 

 

More information about how this came about can be found here at Phoenix Rising’s FDA Resource Center:       http://phoenixrising.me/federal-drug-administration

FDA Stakeholder Meeting

Remember all the requests we made asking that people send emails urging the FDA to meet with the ME/CFS community?

Thanks to everyone who supported this effort we are pleased to tell you (and we quote from Phoenix Rising):


“We are very happy to announce that during a conference call, Dr. Janet Woodcock, Director of Center for Drug Evaluation and Research at the FDA, made a commitment to the ME/CFS community to hold a Stakeholder Meeting. “

 http://phoenixrising.me/

PLEASE take a few moments to pat yourselves on the back for helping our community get this commitment to a Stakeholder Meeting!

THANK YOU !!