Posts Tagged 'disabling'

Dear HealthCare Professional

We have seen all sorts of comments by healthcare professionals about patients with ME (myalgic encephalomyelitis) and what is “really” wrong with them.

These are just a few comments (and what is implied by those comments).

They just don’t want to work (they want to be on disability, they stand to gain from disability),

are bored (so they spend their time imagining symptoms),

can’t cope with everyday stress (they need psychological help),

don’t exercise (and are deconditioned),

are fat (and deconditioned),

don’t want to be responsible for themselves (they want to go on disability and live an easy life),

want stimulants, narcotics, etc (they are drugseekers),

want disabled parking tags (because disability is a perk).

 

Dear Healthcare Professionals,

Comments about patients with ME (myalgic encephalomyelitis) such as those made by healthcare professionals like you are painful. I hope this post helps you understand some of our pain and frustration.

My two young adult sons have ME which is a complex and challenging disease.

Both of my sons meet the IOM criteria (this link is to The Clinician’s Guide and you can access the full report ). They also meet the CCC criteria for ME.

One son suddenly became sick since he was 12 years old. He is in his early 20s now.

My other son gradually sick about 15 months later. He too is in his 20s.

Neither of my sons are/were lazy.

A few specifics about my sons.

One son taught himself to multiply before he went to kindergarten.

The other was reading middle school level books at age 5.

They were lively, curious, and fully engaged with life, family and friends.

At the time they got sick they were competitive swimmers, swimming upwards of 20 miles a week.

The sudden onset of one son’s illness rendered him housebound, unable to do schoolwork, inexplicably unable to do mental math that had been effortless for him at age 5, unable to socialize, unable to leave home for things other than medically related appointments. In other words – no fun.

My other son had a gradual onset and he too is housebound and partially bedbound, unable to retain what he reads, unable to socialize, unable to leave home for anything other than medically related appointments. In other words – no fun.

In the past 10+ years since they got sick my sons have diligently done everything suggested by healthcare professionals (including various specialists) – to little avail. Most medications we have tried have produced no benefit and few produce enough benefit to endure their adverse effects.

My sons have not been able to learn to drive – frequent, unexpected syncopal episodes, impaired decision-making, and slowed reaction times would make their safety and that of those around them a significant concern.

My sons’ limited capacity for (cognitive or physical) exertion is not a result of deconditioning.

For one son, the exertion intolerance (a hallmark of this disease) began quite abruptly with the sudden onset of his illness.

For my other son, the exertion intolerance and the disease revealed themselves more gradually.

And as I mentioned above, when they became ill, they were swimming at least 20 miles a week so they were in great physical shape. Therefore deconditioning is in no way an explanation of their exertion intolerance.

These days cognitive and or physical exertion in excess of the strict limits imposed by this horrible disease, produce a dramatic exacerbation of symptoms (post-exertional malaise or PEM) that can last for weeks and render my sons even more disabled than their baseline (which on a good day is about 30% of normal).

All too often I see comments by healthcare professionals saying these patients want stimulants, narcotics, Xanax, disability and disabled parking tags.

My sons take no stimulants (bad reactions to them), no narcotics, no Xanax, etc.

Neither of my sons want to use the disabled parking tag that we have – they don’t want to feel labeled by doing so. So we use the tag quite sparingly – but then again my sons are out of the house so rarely that any use is sparingly.

My sons don’t get SSDI (Social Security Disability) – they have never been able to work so therefore never accrued work credits to qualify for SSDI and having never worked, they never had the option of long-term disability insurance.

Since my sons’ illness onset and because they are so severely impaired by this disease, I have been their 24/7 unpaid caregiver.

There is NOTHING to gain for us with this disease and we do our best not to think about the significant losses (independence, educational aspirations, making a difference in the world, spontaneity, vacations, and on and on).

So you see, your comments about us are not accurate. Nor are they appropriate. And inaccurate, inappropriate, disparaging, and or demeaning comments such as yours cause significant pain and detract from your job which is to help patients.

We don’t need or deserve additional pain and or hardship in our lives.

In public and in private, we deserve respect, compassion, appropriate diagnoses and appropriate treatment. NOW!

Sincerely,

A parent of two patients with ME

May 12th is ME Awareness Day

One of our ways of explaining

While this was written by way of explanation of some of what people might see if they see my sons, please feel free to adapt it if it might be of help for you.

If you see my sons and don’t get much reaction from them:

They are not depressed.

Please understand that one of their coping mechanisms is maintaining an even keel.

Showing emotional highs and lows, even in greeting someone, uses too much energy for them.

They are not being rude. They are not sullen. They are not displeased. They are not ignoring you.

They appreciate you. They appreciate your presence, involvement, support… But they don’t have energy to show /convey that.

If they make eye contact, that’s acknowledgment.

If they manage a raised eyebrow, a tiny upturned corner of the mouth, the slightest nod of a head – that is huge. And to do that, they must be feeling pretty good (relatively speaking of course).

You may notice a fair amount of non-verbal communication between us. It may be our heads touching, hand on a shoulder, a goofy look from me…. We have developed a shorthand that between us that requires less of their energy to convey and process. It is usually easier for them to talk with me because we have shared so much of this wretched ME experience together and they don’t have to give as much detail or explanation.

Five days into one crash, one of my sons was (still) feeling that breathing was not automatic, that his brain was barely functioning at all.

And yet making the conscious effort to breath requires cognitive exertion and cognitive exertion further exacerbates the crash…..

Don’t ask him to make decisions – he just can’t do it.

Don’t expect him to have ideas.

He may be able to say yes or no to an option but not be able to come up with a substitute if his answer is no.

Don’t be surprised if he “zones out” mid-sentence, mid-bite, mid-word. It’s as though the brain gets stuck, on hold ………

 

Additions from LD

If their response is very succinct, it may well be because they are trying to cope with pain, don’t have energy to explain in depth….

It is often best to ask only 1 question at a time.  2 is too (2!) many.

Just because they “look” fine, does not mean that they feel fine.

If they wince when you hug them, it is not because of you, it is because it causes them real pain.

If they leave an area, it isn’t because they don’t like the company – but it likely that there is too much noise, too much commotion, too much scent, it may be too bright, there may be too many conversations going on to track and by leaving the area they are trying to “last” and not crash.  Their senses go off the charts, way faster than yours do. They will come back if they can.

Don’t feel badly, if they cancel on you at the last minute (or seem to ALWAYS cancel plans) – they don’t know minute to minute, let alone day to day, how they will feel.

Don’t be surprised if they say they feel fine. This is all relative. They always feel “bad” – just varying degrees of it. So if they say they feel fine, it likely means they feel about at baseline, about their usual and not their worst.

 

Webcast of FDA meeting on Thursday 25 April and Friday 26 April

The FDA Drug Development for ME and CFS Public Workshop will be held on Thursday 25 April and Friday 26 April 2013

Note: On Thursday, the meeting begins at 1PM (Eastern Time U.S.)

On Friday the meeting begins at 8:30AM (Eastern Time U.S.)

 

Here is the link for the webcast :

http://www.tvworldwide.com/events/fda/130425/globe_show/default_go.cfm?live=1&type=flv&test=0

 

If this link does not work, there is a backup link on this page

http://www.tvworldwide.com/events/fda/130425/ (right hand side of page, look for the second PLAY button)

 

 

As you watch the webcast, make notes about your answers to these questions from FDA:

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)?

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.

 

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.

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)?

 

How are YOU participating in the April 25/26th FDA workshop?

Hear ye! Hear ye!

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 at https://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

Here’s what you need to do:

  • To attend in person, register first-come, first-serve at http://mecfsmeeting.eventbrite.com by April 8. You will be asked to provide your name and business, organization, or personal affiliation as applies (e.g., industry, government, patient).
  • If you wish to be considered to present comments part of an initial panel discussions, you will need to indicate the topic that you wish to address. You should send a brief summary of your response to the topic(s) you selected to ME-CFS-Meeting@fda.hhs.govby April 8.  You will be notified before the meeting.
  • If you wish to provide comments during the ‘open discussion’, you do not need to register to provide comments or have prepared testimony.
  • 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)?

Thank you M.D. for distilling this info!

Early/pediatric onset list reminder

People within our community have many different perspectives, and yet those differences pale in comparison to our commitment to move things forward.

We are learning a lot from each other as we engage in interesting and informative discussions. We have many common goals.

As we look forward to 2013 we hope that our community continues to build on our strengths so we can make even greater strides for the benefit everyone affected by ME(CFS).

 

PLEASE SPREAD THE WORD:

Speak Up About ME maintains a list of early/pediatric onset ME/CFS.

If you came down with ME/CFS before age 23, please add your name and illness info to the list*!

Provide

name (let us know if we should use your full name, first name only or a nickname that you provide),

age at onset,

age at diagnosis,

current age,

location (country is sufficient).

Encourage others with early (pediatric) onset ME/CFS to get their onset information listed also.

Send your onset info to: speakupaboutme at gmail dot com.