Posts Tagged 'disability'

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.

 

“FDA to rule on ‘Lazarus’ drug” Llewellyn King

http://www.stamfordadvocate.com/local/article/FDA-to-rule-on-Lazarus-drug-4121250.php
“FDA to rule on ‘Lazarus’ drug Llewellyn King Published 4:41 pm, Saturday, December 15, 2012


WASHINGTON –For about a million Americans, Thursday will be a seminal day. That’s when some of them come before the Food and Drug Administration to petition for approval of a potent and controversial drug, a so-called Lazarus drug.


The drug, first synthesized by Hemispherx Biopharma, Inc., of Philadelphia, in the 1970s, is Rintatolimod (tradename Ampligen), which is used to treat chronic fatigue Syndrome, also known as myalgic encephalomyelitis. It is a grim but little-understood disease of the immune system, resulting in collapse, pain, confusion and sensitivity to light and noise.

Patients and their doctors want the drug, but there is concern that the FDA will fault — as it has in the past — the scope of the clinical trials and the documentation of collateral effects.

The FDA is expected to rule early next year.           

…..

The patients turn to the government for recognition, but the government turns them into a statistic.

We are PEOPLE, not statistics.

Please do everything possible to be at the meeting on Dec. 20th, 2012 so that FDA sees people (not statistics)..

.

 

The full article can be found here http://www.stamfordadvocate.com/local/article/FDA-to-rule-on-Lazarus-drug-4121250.php

Message for the ME/CFS community

On June 5, 2012, an alliance of ME/CFS patient organizations and advocates sent a joint request to Secretary Sebelius of DHHS requesting that key deputies meet with us and begin to work with us to formulate a strategic, coordinated and fully-funded response to ME/CFS. We reiterated that request in a letter to Secretary Sebelius on August 8.

To date, we have been unable to secure the meeting with key deputies that we requested.  Four members of the alliance (Joan Grobstein, Jennie Spotila, Mary Dimmock and Charlotte von Salis) did meet with Dr. Nancy Lee, Deputy Assistant Secretary for Health – Women’s Health and Dr. Caira Woods, Advisor for Health and Science Policy, Office on Women’s Health.  The key points discussed in that meeting were:

  • The Ad-Hoc Workgroup (described in DHHS letters to the community and at the CFSAC) is formulating a plan focused on budget sharing and coordination across agencies. It is charged to do what it can with no additional funding. It is not charged with developing a formal strategic plan or action plan. Dr. Lee also stressed that the agencies have a great degree of autonomy, although they do collaborate with each other.
  • Given that DHHS has not agreed to the requested cross-DHHS meeting and has not committed to a cross-agency strategic, coordinated, fully-funded response, other avenues should be pursued. Dr. Lee suggested approaches that include non-profit or private sector solutions and following up with CDC and NIH separately.
  • There are tactical opportunities that could be pursued in parallel and in partnership with CFSAC that include engaging as stakeholders in the case definition process that CFSAC committed to and finding ways to improve engagement and two way dialog within the CFSAC itself.
  • We also discussed the CDC CFS Toolkit. The link to the CDC CFS Toolkit has been removed from the CFSAC website which is very good. Note that the Toolkit is still on the CDC CFS website itself. We will have to pursue that separately.

The need for a cross-DHHS strategic, coordinated, fully-funded response, developed with meaningful stakeholder input is as critical now as it ever was. However, it was clear from this meeting that we will need to use alternative approaches to make incremental progress while working toward the full objective. Besides the approaches suggested above, there are also legislative and other non-governmental options. Given our community’s limited resources, we will prioritize those activities that are most critical to both our short and long-term success.

If you have ideas on the areas that you would prioritize that you’d like to share, please share them with your organization or send them to MECFSACTION@Yahoo.com. We’ll summarize the ideas we get in and share them back with the community.

Thank you very much Charlotte, Joan, Jennie and Mary for meeting with Drs. Nancy Lee and Caira Woods.

Comments due November 1st!

REMINDER – The Patient Focused Drug Development Initiative is an excellent opportunity to help the FDA better understand how ME/CFS affects the patients.

Send your comments in by November 1 to ensure that the FDA understands why ME/CFS should be selected as one of the 20 diseases. A sample letter has been provided in case you want to pull from it to develop your own. As you write your own letter, make points that reflect how ME/CFS meets the criteria above.

Comments should be submitted electronically at

http://www.regulations.gov/

FDA-2012-N-0967

http://www.regulations.gov/#!docketDetail;D=FDA-2012-N-0967

Written comments can be submitted to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. Make sure you include the docket number (FDA-2012-N-0967).

Further information on the patient focused drug development initiative and the list of 39 diseases initially nominated can be found here: https://www.federalregister.gov/articles/2012/09/24/2012-23454/prescription-drug-user-fee-act-patient-focused-drug-development-public-meeting-and-request-for

1. Sample Letter

  • Note that the letters are limited to 2000 characters so you may need to remove some of the sample text provided to allow additional space for your personal story.
  • If you are sending in comments by regular mail, include the’To’, the ‘From’ and the ‘Docket number’. These are not necessary if sending in electronically.
  • If you are sending in electronically, select ‘Individual Consumer’ for the ‘Category’ and ‘None’ for ‘Organization’ if no other choice is appropriate

To: FDA Patient Focused Drug Development Initiative

From: <include your name here>

Docket Number: FDA-2012-N-0967

I am writing to request that chronic fatigue syndrome (also called myalgic encephalomyelitis or ME/CFS in the U.S.) be included as one of the 20 diseases in the patient focused drug development initiative.

ME/CFS is a complex, neuroimmune disease that affects one million Americans of all ages, races and income levels. According to the CDC, ME/CFS can be as debilitating as Multiple Sclerosis (MS), end-stage renal disease, chronic obstructive pulmonary disease (COPD) and similar chronic conditions. Patients can be sick for decades, with 25% house, bed or wheelchair bound. While some patients work part time or rest all weekend to keep going in their jobs, others struggle to take care of themselves, let alone take care of their families or work. One study suggests patients can die prematurely from cancer, heart disease and suicide.

And yet, tragically, in the almost thirty years since the outbreaks that brought ME/CFS to national attention, there are NO approved drugs, NO agreement on biomarkers or outcome measures and there have been almost no clinical trials for drugs to treat the disease course of ME/CFS. Patients have been abandoned with no relief from the devastation of ME/CFS.

<Include personal story here – make points that speak to one of the criteria – e.g. how this has affected you functionally, symptomatically (e.g. cognitive impact, pain), or how it has affected your daily activities, how this has affected your child with ME/CFS, the lack of therapy or your inability to access cause they are not approved for ME/CFS.

By ensuring a thorough understanding of the severity of ME/CFS from a patient perspective, especially given the lack of any treatments to change the course of the disease, the patient focused drug development initiative could make a tremendous difference in the lives of one million Americans.

2. Additional information

FDA Disease Area Selection Criteria and How ME/CFS Meets These Criteria

This table is intended to help you as you are writing your letter by providing additional information about how ME/CFS meets the FDA criteria for selection of disease areas. Try to include points in your letter that hit on these key criteria.

FDA Criteria How ME/CFS meets that criteria – examples as food for thought
Disease areas that are chronic, symptomatic, or affect functioning and activities of daily living;
  • Chronic, sick for decades, low percentage of patients improve
  • 25% bedbound or homebound, unable to work. Patients who do work may work part time and/or are underemployed. Relapsing course means little predictability. Impact of disease on patient’s life is so great that suicide risk increased
Disease areas that reflect a range of severity;
  • Wide range of severity – some patients work a 40 hour week and compensate by sleeping weekends but many patients can not work at all. The most serious patients are unable to do little more than the most basic activities of personal care
Disease areas for which aspects of the disease are not formally captured in clinical trials;
  • There is only one drug in clinical trials and its been there since 1997
  • There are a few INDs but most are focused on nutriceuticals. There have been studies into psychological treatments
Disease areas that have a severe impact on identifiable subpopulations (such as children or the elderly);
  • Severe impact on children because they are unable to attend school or graduate. Even if they get better, they have missed on significant life lessons
Disease areas that represent a broad range in terms of size of the affected population
  • Estimated that 1 million are affects – women and men, all economic levels, all ethnic groups
Disease areas for which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels, functions, or survives.
  • No approved therapies, only limited symptom relief

Letters can be from patients, family members, friends….. and from any country as well!

Please help make sure that ME/CFS is one of the 20 diseases selected.

(During the FDA meeting on Oct 25th 2012, FDA officials said that it will take them a couple of months to determine which diseases will be selected.)

 

 

 

CALL TO ACTION

Summary of the Patient Focused Drug Development Initiative

What is it: As part of its commitment under the recently approved Prescription Drug User Fee Act (PDUFA V), the FDA will be conducting an initiative, called the patient-focused drug development initiative, to provide for a more systematic approach to obtain the patient’s perspective on the disease severity and the currently available treatments. The intent is to ensure a thorough understanding of the severity of the treated condition and the adequacy of the existing treatment options.

This initiative will be conducted for each of 20 different disease areas over a period of 5 years. The FDA has nominated an initial list of 39 diseases, including ME/CFS, using the following criteria:

  • Disease areas that are chronic, symptomatic, or affect functioning and activities of daily living;
  • Disease areas that reflect a range of severity;
  • Disease areas for which aspects of the disease are not formally captured in clinical trials;
  • Disease areas that have a severe impact on identifiable subpopulations (such as children or the elderly);
  • Disease areas that represent a broad range in terms of size of the affected population
  • Disease areas for which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels, functions, or survives.

The next step in the process is for the FDA to gather public input between now and November 1st on which of disease areas should be selected for inclusion in this initiative. Additional disease areas may also be nominated during this time.

Note that initiative is in addition to the ME/CFS FDA stakeholder meeting that Dr. Woodcock, Director of the Center for Drug Evaluation and Research at the FDA, has already committed to.

How can you help: For ME/CFS, this is an excellent opportunity to help the FDA better understand how ME/CFS affects the patients. Your support is essential to ensure that ME/CFS is one of the 20 selected diseases.

Please send your comments in by November 1 to ensure that the FDA understands why ME/CFS should be selected as one of the 20 diseases. A sample letter has been provided in case you want to pull from it to develop your own. As you write your own letter, make points that reflect how ME/CFS meets the criteria above.

Comments should be submitted electronically at

http://www.regulations.gov/

FDA-2012-N-0967

http://www.regulations.gov/#!docketDetail;D=FDA-2012-N-0967

Written comments can be submitted to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. Make sure you include the docket number (FDA-2012-N-0967).

Further information on the patient focused drug development initiative and the list of 39 diseases initially nominated can be found here: https://www.federalregister.gov/articles/2012/09/24/2012-23454/prescription-drug-user-fee-act-patient-focused-drug-development-public-meeting-and-request-for

1. Sample Letter

  • Note that the letters are limited to 2000 characters so you may need to remove some of the sample text provided to allow additional space for your personal story.
  • If you are sending in comments by regular mail, include the’To’, the ‘From’ and the ‘Docket number’. These are not necessary if sending in electronically.
  • If you are sending in electronically, select ‘Individual Consumer’ for the ‘Category’ and ‘None’ for ‘Organization’ if no other choice is appropriate

To: FDA Patient Focused Drug Development Initiative

From: <include your name here>

Docket Number: FDA-2012-N-0967

I am writing to request that chronic fatigue syndrome (also called myalgic encephalomyelitis or ME/CFS in the U.S.) be included as one of the 20 diseases in the patient focused drug development initiative.

ME/CFS is a complex, neuroimmune disease that affects one million Americans of all ages, races and income levels. According to the CDC, ME/CFS can be as debilitating as Multiple Sclerosis (MS), end-stage renal disease, chronic obstructive pulmonary disease (COPD) and similar chronic conditions. Patients can be sick for decades, with 25% house, bed or wheelchair bound. While some patients work part time or rest all weekend to keep going in their jobs, others struggle to take care of themselves, let alone take care of their families or work. One study suggests patients can die prematurely from cancer, heart disease and suicide.

And yet, tragically, in the almost thirty years since the outbreaks that brought ME/CFS to national attention, there are NO approved drugs, NO agreement on biomarkers or outcome measures and there have been almost no clinical trials for drugs to treat the disease course of ME/CFS. Patients have been abandoned with no relief from the devastation of ME/CFS.

<Include personal story here – make points that speak to one of the criteria – e.g. how this has affected you functionally, symptomatically (e.g. cognitive impact, pain), or how it has affected your daily activities, how this has affected your child with ME/CFS, the lack of therapy or your inability to access cause they are not approved for ME/CFS.

By ensuring a thorough understanding of the severity of ME/CFS from a patient perspective, especially given the lack of any treatments to change the course of the disease, the patient focused drug development initiative could make a tremendous difference in the lives of one million Americans.

2. Additional information

FDA Disease Area Selection Criteria and How ME/CFS Meets These Criteria

This table is intended to help you as you are writing your letter by providing additional information about how ME/CFS meets the FDA criteria for selection of disease areas. Try to include points in your letter that hit on these key criteria.

FDA Criteria How ME/CFS meets that criteria – examples as food for thought
Disease areas that are chronic, symptomatic, or affect functioning and activities of daily living;
  • Chronic, sick for decades, low percentage of patients improve
  • 25% bedbound or homebound, unable to work. Patients who do work may work part time and/or are underemployed. Relapsing course means little predictability. Impact of disease on patient’s life is so great that suicide risk increased
Disease areas that reflect a range of severity;
  • Wide range of severity – some patients work a 40 hour week and compensate by sleeping weekends but many patients can not work at all. The most serious patients are unable to do little more than the most basic activities of personal care
Disease areas for which aspects of the disease are not formally captured in clinical trials;
  • There is only one drug in clinical trials and its been there since 1997
  • There are a few INDs but most are focused on nutriceuticals. There have been studies into psychological treatments
Disease areas that have a severe impact on identifiable subpopulations (such as children or the elderly);
  • Severe impact on children because they are unable to attend school or graduate. Even if they get better, they have missed on significant life lessons
Disease areas that represent a broad range in terms of size of the affected population
  • Estimated that 1 million are affects – women and men, all economic levels, all ethnic groups
Disease areas for which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels, functions, or survives.
  • No approved therapies, only limited symptom relief

Letters can be from patients, family members, friends….. and from any country as well!

Please help us make sure that ME/CFS is one of the 20 diseases selected.

October 2012 CFSAC videos

(Sorry if these are not in order…. the last link is to a page that has all of the videos)

 

http://www.youtube.com/watch?v=4Zl2RX4URuI&feature=plcp Welcome & Opening Remarks (CFSAC Fall 2012)

http://www.youtube.com/watch?v=01hZYq4GMtI&feature=plcp Biomarkers – An Overview & Future Look (CFSAC Fall 2012)

http://www.youtube.com/watch?v=oIGpfvfFZ1w&feature=relmfu Public Comment on Day One – AM (CFSAC Fall 2012)

http://www.youtube.com/watch?v=buDxhejdg90&feature=plcp Agency Updates from HRSA, NIH, and FDA (CFSAC Fall 2012)

http://www.youtube.com/watch?v=IGASSh7YV3s&feature=plcp FDA & Drug Development (CFSAC Fall 2012)

http://www.youtube.com/watch?v=_fIEeQBdvxM&feature=plcp Public Comment on Day One – PM (CFSAC Fall 2012)

http://www.youtube.com/watch?v=Fs1A6KhHInU&feature=relmfu Committee Discussion & Plans for Day Two (CFSAC Fall 2012)

http://www.youtube.com/watch?v=PWBmqd8LDhg&feature=plcp Opening Remarks & Agency Updates (CFSAC Fall 2012)

http://www.youtube.com/watch?v=af_r5LiIjXQ&feature=relmfu Social Security Administration (CFSAC Fall 2012)

http://www.youtube.com/watch?v=qGb0U6EaI4o&feature=relmfu Public Comment on Day Two (CFSAC Fall 2012)

http://www.youtube.com/watch?v=MTHpny0u-XU&feature=relmfu ME/CFS Organizations (CFSAC Fall 2012)

http://www.youtube.com/watch?v=NFaXiiSdZqI&feature=relmfu A Path Forward (CFSAC Fall 2012)

http://www.youtube.com/watch?v=CO9bO-udcY0&feature=relmfu Finalize Recommendations (CFSAC Fall 2012)

http://www.youtube.com/user/USGOVHHS/videos?query=CFSAC Search results for “CFSAC”:

 

 

 

Link for CFSAC LiveStream

 

On Wednesday October 3rd and Thursday October 4th, the CFSAC meeting will be LiveStreamed. The link is:

http://www.hhs.gov/live/

 

CFSAC Meeting October 3 – 4

Agenda
October 3, 2012

9:00 am

Call to Order
Roll Call

Gailen Marshall Jr. M.D., PhD
Chair

9:15 am

Welcome Statement from the Assistant Secretary for Health

Howard K. Koh, M.D., M.P.H.Assistant Secretary for Health
U.S. Department of Health and Human Services

9:45 am

Opening Remarks

Housekeeping

Gailen Marshall Jr. M.D., PhD Chair

Nancy C. Lee, M.D.
Designated Federal Officer

10:00 am

Biomarkers
An Overview and Future Look

Jordan Dimitrakoff, M.D., Ph.D.
Mary Ann Fletcher, Ph.D.
Committee Members

11:00 am 

Break

11:15 am 

Public Comment

Public

12:15 pm

Lunch Break

1:15pm

Agency Updates:
HRSA, NIH, FDA

Ex Officio Members

2:15pm

FDA and Drug Development

Sandra L. Kweder, M.D., F.A.C.P.
RADM U.S. Public Health Service
Deputy Director, Office of New Drugs
Center for Drug Evaluation & Research, FDA

3:15 pm  

Break

3:30pm

Public Comment

Public

4:30pm

Committee Discussion and Plans for Day 2

Committee Members

5:00 pm

Adjourn

Nancy C. Lee, M.D.

Agenda

October 4, 2012

9:00 am

Call to Order
Opening Remarks
Roll Call


Housekeeping

Gailen Marshall Jr. M.D., PhD
Chair

Nancy C. Lee, M.D.
Designated Federal Officer

9:15 am

Agency Updates:
CMS, AHRQ, CDC, SSA

Ex Officio Members

10:15 am

Social Security Administration

Arthur R.(Art) Spencer
Associate Commissioner,
Office of Disability Programs
SSA

11:15 am

Break

11:30 am

Public Comment

Public

12:30 pm

Lunch Break

Subcommittee Members

1:30 pm

ME/CFS Organizations

Panel Discussion
ME/CFS Organizations

2:30 pm

Break

2:40 pm

ME/CFS Case Definition – A Path Forward

Gailen Marshall Jr. M.D., PhD
Chair
Committee Members

3:40 pm

Committee Discussion: Finalize Recommendations

Committee Members

5:00 pm

Adjourn

Nancy C. Lee, M.D.