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Update on ME/CFS Patient Alliance meeting of Oct. 18 with Dr. Nancy Lee

Details: Last Updated: 23 January 2016 23 January 2016

Submitted by Mary Dimmock on Oct. 27, 2012

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.

[Editorial Comment: To read these letters, click on DHHS Response to Joint Request for Action prompts second letter.]

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 This email address is being protected from spambots. You need JavaScript enabled to view it.. We'll summarize any ideas we get in and share them back with the community.

Summary of the Patient-Focused Drug Development Initiative

Details: Last Updated: 23 January 2016 23 January 2016

What is it: As part of its commitment under the recently approved Prescription Drug User Fee Act (PDUFA V), the Food and Drug Administration (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/#!docketDetail;D=FDA-2012-N-0967. Select ‘Individual Consumer' for the ‘Category' and ‘None' for ‘Organization' if no other choice is appropriate . 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.

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

Sample Letter

Sample letter must be limited to 2000 characters

To: FDA Patient Focused Drug Development Initiative
From:

I am writing to request that chronic fatigue syndrome (also called myalgic encephalomyelitis or ME/CFS) 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. It affects people of all ages, races and income levels. Patients can be sick for decades, with 25% house, bed or wheelchair bound, struggling to take care of themselves, let alone take care of their families or work. 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. One study suggests patients can die prematurely from cancer, heart disease and suicide.

What is tragic is that almost thirty years after the outbreaks that brought ME/CFS to national attention, there have been almost no clinical trials for drugs to treat ME/CFS and there are still NO approved treatments and NO biomarkers or outcome measures have been agreed upon.

(include your personal story here)

By ensuring a thorough understanding of the severity of ME/CFS from a patient perspective, especially given the lack of any viable 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.

FDA Patient-Focused Drug Development Intiative - Sample Letter (by November 1, 2012)

Details: Last Updated: 01 November 2012 01 November 2012

How can you help: Please submit your comments to the FDA.

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. 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 below.

Please send your comments in by November 1, 2012, to ensure that the FDA understands why ME/CFS should be selected as one of the 20 diseases.You can submit your letter electronically or mail it.

Comments should be submitted electronically at http://www.regulations.gov/#!docketDetail;D=FDA-2012-N-0967 Select ‘Individual Consumer’ for the ‘Category’ and ‘None’ for ‘Organization’ if no other choice is appropriate.
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.

Sample Letter – limited to 2000 characters

To: FDA Patient Focused Drug Development Initiative
From:

I am writing to request that chronic fatigue syndrome (also called myalgic encephalomyelitis or ME/CFS) 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. It affects people of all ages, races and income levels. Patients can be sick for decades, with 25% house, bed or wheelchair bound, struggling to take care of themselves, let alone take care of their families or work. 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. One study suggests patients can die prematurely from cancer, heart disease and suicide.

What is tragic is that almost thirty years after the outbreaks that brought ME/CFS to national attention, there have been almost no clinical trials for drugs to treat ME/CFS and there are still NO approved treatments and NO biomarkers or outcome measures have been agreed upon.

By ensuring a thorough understanding of the severity of ME/CFS from a patient perspective, especially given the lack of any viable 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.

Sincerely yours,

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

The listing that follows 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, and

How ME/CFS meets that criteria

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 affected – 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

Stigma and confusion about nature of disease

Significant confusion on the nature of the disease and whether it’s the same as depression or just ‘all in the patient’s head’ has both
Misunderstanding and misperceptions about the burden of disease from a patient perspective. – its not “just tired”
No agreement on objective measures of patient function although 2 day CPET is a good measure.
Few validated subjective measures of patient function
Limited R&D investment