Posts Tagged 'janet woodcock'

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.

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.

 

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

FDA teleconference of Sept 13, 2012

 

Please read this excellent summary by Ms. Spotila of the teleconference call:

http://www.occupycfs.com/

Here is additional information about the FDA and NIH designations of serious or life-threatening condition:

 

[Code of Federal Regulations]
[Title 21, Volume 5]
[Revised as of April 1, 2012]
[CITE: 21CFR312.81]

 

TITLE 21–FOOD AND DRUGS

CHAPTER I–FOOD AND DRUG ADMINISTRATION
DEPARTMENT OF HEALTH AND HUMAN SERVICES

 

SUBCHAPTER D–DRUGS FOR HUMAN USE

 

 

PART 312 — INVESTIGATIONAL NEW DRUG APPLICATION

Subpart E–Drugs Intended to Treat Life-threatening and Severely-debilitating Illnesses

Sec. 312.81 Scope.
This section applies to new drug and biological products that are being studied for their safety and effectiveness in treating life-threatening or severely-debilitating diseases.

(a) For purposes of this section, the term “life-threatening” means:

(1) Diseases or conditions where the likelihood of death is high unless the course of the disease is interrupted; and

(2) Diseases or conditions with potentially fatal outcomes, where the end point of clinical trial analysis is survival.

(b) For purposes of this section, the term “severely debilitating” means diseases or conditions that cause major irreversible morbidity.

(c) Sponsors are encouraged to consult with FDA on the applicability of these procedures to specific products.

[53 FR 41523, Oct. 21, 1988, as amended at 64 FR 401, Jan. 5, 1999]

http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm

 

 

 

 

Serious or life threatening disease or condition

Consistent with current FDA and ClinicalTrials.gov guidance, the NIH interprets “serious and life-threatening disease or condition” to mean: (1) diseases or conditions where the likelihood of death is high unless the course of the disease is interrupted and (2) diseases or conditions with potentially fatal outcomes, where the endpoint of clinical trial analysis is survival.

Any investigational drug that has received fast track designation by the FDA is considered a drug to treat a serious disease or condition.

The seriousness of a disease is a matter of judgment, but generally is based on such factors as survival, day-to-day functioning, and the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one.  For further elaboration of the NIH’s interpretation of “serious or life threatening disease or condition”, please access the “Fact Sheet: Registration at ClinicalTrials.gov”.

 

 

 

http://grants.nih.gov/clinicaltrials_fdaaa/definitions.htm?print=yes&

A transcript of the call should be available by Sept 21st…    

 

 

 

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

one a day til June 25th LETTERS TO THE FDA TO HOLD A STAKEHOLDER MEETING!

CALL FOR THE FDA TO HOLD A STAKEHOLDER MEETING – LETTER!
June 25
th, 2012

To All who took ACTION requesting THE FDA TO HOLD A STAKEHOLDERS MEETING for ME/CFS: Great job and Thank You!

As a result of your Emails / Letters, patients have been meeting with Congressional offices and support is being gathered from key Representatives.

We do not yet have a meeting set with FDA re: The ME/CFS Stakeholders Meeting.

Therefore, we ask everyone to send in a follow up email / letter.

This letter is directed to FDA Commissioner Margret Hamburg, ensuring that the stakeholder meeting is held. We urge Dr. Hamburg to direct that this critical meeting take place.

PLEASE EMAIL YOUR LETTER DAILY UNTIL June 25thand urge your relatives and friends to do the same. A sample template letter and email addresses are included below. Copy, Paste, add your name etc at the bottom and Send.

YOUR LETTERS COUNT. Remember – LOBBYING & ADVOCACY is how AIDS/HIV got $3 BILLION a year for research, CFS gets $6 million. AIDS/HIV sufferers now have multiple drugs to fight the disease. We have NONE.  Your previous letter/emails did generate a response, just no confirmed Date.

WE MUST KEEP THE HEAT ON, Those who work for our benefit need to Act!

Thank you everyone for taking ACTION for YOUR HEALTH!

Copy & Paste email addresses into the To: Slot of your email

To: margaret.hamburg@fda.hhs.gov, janet.woodcock@fda.hhs.gov, Kathleen.Sebelius@hhs.gov ,

matthew_prowler@casey.senate.govmonica.volante@mail.house.gov

From: Your Name June 25, 2012

To: FDA Commissioner Margaret Hamburg, M.D

cc: Janet Woodcock Director CDERFDA, Secretary of Health Kathleen Sebelius, Senator Casey and Congressman Pitts

Subject: Request for FDA Stakeholder Meeting on Chronic Fatigue Syndrome

Dear Dr. Hamburg

As patients suffering from Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS), we believe your commitment of the FDA holding stakeholder meetings is critical to the FDA understanding and evaluating treatments, particularly to those of us suffering from debilitating/devastating chronic illnesses. As a patient population we are long overdue for treatments and the movement through six divisions at the FDA has contributed to ZERO treatments being available.

We are asking you to convene a “true” stakeholder meeting to examine the treatments currently in use for ME/CFS both in trial and those being used off-label. We anticipate that this meeting will be at least a full day meeting in order to accommodate the experts, clinicians, researchers, patient advocacy groups and patients as well as FDA expertise from a variety of divisions to ensure understanding of this complex illness. With millions of patients suffering from ME/CFS at a cost to our nation of $23 billion, it more than outweighs any cost to the FDA to convene such a meeting. The goal of the meeting is to examine benefit/risk and develop a pathway for the acceleration of treatment, a pathway that can be agreed upon by the ME/CFS community with a commitment by the FDA to change the course of this disease.

As one of the millions of people suffering from ME/CFS, I cannot even dream about a better quality of life because there is simply no avenue to get there. I live every day with a disease that the CDC has said is as disabling as multiple sclerosis, heart disease, end-stage renal disease and similar chronic conditions; AND YET THERE ARE ZERO TREATMENTS TODAY. As this falls within your responsibility to address this “unmet medical need”, please Act Now.

Your Name

Address

City, State – Zip code