Reminder – FDA webinar Wednesday (23 April) 1pm eastern time

This webinar is about the Draft Guidance to Industry on Drug Development for ME/CFS (http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM388568.pdf)

The webinar will have some Q&A time – so make submit your questions.

“Guidance Webinar Online-Access Instructions:

To access this webinar, follow the link provided below. Audio will broadcast from your computer speakers.

After following the link, enter as a guest and provide your FULL NAME and organization (i.e. “John Smith – FDA/CBER”). The host will then allow you to enter. If you experience technical difficulties email Jeffery.Rexrode@fda.hhs.gov for assistance. Closed captioning will be provided. Questions/Comments can be submitted live via a Q/A chat window.”

http://www.fda.gov/Training/GuidanceWebinars/ucm392577.htm

In your public comment on the Draft Guidance (due 12 May 2014), consider including the positives in the guidance as well as concerns you may have.

Submit your written comments to the docket (Docket No. FDA-2014-D-0264 ) http://www.regulations.gov/#!searchResults;rpp=25;po=0;s=FDA-2014-D-0264;fp=true;ns=true

Summary of April 26, 2013 FDA meeting (day 2)

Recently FDA released a summary (http://www.fda.gov/Drugs/NewsEvents/ucm386705.htm) of the April 26, 2013 FDA Workshop on Drug Development for Chronic Fatigue Syndrome (CFS) and Myalgic Encephalomyelitis (ME). You may recall that this was the second day of a 1.5 day meeting and on this day, there were discussions about identifying quantitative outcome measures and determining which treatments produce improvements.

Remember that FDA is currently producing a guidance document for CFS and ME drugs that will provide advice for pharmaceutical companies and industry to expedite research for treatments. Our input will be needed on this. Note – typically when a draft guidance is released there is a 60 day comment period.

Additional information about the meeting and links to transcripts etc. can be found here: http://www.fda.gov/Drugs/NewsEvents/ucm369563.htm

FDA CFS and ME Teleconference – October 16, 2013

 

In case you have not seen it, FDA has produced a report based on the April 25^th meeting. You can download it or read it here http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM368806.pdf. Based on the input received at the meeting and from material submitted to the docket, their characterization of ME is quite good.

As a followup to the April FDA meeting, FDA is hosting a teleconference on Wednesday Oct 16^th fro 1:30-3pm (eastern time). Details below.

On October 16 from 1:30 PM – 3:00 PM EST, FDA will hold a teleconference that is open to the public. FDA will provide introductory and background remarks then the teleconference will be open for public comment on the following three topics:

1.            Feedback from the CFS and ME public workshop.

2.            Current projects related to drug development in CFS and ME.

3.            Potential methods to catalyze CFS and ME drug development.

Registration (Closes October 11, 2013): https://cfsmeteleconference2013.eventbrite.com/

FDA CFS and ME Activities Website: http://www.fda.gov/Drugs/NewsEvents/ucm319188.htm

Please contact Randi Clark (Randi.Clark@fda.hhs.gov) or Mary Gross (Mary.Gross@fda.hhs.gov) if you have any questions.

There’s still time to submit comments to FDA docket on Drug Development for ME and CFS

Did you know, as of July 17th 2013, only 193 comments have been submitted to the FDA docket on Drug Development for ME and CFS?

Comments (from any country) can still be submitted (http://www.regulations.gov/#!submitComment;D=FDA-2012-N-0962-0004 ) until August 2, 2013.

Remember you can attach a document if your comments are more than 2000 characters long and/or you can provide your comment via video with a YouTube link.

FDA suggested two topics for comment. Consider including medicines you have tried – even if off-label – and their effects (positive, negative, no effect,side effects, intolerable side effects, etc.).

 

FDA Suggested Topic 1: Disease Symptoms and Daily Impacts That Matter Most to Patients

1. What are the most significant symptoms that you experience resulting from your condition? (Examples may include prolonged exhaustion, confusion, muscle pain, heat or cold intolerance.)

2. What are the most negative impacts on your daily life that result from your condition and its symptoms? (Examples may include difficulty with specific activities, such as sleeping through the night.)

a. How does the condition affect your daily life on the best days and worst days?

b. What changes have you had to make in your life because of your condition?

FDA Suggested Topic 2: Patients’ Perspectives on Current Approaches To Treating CFS and ME

1. What treatments are you currently using to help treat your condition or its symptoms? (Examples may include FDA-approved medicines, over-the-counter products, and other therapies, including non-drug therapies such as activity limitations.)

a. What specific symptoms do your treatments address?

b. How has your treatment regimen changed over time and why?

2. How well does your current treatment regimen treat the most significant symptoms of your disease?

a. Have these treatments improved your daily life (for example, improving your ability to do specific activities)? Please explain.

b. How well have these treatments worked for you as your condition has changed over time?

c. What are the most significant downsides of these treatments (for example, specific side effects)?

Transcript Day I April 25, 2013 http://www.fda.gov/downloads/Drugs/NewsEvents/UCM354951.pdf

Transcript Day II April 26, 2013 http://www.fda.gov/downloads/Drugs/NewsEvents/UCM355406.pdf

 

Video of FDA meeting (April 25/26 2013) now available

The FDA public workshop on Drug Development for CFS and ME ended on Friday at 5pm and the video is already available. (Pretty good turnaround time!)

Video of the April 25/26 2013 FDA meeting is now archived and available for viewing.

http://www.tvworldwide.com/events/fda/130425/

(Scroll down on the right side under webcast archives.)

 

Thanks for eagle-eyed Nielk for spotting it! 🙂

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.

 

FDA meeting agenda and update on submitting comments – Spread the word please

Final agenda for FDA April 25/26 2013 Drug Development for ME and CFS public workshop

http://www.fda.gov/downloads/Drugs/NewsEvents/UCM344665.pdf

You have until 2 August to submit your comments to FDA via this docket – http://www.regulations.gov/#!submitComment;D=FDA-2012-N-0962-0004

Online comments are limited to 2000 characters however, you can attach documents and PDFs.

INTERESTING DEVELOPMENT as of 19 April 2013:

Comments and attachments can include YouTube links. For the severely ill this is a great way to be seen even though they can’t get to the meeting.

FDA wants your answers to these questions:

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.

 

 

Thank you to Ms Spotila for working with the FDA to get inclusion of YouTube links.

Among the group of 16

A few days ago the Federal Register had the announcement of the disease areas FDA will hold meeting about: “Prescription Drug User Fee Act Patient-Focused Drug Development; Announcement of Disease Areas for Meetings Conducted in Fiscal Years 2013-2015”

http://www.gpo.gov/fdsys/pkg/FR-2013-04-11/pdf/2013-08441.pdf

“…. FDA has selected the following diseases to be addressed in FY 2013-2015:

• Alpha-1 antitrypsin deficiency;
• breast cancer;
• chronic Chagas disease;
• female sexual dysfunction;
• fibromyalgia;
• hemophilia A, hemophilia B, von Willebrand disease, and other heritable bleeding disorders;
• HIV;
• idiopathic pulmonary fibrosis;
• irritable bowel syndrome, gastroparesis, and gastroesophageal reflux disease with persistent regurgitation symptoms on proton-pump inhibitors;
• lung cancer;
• myalgic encephalomyelitis/chronic fatigue syndrome;
• narcolepsy;
• neurological manifestations of inborn errors of metabolism;
• Parkinson’s disease and Huntington’s disease;
• pulmonary arterial hypertension; and
• sickle cell disease.  ….”

Remember that the FDA meeting on April 25/26 is the first of the meetings in this initiative. This is an impressive list of diseases and we are right in there!

We are setting the stage for the disease groups that come after us. Let’s shine like never before!

FDA has a Web site on Patient-Focused Drug Development: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm. This Web site contains the general schedule of upcoming meetings for FY 2013-2015, information on how stakeholders can prepare for upcoming meetings, and information on how stakeholders may leverage Patient-Focused Drug Development to generate input on disease areas not addressed through the Patient-Focused Drug Development PDUFA V commitment. The Web site will be updated as new information becomes available.

Web page for scheduled meetings: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm347317.htm

PDUFA 2012 slides includes info PFDD meetings http://www.fda.gov/downloads/AboutFDA/CentersOffices/CDER/UCM310754.pdf

More info from FDA about PFDD meetings:

Enhancing Benefit-Risk Assessment in Regulatory Decision-Making

Ensuring the safety, effectiveness and quality of human drugs is an increasingly complicated regulatory task, requiring FDA’s expert consideration of a multitude of complex factors. Over the past several years, FDA has developed an enhanced structured approach to benefit-risk assessment in regulatory decision-making for human drug and biologic products.

The Benefit-Risk Assessment Framework was developed through extensive review and analysis of previous and ongoing regulatory decisions. PDUFA V commitments include further development and implementation of the Framework into FDA’s human drug and biologic review process. Section 905 of the FDA Safety and Innovation Act also requires FDA to implement a structured benefit-risk framework in the new drug approval process.

In PDUFA V, FDA also committed to a new initiative called Patient-Focused Drug Development with the goal of obtaining the patient perspective on certain disease areas during the five year period of PDUFA V. Assessment of a product’s benefits and risks involves an analysis of the severity of the condition treated and the current treatment options available for the given disease. This information is a critical aspect of FDA’s decision-making as it establishes the context in which the regulatory decision is made. FDA believes that drug development and FDA’s review process could benefit from a more systematic and expansive approach to obtaining the patient perspective on disease severity and current available options in a therapeutic area.

This page contains information related to FDA’s implementation of the PDUFA V commitments regarding enhancing benefit-risk assessment in regulatory decision-making.

http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm

Comments can still be submitted to the FDA

Registration for the April 25/26^th FDA meeting has closed but you can still provide comments directly to the FDA

• 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.

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 8^th, 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

• The meeting will also be webcast at http://mecfsmeeting.eventbrite.com. See http://www.fda.gov/Drugs/NewsEvents/ucm319188.htmfor additional information about joining the meeting.

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.