Posts Tagged 'participation'

Connections, study contact info

One of the highlights of (most) meetings is the opportunity for connecting with people. Such was the case with the IACFS/ME conference held last week in San Francisco.

(There have been some excellent summaries of the IACFS/ME conference — among them: http://phoenixrising.me/archives/24323 , http://phoenixrising.me/archives/24390 , http://phoenixrising.me/archives/24452 , http://phoenixrising.me/archives/24522 , http://phoenixrising.me/archives/24553 , and http://quixoticmeblog.blogspot.com/2014/03/detailed-notes-from-all-4-days-of.html .)

Over and over people talked about how they had exchanged emails with this person or that person for years but had never met in person until this meeting.
ME is such a horribly isolating illness. Even caregivers are often isolated.

Therefore being able to meet other advocates in person, being able to exchange ideas with clinicians/researchers, etc. is exciting and invigorating (and exhausting).

Among the people I spoke with was Dr. Jim Baraniuk who has a study that will start soon (he did not say when). He provided the following contact info for it:

Exercise and Brain Scan Study
Purpose: to understand Chronic Fatigue Syndrome (CFS)
Who: Adults (over 18 years old) with and without CFS are invited to participate.

What: Subjects will have magnetic resonance imaging (MRI) brain scans then bicycle exercise tests.
They will stay overnight, then have a 2nd exercise test and MRI the next day.

Where: Clinical Research Unit, Georgetown University, Washington D.C.
Compensation for time and travel will be paid.

Contacts: Telephone 202-687-8231, FAX 202-687-9886

E-mail:cfsresearch@georgetown.edu or baraniuklab@gmail.com
Georgetown University Medical Center (edit *)

When you contact the lab you might ask (among other things) for clarification about the extent of the bicycle exercise testing, the criteria for patient selection, whether or not any cognitive testing will be done as well (if so, at which points during the study). Please share the responses you get and help us make them widely available.

Many of us came away from the IACFS/ME conference with information on numerous studies and avenues of exploration, getting lots of ideas, hearing hints about soon-to-be-published studies and great connections (some new ones, some long-standing ones but first time in-person meetings)…..

This was a real treat and I am deeply grateful to everyone who helped facilitate attendance at the meeting.

 

(*edited  – disclaimer – I have no connection to and accept no responsibility for the study or participation in it. Thanks for the reminder TK.)

I live in fear


(Permission to share)


 


Some ME(cfs) community members feel their voice is constantly threatened from within the community. This post (https://speakupaboutme.wordpress.com/2013/10/13/crimes-criminals-and-the-need-for-justice/) helped remind people that those of us in the ME(cfs) community are not “the enemy”. That reminder prompted one patient to ask that the following be posted:



I live in fear – all day every day.


It shouldn’t be this way but it is.


I live in fear of the long-term effects of ME/CFS and of being so severely sick starting at such a young age.


But this is not my only fear.


I live in fear of not being appropriately treated for my illness (or for additional illnesses) by medical professionals who disbelieve me and belittle me if I reveal that I have ME/CFS.


But this is not my only fear.


I live in fear of people finding out that I have ME/CFS and belittling the significance/severity/impact of ME/CFS.


But this is not my only fear.


With so much fear in my daily life, I would like to feel I get support within the ME/CFS community.




Yet even within the ME/CFS community


I live in fear.


I live in fear of adding my voice within the community.


I live in fear that if my views aren’t as outspoken as the views of others I will get shouted down – this is often done publicly, loudly, and vehemently.


I live in fear that I will be denounced by those who say (or imply) that any view that differs from theirs is worthless, is shameful and makes me an enemy.


I live in fear of getting involved – even though the community needs all the help it can get – because my voice will not be valued or respected, or even heard above the clamor of those who proclaim theirs is the one true voice.




I am not alone….


How many others are there like me in our community, who live in fear?


How many others are there who, like me, fear giving voice to their views?


How many others are there who, like me, fear getting involved in advocacy because they will be labeled, demeaned, belittled and demonized because of their views?”




We all want better lives for people with ME(cfs). We all deserve to be seen, to be heard and to be valued.




BUT


If only certain points of view are considered valid – we shut out many voices.


If we shut out many voices and viewpoints, we are also shutting out many potential advocates.


If we shut out many potential advocates, we limit the work we can do.




In supporting each other, in listening to and respecting differing views, more people will work with us because they feel respected, supported and valued.


With more people working with us, we can get more done.


With more people working with us, sharing the work that needs to be done, we might even have a little bit of energy left over at the end of the day. Wouldn’t that be nice?


 


 


 

 

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

 

Effective messages

A very worthwhile post at http://www.occupycfs.com/2013/06/25/precision-is-required/ highlights the importance of clarity, accuracy and effective messages in advocacy (applicable elsewhere also).

…Effective communication requires understanding the context and language of the audience. If we are imprecise or inaccurate, the audience will get distracted by our mistakes and lose sight of what we are actually trying to say. In turn, this can lead to the failure of the overall communication through misunderstanding or even outright dismissal. ….

It is worth the time and energy invested to craft a specific, accurate and strategic request that speaks to the context of the audience. …

We owe it to each other and the ME/CFS patient community to get things right. We must speak with precision and clarity about complex topics, while also presenting a strong case for what ME/CFS patients need. It is not an easy thing to do, but it is essential to our collective and ultimate success.”

Register for CFSAC May 2013 attendance and for public comment

The Spring 2013 CFSAC meeting will be held May 22–23, 2013

Registration is open:

http://www.blsmeetings.net/CFSACMay2013/

To register for public comment:

http://www.blsmeetings.net/CFSACPublicCommentMay2013/

 

The Federal Register notice for the May 2013 CFSAC meeting:

http://www.gpo.gov/fdsys/pkg/FR-2013-04-30/pdf/2013-10083.pdf

The meeting will be held at the Department of Health and Human Services; Hubert H. Humphrey Building; 200 Independence Avenue SW., Room 800; Washington, DC 2020

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