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FDA hearing 29/30 Jun 2010 on Orphan Drugs
 Moderated by: Prof Trevor Marshall
 

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Prof Trevor Marshall
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 Posted: Tue Jul 6th, 2010 11:41

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Last week the FDA held a Public Hearing to examine weaknesses, and accept suggestions, related to the way that FDA currently processes applications for drug approvals in the rare (orphan) diseases. Amy and I both traveled to FDA White Oak for the hearing.

Over the past five years, this Foundation has applied for orphan designations in Sarcoidosis, PTLDS, Multiple Sclerosis  and ALS, with some limited success.

My presentation details the troubles we have had, and points to 'old science' as being the primary hindrance to our pathway forward. FDA reviewers are still stuck in the 20th Century. But I guess we already knew that :)

Anyway, Janet and Chris have helped me put together a webpage which you can refer your friends to, urging support of CureTheProcess.org

We are also working on more web-pages, which describe the successes our members have had treating MS and Sarcoidosis with Olmesartan. Later we will extend this initiative with pages on CFS, arthritis, and all the other major Th1 indications.

Here is the new page. It includes video of my presentation, and also of Mary Pendergast, a past Deputy-Commissioner of the FDA :)

http://www.autoimmunityresearch.org/MS/

We have decided to move forward to the IND process, and leave the folk at OOPD behind us until we have more data in our hands :) We met lots of important and helpful people while we were at the hearings, and have been very busy following up all the new avenues which opened up for us :)

..Trevor..
 

Grateful Survivor
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 Posted: Tue Jul 6th, 2010 15:25

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I found your presentation clear and irrefutable... but I'm sure I would have granted rather than rejected the unsuccessful applications, too, as full of promise--if the desired end result is a cure for people rather than an improvement in blood work for mice. It sounds as if the decision to pursue the IND (Investigational New Drug) process instead may be more "promising." :-) Thank your for your continuing efforts to move forward through the muck of late 19th and 20th century leftover thinking. Gratefully, Dody



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Freddie Ash
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 Posted: Tue Jul 6th, 2010 15:42

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HI DR MARSHALL

This is Fred in WV.  Thank you and Amy for going to the FDA and standing up for all of us here with all these diseases.  We can never repay you for all you have done for us.  YEA!! For Dr Marshall and his staff here.

Remember, we are all in this together and I am pulling for us.

Your friend in Sarcoidosis
Freddie



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Prof Trevor Marshall
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 Posted: Tue Jul 6th, 2010 20:16

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 Dr Emil Kakkis'  presentation, describing why we need to "CureTheProcess," is now online at URL:

http://www.youtube.com/watch?v=IDTqX0fpKWs

I have also integrated it into the webpage at
http://AutoimmunityResearch.org/MS
 

ChrisMavo
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 Posted: Wed Jul 7th, 2010 10:46

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Dr Marshall, very good presentation!  Let's hope you and the others who presented can get the FDA to change it's procedures for approving drugs to be used in treatment of rare terminal illnesses.  Why they would not approve the orphan use of olmesartan for ALS, a disease with NO current treatments, is frustrating to say the least!! 

Is there a presentation by Amy in the works ... or did she just assist in your presentation? 

Thanks for ALL you do for ALL of us! 



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Prof Trevor Marshall
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 Posted: Wed Jul 7th, 2010 10:53

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Chris,
Amy focused on helping me talk with the people who were there. People from other foundations, from PhRMA, and from the FDA. There was a lot of talking to do, and very few breaks to do it in.

There are some very good people in FDA, and some who just see their job as administering regulations. Our job is to find more of the good people, and help them understand why killer diseases, with no effective therapies, need especial help :)
 

Deedee
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 Posted: Thu Jul 8th, 2010 06:29

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Thanks for posting. The problem reminds me of the description of the problem in this 2010 Newsweek article, Desperately Seeking Cures: http://www.newsweek.com/2010/05/15/desperately-seeking-cures.html

Excerpt:
More and more policymakers and patients are therefore asking, where are the cures? The answer is that potential cures, or at least treatments, are stuck in the chasm between a scientific discovery and the doctor’s office: what’s been called the valley of death.

Perhaps now is the right time for change.



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Freddie Ash
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 Posted: Thu Jul 8th, 2010 08:13

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HI DEEDEE

This is Fred in WV.  Your post reminds me of my story.  When I was first diagnsised with the sarc and I found out I could not find a cure for a disease.  So one day not too long ago I ask one of my doctors to name one disese that they had a cure for and he did not have any answer.  The Marshall Protocol is the only treatment out there that has a cure in it.  So once again, Yea! for the Dr Marshall and the Marshall Protocol, a first???

Remember, we are all in this together and I am pulling for us.

Your friend in Sarcoidosis
Freddie



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Sunset
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 Posted: Sun Jul 11th, 2010 14:16

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Freddie, I had a similar experience with a nurse practitioner back in 2003. She told me that legally she could not tell any patient that any treatment she recommended would produce a cure for any disease. I stopped seeing her as a patient.

Unfortunately that is the mentality of many health providers these days (think "defensive medicine"); they are unwilling to offer 'cures' to patients for fear of being sued if the treatment does not result in a cure for the disease treated.

Best regards,
Sunset



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eClaire
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 Posted: Sun Jul 18th, 2010 11:14

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One can only hope that the people who review Orphan Drug status can also hear valid, constructive criticism... since it appears that they were in charge of the review of the review process.

Thanks for your efforts Dr. Marshall, given the responses to your applications, it is easy to understand the frustration you must be feeling with this process.

Claire



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 Posted: Sun Jul 18th, 2010 14:07

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I enjoyed the video and the thoughtful content to the FDA.

Come what may, I am firmly convinced the MP's thesis is correct.  The "society of white coats" will at some point be as embarassed as civil war surgeons. 

Our democracy is slow to pick up on this, but stay tuned.

Sherry 

 



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Deedee
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 Posted: Thu Aug 5th, 2010 04:34

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CURETHEPROCESS™ UPDATES

A Great Win for Rare Diseases in U.S. Senate Appropriation Bill
The FY 2011 Agriculture, FDA, and Rural Development Appropriations Bill (S. 3606 Section 741) has been introduced and includes language supporting two of our CURETHEPROCESS Campaign's goals. Specifically, the Bill supports the creation of new guidances that could improve the scientifically sound use of surrogate endpoints and new clinical study designs and analysis. The language builds on the Brownback/Brown Amendment language that was included in the FY 2010 Appropriations Bill (Section 740). The Bill includes specific funding for the Office of the Associate Director for Rare Diseases in the Center for Drug Evaluation and Research (CDER). Funding for this office is increased by $1,000,000 to hire additional staff with specific expertise in facilitating the development and review of products to treat rare diseases. The Bill also includes the first increase for the Orphan Product Development Grant program since FY 2005. The program is increased by $2,000,000 for a total grant level of $16,035,000. We are especially grateful to Senator Sam Brownback (R-KS) for his leadership on this issue and to Senator Herb Kohl (D-WI) for his support. Click here to read the press release.

http://www.kakkis.org/Documents/press_release_7_15_10_senate_bill_final.pdf



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Marysue
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 Posted: Sat Aug 7th, 2010 19:16

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Thanks for sharing Deedee. Could someone tell me what the term "surrogate endpoints" means?
And also how we fit into the category of "rare diseases"?
:)
Marysue



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 Posted: Sat Aug 7th, 2010 22:12

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There might be something in here to help with understanding:

Video: Ms Mary Pendergast's submission to FDA Brown/Brownback Hearing

Summary:

On 29/30 June 2010 the US FDA held a Public Hearing on how to improve the availability and processing of applications for drugs in the rare diseases (called 'orphan drugs'). This hearing was mandated by the amendment moved by Senators Brown and Brownback to the 2010 FDA Appropriations Bill.

This is the submission made by Ms Mary Pendergast, JD, President of Pendergast Consulting, who held senior positions in FDA for 33 years. She explains how Congress has already given the FDA flexibility to approve drugs for needy groups of patients, bypassing much of the paperwork, delay and expense required by current FDA management.



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Prof Trevor Marshall
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 Posted: Sat Aug 7th, 2010 23:07

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A surrogate endpoint would be, for example, the appearance of Immunopathology. It could be considered as a surrogate predictor for cure, as the endpoint "cure" might be harder to measure and take longer to demonstrate. Therefore IP could be a surrogate endpoint to indicate the likelihood of eventual success :)
 

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 Posted: Sun Aug 8th, 2010 02:38

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Is immunopathology, or herxheimer, an accepted concept in the medical community?

Last edited on Sun Aug 8th, 2010 06:12 by positiveminds



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 Posted: Sun Aug 8th, 2010 10:39

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In my opinion, not yet.  But stay tuned...

Sherry



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D25, Total: 12 measured 11/3/15 Started MP=01/04/05 Diagnosis: Sarc 12/04; "cat scratch disease" or necrotizing graunulomas 10/88; Raynaud's (diagnosed 1980?)
Prof Trevor Marshall
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 Posted: Sun Aug 8th, 2010 12:04

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positiveminds wrote: Is immunopathology, or herxheimer, an accepted concept in the medical community?
We are the first to reliably induce immunopathology in chronic disease. Others do use the term, but they do not understand the potential intensity of IP, and the problems it might cause.
 

Deedee
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 Posted: Sun Sep 19th, 2010 08:46

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The cruelty of rigid requirements for clinical trials. Relevant to the discussion on how the FDA rules need to change:

http://www.nytimes.com/2010/09/19/health/research/19trial.html?_r=1&hp

Excerpts from article in today's NY Times:


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“With chemotherapy, you’re subjecting patients to a toxic treatment, and the response rates are much lower, so it’s important to answer ‘Are you really helping the patient?’ ” said Dr. Charles L. Sawyers, chairman of human oncology at Sloan-Kettering. “But with these drugs that have minimal side effects and dramatic response rates, where we understand the biology, I wonder, why do we have to be so rigorous? This could be one of those defining cases that says, ‘Look, our system has to change.’

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But with the trial now under way, a few attending the Las Vegas meeting had already had to tell patients they had been assigned to the trial’s chemotherapy control group. And some had begun to question whether an ethical code that calls for doctors to be genuinely uncertain about which of a trial’s treatments will be more effective had been breached when it came to PLX4032 versus dacarbazine.

After Dr. Chapman presented the recent data from the drug’s promising first trial to a packed room, Dr. Neal Rosen, a friend and Sloan-Kettering colleague, stood up.

“Excuse me,” Dr. Rosen said with unusual formality. “But if it was your life on the line, Doctor, would you take dacarbazine?”

The room was silent.

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Several of the most veteran melanoma doctors agreed with him. But others argued that oncologists had an ethical obligation to push both the F.D.A. and Roche to make the drug more immediately available.

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“It’s much easier to tell patients, ‘We’ll try this for six weeks; if it’s working, great, if not, we’ll shift you right away to the other trial,” said Dr. Jeffrey A. Sosman of the Vanderbilt-Ingram Cancer Center in Nashville. “That’s how I’m going to be able to live with the randomization.



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06/08 Sarc in lymph nodes. Chronic high lipids. 2010 heath cat scan-zero plaque. 11/12 D8, 12/12 Normal CXR except scarred lymph node, normal PFTs 8/12 Off all MP meds. Metabolic Syndrome

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