I spent the better part of the week in Washington, DC with a full agenda. On Monday, I met with Peter Saltonstall (president of NORD). One of NORD’s major interests is the FDA. You are probably familiar with our advocacy agenda, this year concentrating on several areas relevant to clinical trials – one very specifically directed toward FDA. One of our requests focused on securing additional resources for the FDA, specifically more expertise and experience on rare disease (Duchenne), communication, and collaboration with the orphan products division and efforts to harmonize interaction between EMEA (European regulatory agencies) and FDA. NORD is focused on the same issues and when voices join together with specific messages, things happen. Anne Pariser, MD is now the Associate Director of Rare Disease, a new position with FDA.

On Monday evening, I served on a NORD panel to discuss barriers facing therapies for rare diseases. I spent some time discussing barriers such as risk benefit (such as asking for definitions of risk when the condition is fatal) and discussing TIME (how minutes matter when, in progressive conditions, each minute represents cell degeneration and death). Time. Loss of function. Lost time with someone you love. And steady state. How many times have we all prayed, that Duchenne stops now. Today. Steady state, no further degeneration.

On Tuesday I met with Anne Pariser. Anne has worked for the FDA for over 10 years, working in the area of inborn errors of metabolism. She is wonderful and anxious to accelerate therapies for rare disease. We discussed exon skipping and meetings planned for Fall. And I took a left turn in the conversation. You may already be aware, NIH (NINDS/John Porter/Berch Griggs/Katie Bushby-Treat NMD) have been awarded a
grant for a multi-site steroid trial. The trial will have several arms, evaluating an intermittent regimen with daily
Deflazacort and daily Prednisone. Makes no sense to me unless, at the end of the day, should the data suggest Deflazacort has less side effects (weight gain/behavior) it would be available (and covered by insurance) for families in the US. Anne said she had no knowledge of the trial. Now she does. And John Porter is connected to Dr. Pariser. And those planning the trial are connected to Dr. Pariser and to Elizabeth McNeil (orphan products). One of the goals of this study will be to (FINALLY) have Deflazacort available and approved in the US and hopefully elsewhere. Families deserve choices…and trials must consider access to whatever they are testing. Families already have significant financial burden in Duchenne. ALL therapies must be available and ALL boys must have access without additional expense.

Wednesday and Thursday I was in closed meetings with the Institute of Medicine (IOM). Our task is to produce a report (Fall, 2010) on “Accelerating Rare Disease Research and Orphan Product Development.” The report will include discussions and recommendations on discovery, drug development, FDA, the Clinical trial process, insurance, Medicare/Medicaid and devices. You might be aware of Senator Spector’s CAN legislation – $500 million for rare disease. Cross fingers and pray this legislation becomes law.


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Comment by Pat Furlong on April 28, 2010 at 8:11am
Senator Specter has always been a champion for NIH. and the CAN Legislation within the Healthcare Reform is amazing (though the request is subject to change with budget constraints) and the IOM report will be released in September -keeping in mind the task of the IOM is 'accelerating research and product development for rare diseases' . The stars lining up for rare disease...
Comment by Keith Van Houten on April 27, 2010 at 9:12pm
Awesome. I heard zero about that during the health care debate. Specter sure has made some things happen. He was a big part of the $10B for NIH in the stimulus bill too.

Comment by Pat Furlong on April 26, 2010 at 7:51pm
Nature Medicine: ‘Cure Acceleration’ Funds Woven Into Health Reform Legislation
Friday, February 5th, 2010
An article by Meredith Wadman in the February 2010 issue of Nature Medicine explains an amendment to the health care reform bill inserted by Senator Arlen Specter which would create the Cures Acceleration Network (CAN). The network, managed by the National Institutes of Health director and “a board of 24 experts drawn from academia, venture capital firms, government agencies and disease advocacy groups,” would award grants to facilitate translational research. Research!America President Mary Woolley is quoted in the article:

Comment by Pat Furlong on April 26, 2010 at 7:46pm
There is 'cure' legislation wrapped into the healthcare reform. This is Sen. Specter's approps. for 500M for rare disease research.
Comment by Keith Van Houten on April 26, 2010 at 6:18pm
Pat - I can't find Spector's bill you refer to. Has it been introduced, and what's the bill number?
Comment by cheryl cliff on April 20, 2010 at 9:55am
Glad you are in the right place - and at the right time Pat. Especially now Washington needs to hear our voices. Sounds like things are progressing nicely!

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