Did I miss Spring? I spent the last week of May in Washington, DC. I had meetings on Monday and Tuesday centered around regulatory issues (FDA and OOPD), compounds, small clinical trials, webinars, and workshops in the planning stages and tossing around some ideas about how to design new models for trials, or how we might encourage companies to think a bit outside the box. Discussions like this are like taking a breath of fresh air into your lungs. I find them invigorating, hopeful, and promising, incredibly thankful to see the results of our early investments in research now move from the bench to the bedside . On the other side, I realize nothing will ever move fast enough for our boys.

By midweek, the conversation changed slightly to include the range of interest, opportunities, and programs focused around Rare Diseases (R.A.R.E, Every Life Foundation, Rare Disease Caucus, GrandRx, Cure the Process, etc), how the Rare Disease Community is focused on developing tools to streamline the drug development process for rare disease and the worldwide focus on RARE.

At the end of the week, I participated in my last face-to-face meeting with the Institute of Medicine Committee. I have been serving on this committee for the last year. While some of the committee members knew each other in a past life, the charge of this committee was new and quite daunting. We were charged with writing a comprehensive paper on Accelerating Rare Disease Research and Product Development in Rare Diseases and writing recommendations that are expected to result in legislation. This experience has been intimidating, amazing, and wonderful all at the same time. The committee is comprised of individuals representing a broad range of expertise –academia (clinical trial experts, epidemiology, rare disease), researchers, venture capital, drug/device executives, attorneys, and foundations. This last meeting was bittersweet. We came to the end of our investigation, reporting progress to date and making recommendations for changes that we hope will positively impact the trajectory of treatments and cures. For me, it was a incredible learning experience, to systematically go through what we now know about rare disease, what we need to know, what tools need to be developed, what processes need to be streamlined, and where resources would be useful. What was even more moving was to understand that each member of the committee had been impacted by a rare disease, that each member understood how rare disease isolates, how rare disease impacts individuals, and how we, as a country might work together to break barriers that get in the way.

The report is expected to be published this Fall.

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Comment by RAKTIM SINGH on June 11, 2010 at 9:49am
Thanks Pat. We all appreciate your efforts and dedication in finding a cure for us.The road to cure is full of hurdles and you are clearing of those hurdles.
Comment by cheryl cliff on June 10, 2010 at 4:59pm
I can't imagine being in your place Pat. So many difficult and extremely important elements to disect and reassemble. I would find myself completly intimadated and with eyes glazed over. You however, are well equipped and can (already have) handle this with grace. The report must have been huge in terms of thinking thru every aspect of rare diseases. Go getum Pat! I appreciate you and all you are doing to kill Duchenne. So will Alexander when he is older and can comprehend dmd better. THANKS

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