I met our rare disease campaign Change.org team a little over a week ago (by telephone). They sound great – Matt, Robin, Josie and Maria.
I’ve been chatting with rare disease community folks getting thoughts on the campaign – Sharon Terry, Emil Kakkis, Chris Austin and Brad Margus.
I’ve been reading lots of different things just so I can understand the whole drug development process better – all the pieces and parts. If you have a minute, check some out:
Bench to Bedside (by Sharon Begley), & Where are the Cures? (also by Sharon Begley)
Biotech Needs (by Richard Gayle), & Health Care Reform and Biotech (also by Richard Gayle)
Savior Parents (by Elizabeth Weill), & Homegrown Miracles (by Mary Carmichael)
Issue Brief: Investing in Innovative Medical Research (by Eric Kessler and Margaret Anderson)
What do leaders of disease-specific advocacy organizations know about pharmacogenomics and biomarkers, anyway? (by Sharon Terry, subscription/fee)
Tool in Cystic Fibrosis Fight: A Registry (by Milt Freudenheim), & Research Trove (by Sarah Arnquist)
Listen to a great webinar here (Developing Cures with Less Time and Capital), transcript and slides available. I was really excited to listen and read through these materials. I sent “Where Are the Cures” an email on the model discussed in the webinar as applied specifically to rare disease. A quick reply: “There are no particular limitations of the ARC model for rare diseases. But one of the challenges with rare diseases is that in some cases there is so little basic science that it could be difficult to choose a narrow focus for the research.”
I had forgotten as I gathered all these different materials together that the article by Mary Carmichael really got under my skin awhile back. She writes in Homegrown Miracles: “it’s not always a good idea to try and speed up science. There’s a reason for all that protracted, agonizingly meticulous work that medical researchers do: most of the time, it’s necessary.”
I shot off an email: “The deal with rare disease is this – no parent should ever assume that someone is ‘working’ on the disease, my son has a crippling and painful bone disease (he is 7), sometimes he can’t move for pain (again, he is 7), do you know how many researchers and docs are working on this?  That would be zero.  Most parents are so beaten down they can’t even summon the energy to care.”
I know, I know, kind of hot under the collar there (no surprise that I got no quick reply). And to think one of the reasons I went to law school was because I hoped it would toughen me up (as a kid I cried when kids called me “freckle face”, I was a super softie).
[We do have researchers and doctors working on other parts of the disease but no pain experts, yet.] I am still surprised when I think back to several years ago and my assumption that of course someone is working on these things related to my son’s disease. I kind of miss that naïve hope. How ‘bout I replace it with informed and empowered hope instead? Our squeaking into the “Top 10 Ideas” gives me that hope.
I will keep the Change campaign updates coming.
:: Catherine Calhoun
Thoughts on Change campaign?
(e) hellocatcal AT gmail DOT com
(t) @supercatcalhoun
(Pretty nerdy huh?)

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