DrPH Dissertation Research in Rare Diseases?

Last week I had an opportunity to talk with Dr. Glenna Crooks, former Chairman of the National Commission on Rare Diseases, and recipient of the Congressional Exemplary Service Award for Orphan Products Development. I also met with Diane Dorman, NORD’s Vice President for Public Policy.

I am considering potential topics in the realm of rare diseases for a DrPH dissertation.

Here’s some of the topics I am starting to explore in more depth based on those discussions:

(1) HR 2538 – Undiagnosed Diseases Registry Act of 2009 – Feasible? what about Centres of Expertise and European Reference Networks (more for pediatrics surveillance)?
(2) Understanding the pathogenesis of rare diseases: relevance in increasing the understanding of more common medical disorders; translational clinical research.
(3) Tax Credit, Market Exclusivity, Incentive vs Removing Disincentive for Orphan Drug Development, Creating Parity
(4) Patient-Centric models for Rare Disease care – where are the regulatory barriers? Risk taking (ie patients setting up their own clinical trials @ patientslikeme.com). Are sites like Inspire and Rareshare having any impact?
(5) Cross-border collaboration on drug development… Eurodis; French-National Plan for Rare Diseases.
(6) Analytical Comparison – What if “200,000” were “100,000” or “1 million”? – More drugs be available? Less? Would clinicians be doing more education/diagnostic efforts/surveillance?  Europe – 1 in 2,000 citizens.
(7) What’s happening over at the NIH Therapeutics for Rare and Neglected Diseases Program and the Undiagnosed Diseases Program?

What are your thoughts? Share your experience with me and comment.

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~ by marbleroad on June 8, 2009.

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