Email to Drs. Hamburg and Woodcock from a Physician

Posted by: Christine McSherry on March 10, 2014

There are many letters of support for accelerated approval of eteplirsen being sent to the FDA.  Here is one that I found interesting.

Email to Woodcock and Hamburg…

Dr. Woodcock,

I am a physician with about 35 years of medical practice experience (recently retired) and a Life Fellow of xxxx. I have communicated with you, and some/all of the DNP on 3 prior occasions. I have made a strong effort not to write another time. However, as time passes, it seems travesty is rapidly approaching farce; and your assurances that DMD is FDA’s ‘highest’ priority is sounding like spin—not verity.

My first letter was a five page data analysis of your departments’ shocking assertions in November about the clinical nature of DMD and expectations from ‘recent data’ and ‘steroids’, etc. The multiple articles I cited—all recent—clearly did not lend support to ANY of the objections, and in some cases, refuted them.

The second letter was after the December 12 meeting at which some of FDA personnel spoke. Several of the statements attributed to your personnel were hard to believe. They didn’t seem to exhibit any interval increase in understanding of the disease process from that disastrous beginning, or even indicate ANY consideration of anything but an old, fossilized approach to looking for clinical benefit in DMD. It was as if FDA-ISA doesn’t apply to DNP (Yes, I have read—in detail–that law). In that letter, I indicated that your DNP personnel somehow managed to be confused about things that were clear to most other clinicians who bothered to look at the literature—even this ob-gyn. I have, like most MD’s, reviewed thousands of medical articles throughout my career. Since data speaks for itself, I can’t fathom your personnel’s perplexity about the astounding significance of what is happening clinically with eteplirsen. That letter pointed out the linear downward course in DMD which, once begun–independent of group stats–is predictable and heretofore irreversible. I then pointed out the never-before-seen-in-history reversal of some of these boy’s declines.

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