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Sample letter for FDA 'folks'.... A big 'huh?' for me from the FDA response is that they act as if 'robust' dystrophin is the only basis for NDA/approval. Unless the company has put all its apples in the proverbial one basket, it seems to me that the best case for ETEP (as opposed to a class approval track) is in the clinical data. I've had multiple communications with(to) the DNP folks and especially Dr Woodcock with the latest being yesterday AM. As I don't want to come across as banned from such in future, I hesitate to write another (intense) email one day later! However, I think this is a good case to be made and anyone can feel free to copy/paste, modify,, start an email campaign with, etc in his/her own communication to the FDA officials (or other policy makers) if so desired. _________
Dear_____,
I read with some perplexity the FDA's response to Sarepta's announcement of a delay in filing an NDA, and the apparent noisy response it excited.
My perplexity is that it appears that FDA is/has been ONLY considering an NDA based on a proof of sufficiency of dystrophin production--a still open question, per your position. However, for eteplirsen, it seems the strongest basis, which seems to me unequivocal, is the actual clinical benefit shown in the boys themselves. This is true from some boys' actual improvement from a line of decline which had already begun; others with stabilization though previously on the decline line; others with apparent decrease in the slope of such decline. In addition, any apples-to-apples comparison with natural history studies show that as a group, a significant and beneficial effect from eteplirsen is the best, if not, only explanation for the boys' performances.
From group-level data, with the eteplirsen boys baselined at 1 test before presence of dystrophin demonstrated:
(Per choice of baseline date: Before study began it was not known how long, if at all, it would take for etep to begin a detectable effect. Since this data, it seems evident that is at least MORE than 3 mo, and UP TO 6 mo before such effect begins. In the interim, the boys essentially continue to be in a 'natural history' state.) The FDA has also asked the company to get patient-level data, for more direct matched subgroup comparison, I suppose. The only data set published from which such patient level data can be extracted is the McDonald paper on the Ataluren placebo group. Extracting such data for a matching subgroup for the eteplirsen placebo/delayed treatment group yields this: (Most slides have grouped the PBO/delayed group in a comparison with the over 350m/over 7yr natural history. However, by the time rx was 'on board' this group, n=4, had a mean of 317m 6MWD--clearly in a higher risk category. This is a matched comparison for such a subgroup) lllll Seeing the above, and understanding that many of the families of the boys are actually living with, and seeing such stability/improvement every day, it is no wonder that they express confusion and strong frustration in dealing with the bureaucratic processes which actually do seem to miss something obvious. Add to this the fact that in multiple patients there have been demonstrated NO significant adverse reactions, and the Benefit/Risk ratio seems to be screaming "What are they waiting for!!!" Indeed, what ARE you waiting for. "Robust" what? Sincerely,, |
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Msg # | Subject | Author | Recs | Date Posted |
10529 | Re: Sample letter for FDA 'folks'.... | clarksterh | 1 | 10/31/2014 12:36:26 PM |
10534 | Re: Sample letter for FDA 'folks'.... | ru.empeirikos | 0 | 10/31/2014 6:13:12 PM |
10537 | Re: Sample letter for FDA 'folks'.... | pineappleguava | 0 | 11/1/2014 11:27:38 AM |