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Biotech / Medical : Biotech Valuation -- Ignore unavailable to you. Want to Upgrade?

To: John Metcalf who wrote (7623)	12/28/2002 11:58:29 AM
From: The Dodgy Ticker	Respond to of 52153

John, Here is a "Letter to the Editor" in the WSJ (either last Wed. or Thurs.) from Lester M. Crawford, Deputy Commissioner of the FDA re The FDA's policy on experimental drugs: Title: The FDA's True Policy on Experimental Drugs" Cancer patient Edie Bacon's Nov. 29 editorial-page essay, and the Dec. 6 Letters reacting to it, about her inability to obtain a potentially life-saving experimental drug were poignant, and Ms. Bacon's deep concern about her lack of access to a treatment that she believes to be beneficial is understandable. The policies of the FDA, however, are not an obstacle to the use of experimental treatments outside clinical trials. Such use would not prompt, as Ms. Bacon appears to believe, an FDA request for additional studies. The FDA, categorically, does not attach special significance to adverse events reported from such expanded access programs as Ms. Bacon has tried to join. We recognize that these programs involve less-controlled use of new drugs, and we assess the reported data accordingly. The development of a new medication is not slowed by side effects occurring outside clinical trials. The FDA strongly supports expanded use of promising therapies for serious and otherwise untreatable diseases. In the past two years alone, 10,000 patients with myeloid leukemia have been given access to Gleevec; Iressa, a medication that's currently being reviewed for lung cancer, has been made available to 15,000 patients. So far, 67 patients have been given outside-trial access to ET-743, the drug that Ms. Bacon wants to use. The FDA encourages patients to participate, when appropriate, in well-designed clinical trials. Information about studies is available at clinicaltrials.gov. To obtain an experimental drug under expanded access, patients should first contact the manufacturer. If the result is unsatisfactory, our Office of Special Health Issues (301/827-4460) can often provide helpful information about access to experimental drugs. Best wishes to all for the New Year, Bob

To: John Metcalf who wrote (7623)	12/28/2002 1:50:41 PM
From: Miljenko Zuanic	Respond to of 52153

They are partially correct, but very subjective in criticizing the FDA. DNDN first hormone refractory prostate PIII trial had large pts population with Gleason score >8, so trial with sub-group analysis was too small for clear evidence of drug benefit. <<This study contained a disproportionately high number of men with a Gleason score of 8 or above, which contributed to the results we saw with our interim analysis in January. After adjustment for these patients, the study yielded a significant therapeutic effect for Provenge.">> Second, they have problem with vaccine manufacturing issue, and a second PIII trial was put on hold. This was cleared with FDA. Third, they amendment second PIII protocol (this month), where pts only with score of <7 will be allowed. So, FDA was not too harsh on them. Initial sloppy work in trial designee and issue on vaccine cellular composition was main factors to blame for delay, not FDA approach toward new drugs. If they confirm results with second PIII trial FDA will be on their side, IMO. Miljenko PS: BTW, I am not sure that placebo as control arm is right choice here??? Happy and Prosperous New Year to all!

To: John Metcalf who wrote (7623)	12/28/2002 5:59:49 PM
From: A.J. Mullen	Respond to of 52153

You see, when Dendreon analyzed the entire population in the study -- i.e. patients with any and all Gleason scores -- they could not tell the FDA with better than 95% confidence the treatment effects shown by Provenge were due to the drug and not random chance. All they could manage was 94% confidence. To be precise, the 95% confidence refers to the probability that one would attain differences in the statistics between the control and experimental as great as those found if the protocol had no effect. 95% is arbitrary, but customary. Using it is accepting that there is a one in twenty chance that a drug will be disgarded for having no significant effect when in fact more testing would show that it did have such an effect. If there had been one more subject in the experiment the "% confidence " would have been greater or less than 94%. Perhaps it would have been above the criterion, but it's just as likely that the margin of failure would have been greater. The time to set boundaries of acceptability is before the experiment. The company would have had the opportunity to argue that less stringent conditions should apply before agreeing on the protocol. They could also have included more subjects which would have made the experiment more robust. It is totally spurious to take a sub-group for which the numbers look good and say that these pass the criterion. If the group between two and seven on the Gleason scale had not "qualified" would they have partitioned the group further? Anyone can see the ridiculousness of claiming, "for patients at 2, 5 & 7 on the Gleason scale we have proved that the difference to those to whom we gave the drug is greater than would be expected by chance." What the company was trying on is almost as shaky. Ashley