DNDN - CU Why is it an inconvenient idea? IMCL didn't bother with much of a CU program because they said they were using all they could make to run clinical trials and that it would delay Erbitux reaching the market if they had a large CU program. DNDN said at the advisory panel meeting that they didn't have a lot of cash to provide Provenge on a CU basis to everyone who might want it and wait several years for the results of the 9902b trial before getting approval.
I don't recall your complaining about IMCL having a minimal CU program before Erbitux was approved.
Of course, a compassionate use program is good for public relations if the drug works.
On a separate nore, people have discussed whether the "placebo" could be causing harm in an unknown fashion. Something that hasn't been brought up here, but was discussed at the advisory panel meeting, is that the actual composition of the treatment given to the patients is not well characterized and varies from patient to patient. DNDN does look at the APC cells given, but they don't fully characterize the other cells given in the treatment and what effect they may have.
The possible effects of treating the patients with frozen samples was mentioned. Jim Mule did ask if three treatments were needed.
There are still a lot of questions, but my impression was that the advisory panel voted favorably because they didn't want to choke off the immunotherapy approach. The small sample size and post hoc analysis was of concern to the advisory panel, and to several members of the audience. It will be interesting to see what the FDA decides.
I strongly feel that the 9902b results will be considered, but the FDA may allow limited marketing before those results are ready. This would be a form of post-marketing follow-up, which is a weak point for the FDA.
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Some other comments I have on the advisory panel meeting:
I don't think many on the panel were overly concerned about unknown toxicity twenty years out (especially when 36 months seems the average lifespan for these patients. Some of the patients were saying an extra 4 or 5 months would be great.)
One of the panel members said that if he had PC then he would take Provenge, even if he wasn't sure he could vote yes on its efficacy in the initial wording of question 8. When question 8 clarified, he switched his vote to yes.
One panel member said as a clinician seeing patients he would vote yes on 8, but when he put his scientist hat on he would vote no. He too switched to yes after the clarification.
Several members of th panel said that they did not want to stifle progress in a new approach in the field by voting no, even if it wasn't the most robust data package.
Several members in the audience were surprised at the yes vote, given the post hoc analysis ("cherry picking" the data), the small sample size, and the failure to meet the desired endpoints.
Let me repeat that the median time to progression was PRIOR to the second set of scans, which made it next to impossible for DNDN to demonstrate stat sig in TTP. Also, when the trial design was agreed to seven years ago, people didn't fully realize that TTP didn't really correlate with survival, at least not for Provenge.
I think this sort of active immunotherapy involving ex vivo manipulation of the patients cells really is an apples and orange comparison to the "normal" chemotherapy cancer drugs, or even biologic cancer drugs, so does belong in CBER rather than CDER. It's much more a question of immunology and understanding cells than it is a matter of PK/PD data.
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