RE:RE:RE:one missing item on websiteI agree with the efficacy data Beech is quoting. However, I am am reluctant to fault TST management for not promoting it aggressively. It may be a smart strategy on their part.
The reason has to do with statistics and how you do statistical tests on small sample sizes. TST is making their BLA submission to the FDA using the 129 people in the Phase 3a trial results. This avoids having to do an additional clinical trial with more people, thereby avoiding spending at least another $100m.
In statistics, every time you add a variable to your analysis, you shrink the sample size which you are studying.
For example, in Phase 3a, 18 people of the 129 had MCNA instilled immediately after TURBT, instead of waiting 30 days after surgury like BCG for healing to occur because of BCG's toxicity. These 18 were primarily patients of Canadian doctors because Canadian doctors had participated in the Phase 2 clinical trial, had experience with MCNA and were comfortable with it. Immediate instillation was not required by the trial protocol. According to the published data, these 18 showed 39% efficacy instead of the average 25% for the study. Mr. Morales gave a presentation a few years ago (in Philadelphia?) which discussed this. Moreover, the 18 people were a combination of papillary and in-situ. So if you looked at how the papillary people did with early instillation, it could be in the range of 45-50% efficacy. This level of detail was never published. However, I estimated a likely result in a post dated 11/9/2012 titled "Urocidin P3a Efficacy Table". (Post #31760827).
Its been years since I finished my university statistics course, but 18 people seems like a small sample size. There are ways to handle small sample sizes in statistics but they make it harder to say there is a real difference at a 95% confidence level.
It is not necessary for TST to make high efficacy claims to get FDA approval, since the only other approved treatment is so much lower than the 25% average for the total 129 person study. You will still sell the same amount of stuff if the FDA approves it.
Therefore, I think it is a smart stategy to promote what you can actually prove statistically at high confidence levels. The FDA would appreciate the consistency with what you say to them and what you say in your promotional material.