TELESTA THERAPEUTICS INC T.TST

RE:RE:Thoughts on why our phase II targeted a 40% efficacy....

[SteveMcM1]

Please see item #5 below, from the FDA panel on nmibu bladder cancer. The FDA felt that a single-arm trial would be sufficient for approval if a very high response rate was achieved....Telesta probably had no choice but to shoot for this target after the difficulty recruiting the Mitromycin control arm from the Urodicin trial. Just my thoughts, anyone think differently?
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Clinical Trial Design for the Development of New Therapies for Nonmuscle-invasive Bladder Cancer: Report of a Food and Drug Administration and American Urological Association Public Workshop

https://bit.ly/1SJq1wX

Objective

To summarize the discussion at a public workshop, cosponsored by the U.S. Food and Drug Administration (FDA) and the American Urological Association, reviewing potential trial designs for the development of new therapies for non–muscle-invasive bladder cancer (NMIBC). There have been only 3 drug approvals for NMIBC in the last 30 years, and product development for this disease has been stymied by difficulties in trial design and patient accrual.

Methods

A workshop evaluating potential trial design for the development of therapies for NMIBC was held in San Diego, CA, in May 2013. Invited experts representing all stakeholders, including urology, medical oncology, radiation oncology, industry, and patient advocates, discussed development of products for all risk strata of NMIBC.

Results:

Trial Design for Patients With BCG-refractory NMIBC

1) The panel defined BCG-refractory disease as patients who received 2 induction courses of BCG, induction plus maintenance (usually within 6 months), or were intolerant of BCG.5, 6 and 7

2) The panel could not agree on a standard of care for the treatment of these patients to use as a control arm but did agree that additional BCG is not appropriate.

3) There was broad consensus that a placebo arm was inappropriate for ethical and practical reasons.

4) There was discussion by the panel of the use of physician choice for a comparator in a randomized controlled trial in this patient population.

5) There was broad consensus by the panel that provided the results were robust, a single-arm trial could provide sufficient evidence of benefit. For patients with BCG-refractory CIS, the panel felt that an initial complete response rate of 40%-50% at 6 months and a durable response rate of at least 30% for 18-24 months with the lower bound of the 95% confidence interval excluding 20% could be clinically meaningful.8

6)There was discussion on how to handle a patient who recurred with low-grade papillary disease and whether to call them a failure without development of a consensus.

7) There was no discussion of what would be an acceptable level of response/recurrence-free interval for patients with BCG-refractory papillary disease without CIS.