Interim Data Can Bias End Results Itchy as we all are for Tefina results, this is likely a good decision. 'Futility studies' are generally done to prevent patients from being given an ineffective treatment, to stop ineffective trials early and to save cash. Tefina is not targeting a terminal illness here.
In the case of Tefina there is a big risk interim data could flaw the results of the full trial and weaken confidence in the final conclusions (see FDA guidelines below). Positive early results for a 'female viagra' would go viral and could easily be seen by patients taking part in the study as well as investigator staff, introducing bias. What we do not need is the FDA asking Trimel to repeat the Phase 2 trial!
Summary:
Trimel seems to be trying to provide information to shareholders as expediently as possible. It will be interesting to hear what additional info is provided in the early conference call.
Normal cash burn is down and with $24 million cash at end of September, there is no early need for additional financing. The bulk of expenses for the Tefina study will have been made upfront and Compleo isn't costing much at this point.
Tefina is >75% enrolled; and as promised previously, treatment sites have increased - up 50% from 32 to 48. Increased recruitment efforts appear to be successful and full enrollment is expected to be achieved before the year end. They are not going to do an interim so close to completion.
They have sent an answer re Compleo to the FDA promptly - Nov 1; they are sticking with the original NDA (which could mean they believe they have defended well).
From FDA Guidelines on Interim Trial Analysis:
"Futility analyses could stop some trials with no benefit, but not all. After observing 50% of the target number of events, 3 out of 5 trials with no benefit could be stopped early (low CP ≤ 15%). Trial duration for two studies could be reduced by 4-24 months, saving £44 000-231 000, but the third had already stopped recruiting, hence no savings were made. However, of concern was that 2 of the 4 trials with moderate treatment effects could be stopped early at some point, although they eventually showed worthwhile benefits.
Careful application of futility can lead to future patients in a trial not being given an ineffective treatment, and should therefore be used more often. A secondary consideration is that it could shorten trial duration and reduce costs. However, studies with modest treatment effects could be inappropriately stopped early. Unless there is very good evidence for futility, it is often best to continue to the planned end.
Any interim analysis that is not planned appropriately (with or without the consequences of stopping the trial early) may flaw the results of a trial and possibly weaken confidence inthe conclusions drawn. Therefore, such analyses should be avoided. If unplanned interim analysis is conducted, the clinical study report should explain why it was necessary and the degree to which blindness had to be broken, and provide an assessment of the potential magnitude of bias introduced and the impact on the interpretation of the results."