Peter Thall writes:
Some years ago, after I gave a talk at Columbia that you attended, you told me that you would like to see a decision-theoretic analysis formulated and carried out to completion by Donald Berry, who had been quite vocal for some time about the importance of such a “fully Bayesian” analysis.
I do not work with Berry. But, in recent years I have begun to do utility-based clinical trial design. The trial described in the attached paper [by Thall and Hoang Nguyen] enrolled its first child very recently. While the methodology is not terribly sophisticated, I consider this to be one of the most ethical trials that I have designed. The utility was elicited from the two trial PIs. When we had the pre-trial start-up meeting, a third oncologist looked hard at the utility table, and he said that he agreed completely with the numerical utilities.
I doubt that this trial will cure this type of brain tumor, but I do think that the design gives the children a better chance than they would get otherwise.
Still, I think we can improve this methodology by adding some refinements, and we are working on them now.
I indeed have been long interested in seeing a formal analysis balancing the goals of medical research, reducing future mortality and morbidity, saving cost, and reducing the risk to experimental subjects. The risks to present patients are balanced by potential future gains for the lifetime of the new procedure. But these tradeoffs always seem to be implicit. It’s hard to find an example where the costs and benefits are quantified. So I appreciate that Thall sent me this note.