Destined to fail, page-12

Without judging the trial investigators or the conditions imposed upon them by ethics committees, the standard of scientific reasoning here is impressive. The study protocol suggests an attempt to narrow the target population - without being so rigid (exclusive) as to prevent meaningful recruitment, or to prevent generalisations from study conclusions. Unfortunately, it may be that further debate is difficult without appreciating the detailed study methodology (hopefully disclosed within two months ... long before peer-reviewed publication).

The reasons for defining a hard (unambiguous) endpoint have been identified above. Given the law of diminishing returns in new therapies, many specialties (in developed countries) are at a level where only marginal improvements in outcome tend to be the rule, and dramatic improvements in outcome tend to be the exception. As a result of evidence-based medicine, the hurdle against which any new therapy is tested against becomes raised ever so slightly.

Given that these preliminary results are subject to interpretation and opinion, I think the most favourable result from here may be if a clinically significant advantage was observed locally (such that oncologists would have some justification to expand the indication for administration of an expensive new therapy). Conversely, I think a particularly disappointing result would be if only a statistically significant benefit was achieved in a particular subgroup.

Reassuring to see at least two colleagues with an interest in this company!