In September 2007, a small test-of-concept efficacy trial
(STEP) of an HIV vaccine candidate developed by Merck was stopped early
for futility. Results from subsequent exploratory analyses of the
relatively small amount of data from this trial has had a profound
impact on the field of HIV vaccine research and development.
Specifically, a companion efficacy trial being conducted in Africa
(Phambili) of the Merck vaccine candidate was immediately stopped,
initiation of a large efficacy trial testing a different but related
vaccine candidate was stopped and a complete re-examination of the
scientific strategies and funding of HIV vaccine research was performed
by NIH. In this talk, I will provide a brief description of the study
design and present results from the primary, secondary and key
exploratory analyses of data from the STEP trial. I will then discuss
some of the decisions that were motivated by the STEP trial results and
describe the relative roles in these decisions of formal statistical
inference and of non-statistical judgments about generalizability of
results. Questions will be raised about the appropriate role of
statisticians in this process and potential implications for statistics
training programs.