Safety decision-making with multiple sources and different types of studies—recent examples from FDA Advisory Committees

Randomized controlled trials including superiority and non-inferiority trials, meta-analyses, re-analysis of controlled trials, and various types of observational studies may each contribute information to the knowledge of the safety of a drug. FDA and FDA Advisory Committees have had to weigh the evidence from such studies and either reach a regulatory decision or advise on one. Each of the types of studies has advantages and disadvantages, including size, unbiasness, generalizability, and feasibility. Some common principles of study quality such as the use of prospective analysis plans and rigorous validated endpoints apply to each of the types of studies. This talk presents recent examples from FDA Advisory Committees of the setting of multiple and diverse studies of a drug safety issue. In particular, this talk discusses the 2010 rosiglitazone FDA Advisory Committee meeting, at which the committee was presented with a range of studies of differing types.