Risk-benefit decisions in licensing changes

Abstract

At the time drugs are licensed evidence for the efficacy and safety of a new compound will have been more or less secured. At this stage, efficacy has often only been established in a limited number of patients under a narrow range of conditions, and safety in relatively small numbers for a short duration. During the post-marketing period, evidence may accumulate to suggest that the efficacy is considerable in a wider group of patients than was originally indicated, and that safety during long-term use in a more heterogeneous population is acceptable. Alternatively, efficacy may appear to be less satisfactory than originally believed and safety becomes prejudiced. The good news is relatively easy to evaluate, and I am going to concentrate on the bad news which is much more difficult to assess. My paper will, therefore, address risk-benefit assessment when there is an apparent problem.