ABSTRACT
Some benefit-risk assessments are performed internally by pharmaceutical companies for Go/No-Go decision. Quan et al. (2010) applied benefit-risk assessment in a phase II dose finding trial to identify the doses that are sufficiently effective and safe for future late-phase trials. It may be informal at the early stage and become formal as the drug advanced through the development process. To aid the decision process, the core data sheet should capture the up-to-date assessment. Perhaps, the most important and comprehensive benefit-risk assessment is the one conducted at the final new drug application review. If the assessment is positive, the drug will be approved. Thus, a well-conducted benefit-risk assessment is essential in the drug approval process. It is also important that the benefit-risk assessment process is consistent, objective, and transparent. Both the EMA and the FDA have made great effort toward this goal.
