Post-Approval Research in Drug Development: Priorities and Practices.

A prescriber might ask if a new medication is a good option for use in the patients he or she sees in clinic, with their particular blends of demographic and comorbid clinical characteristics. Is this medicine more effective, safe, tolerable, or affordable than the options used in the past? A payer may ask if the new medication offers a more effective, cost-efficient, or convenient alternative to those treatments already being covered. These are the types of questions that are often difficult to answer on the basis of the clinical trials used to support a medication's initial approval, which are generally designed to evaluate a medication's efficacy, safety, and tolerability in narrowly defined patient populations. Consequently, in order to answer the questions most relevant to key stakeholders (i.e., regulators, patients, and clinicians), it is important to continue to examine a medication's impact and characteristics after it has received regulatory approval. Such studies vary in their purpose, scope, and methodology. In this chapter, we review the types of questions most likely to be investigated after regulatory approval, the methods generally used to investigate them, and the characteristics typically considered when prioritizing the allocation of resources.