Clinical Trials

■■❚❙❘Chapter 4EndpointsAmeet Bakhai, Amit Chhabra, and Duolao Wang“The greatest challenge to any thinker is stating the problem in away that will allow a solution,” said Bertrand Russell (1872–1970).This maxim holds as strongly for clinical trials as for any questin life. While the first clinical trials asked questions about eventssuch as the rate of death or recurrence of infection, researchersare now interested in more sophisticated clinical trial endpointsfor measuring combined outcomes (eg, avoiding death orhospitalization, or disease-specific outcomes such as asthmaexacerbations or heart attacks). Researchers are alsobecoming more interested in economic outcomes for evaluatingwhether clinically effective treatments are also cost-effective.Surrogate endpoints are also used in trials that are related todisease-specific outcomes; eg, cholesterol level is a surrogateendpoint related to the risk of heart attacks. In this chapter,we discuss the types of endpoints that can be used inclinical trials.37