Coping with small populations of patients in clinical trials

Abstract

Rare disease research involves small numbers of patients, and this presents challenges in the design, conduct, analysis and interpretation of clinical studies. There are no special methods for coping with small populations of patients in clinical trials; however, there are many different types of clinical study designs and approaches to increase the efficiency and utility of clinical trials. Common to all approaches is the requirement for rigorous planning to ensure that every patient participating in a clinical study contributes as much information as possible. Current approaches aimed at generating the best possible evidence base are discussed, including a focus on regulatory considerations and research initiatives. © 2014 Informa UK, Ltd.