Developing Drugs For Rare Diseases: A New Approach To Generating Clinical Evidence

By Janet Woodcock, former director, Center for Drug Evaluation and Research (CDER), US FDA

The past half-century has witnessed remarkable progress in biomedical research, unraveling the complexities of human disease and offering new therapeutic possibilities. For rare diseases, this progress has been particularly significant, with researchers identifying the molecular basis of many conditions and developing potential treatments. While the path to drug approval is complex, especially for rare diseases, innovative solutions are within reach. This article addresses the limitations of applying standard clinical trial designs to rare disease drug development and proposes alternative approaches to generating robust evidence of efficacy.