Pathology

Bioinformatics

Biology

Clinical study design

Intensive care medicine

Medical physics

Drug

Drug development

Clinical trial

Medicine

Orphan drug

Pharmacology

Nature Reviews Methods Primers

Orphan drug development is a rapidly expanding field. Nevertheless, clinical trials for rare diseases can present inherent challenges. Optimal study design and partnerships between academia and industry are therefore required for the successful development, delivery and clinical approval of effective therapies in this group of disorders.

Designing clinical trials for rare diseases: unique challenges and opportunities