Amyotrophic lateral sclerosis is a neurodegenerative disorder affecting predominantly central and peripheral motor neurons, resulting in progressive weakness and atrophy of voluntary skeletal muscle. It currently has no effective therapeutic strategies. Dr. Tomislav Babic and Michael Murphy of Worldwide Clinical Trials examines how clinical presentation, diagnostic criteria, traditional and evolving standards for trial design and the increasing importance of including quality of life and healthcare utilisation data in programmes provide the basis for a review.