Access to investigational drugs for patients with amyotrophic lateral sclerosis in the USA

Patients around the world face challenges accessing investigational drugs through compassionate use programmes. In the USA, the Accelerating Access to Critical Therapies for ALS Act, signed into law in December 2021, aims to improve access to investigational drugs for people with amyotrophic lateral sclerosis (ALS), while promoting research for neurodegenerative diseases. This legislation provides an opportunity to address crucial questions about evidence, resource allocation, and equity in compassionate use programmes.