Societal participation in the development of orphan drugs: a systematic review.

ABSTRACT: The development of orphan drugs (ODs) remains constrained by structural barriers, including limited scientific knowledge, high clinical uncertainty, and fragile commercial incentives. In parallel, an alternative ecosystem has emerged in which non-commercial societal actors (patients, advocacy groups, and philanthropic entities) contribute to the design, evaluation, and dissemination of therapies for rare diseases. This systematic review provides the first structured synthesis of empirical evidence on societal participation in OD development, with a specific focus on neuromuscular and neurodegenerative disorders. Twenty-one peer-reviewed studies were included, spanning a range of designs from case reports to stakeholder surveys. A functional typology of societal roles was developed, identif

SUBMITTER: Akodad S 

PROVIDER: S-EPMC12457289 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature