Congress has set a target date of June 29 for final passage of this comprehensive legislation, which includes a number of measures beneficial for the rare disease community. The EXPERRT Act aims to promote collaboration between FDA reviewers and rare disease experts and give patient groups a greater voice during the drug review process. The full legislative package also facilitates future cystic fibrosis drug approvals by mandating more training for reviewers on issues specific to rare diseases and providing the FDA with greater flexibility to swiftly and safely approve groundbreaking new treatments.

Through greater collaboration, improved access to rare disease expertise, and more efficient drug review channels, breakthrough new treatments can more quickly reach people with cystic fibrosis and others with rare diseases.