2025 second_quarter Filing

Support for H.R.946, Optimizing Research Progress Hope And New Cures Act (ORPHAN Cures Act). Legislation regarding orphan drug pricing, value, and reimbursement, relative to the Inflation Reduction Act of 2022 (Pub. L. No. 117-169, 136 Stat. 1818 (2022)). Policies regarding copay accumulators and impact on rare disease patients. Support of reauthorization of priority review voucher program. H.R.1, One Big Beautiful Bill Act, provisions regarding orphan drugs.

Legislation regarding clinical research for rare diseases and orphan drug development, and the Accelerated Approval pathway. Support of reauthorization of priority review voucher program.