Lawmakers Review Several Legislative Proposals Intended to Accelerate Innovation of Therapies, Including STAT Act

On Thursday, March 17th, the House Energy & Commerce Subcommittee on Health held a hearing regarding The Future of Medicine: Legislation to Encourage Innovation and Improve Oversight.

This hearing explored a number of legislative proposals intended to accelerate innovation of therapies, while ensuring appropriate levels of oversight, including H.R. 1730, the Speeding Therapy Access Today (STAT Act) sponsored by U.S. Representative Gus Bilirakis (R-FL) and U.S. Representative G.K. Butterfield (D-NC).

The STAT Act will improve rare disease coordination, stakeholder engagement, and policy development within the U.S. Food and Drug Administration (FDA) by expanding existing authority to create a Rare Disease Center of Excellence, inform rare disease policies and actions by creating a Rare Disease and Condition Drug Advisory Committee and fund regulatory science and related activities to support development of therapies to treat very small rare disease populations. It will also strengthen rare disease patient access to FDA-approved therapies in both public and commercial plans through enhanced FDA and Centers for Medicare and Medicaid Services coordination, proactive engagement of payers, and specific actions intended to strengthen Medicare and Medicaid beneficiary access to novel therapies.

In addition to the STAT Act, the hearing addressed other bills of interest to the rare disease community:

• The Cures 2.0 Act, H.R. 6000, which would include authorization of a Rare Disease Center of Excellence and contains several other rare disease-focused provisions
• The Helping Experts Accelerate Rare Treatments (HEART) Act, H.R. 6888, which would enhance reporting on rare disease activities and authorize a GAO study of the European Union’s best practices in handling rare disease applications
• The Better Empowerment Now to Enhance Framework and Improve Treatments Act of 2021 (BENEFIT) Act, H.R. 4472, which would build upon existing law and policies to ensure patient experience data is fully reflected as part of FDA’s benefit-risk regulatory framework
• The Advanced Research Project Agency-Health (ARPA-H) Act, H.R. 5585, which would establish a new, collaborate agency within the Department of HHS to catalyze the development of high-impact, novel treatments, diagnostics, cures, and preventative measures for communities with significant unmet need
• The Diversifying Investigations Via Equitable Research Studies for Everyone Trials (DIVERSE) Act, H.R. 5030, and the Diverse and Equitable Participation in Clinical Trials (DEPICT) Act, H.R. 6584, which would enhance clinical trial diversity by decentralizing trials and making participation easier and more accessible

PWSA | USA would like to thank all of our advocates for proactively engaging with your elected officials! It is through constant and consistent engagements that our community can help bring these crucial pieces of legislation to the attention of policy makers and the general public.