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.
Share this!
Jennifer Bolander has been serving as a Special Education Specialist for PWSA (USA) since October of 2015. She is a graduate of John Carroll University and lives in Ohio with her husband Brad and daughters Kate (17), and Sophia (13) who was born with PWS.
Perry A. Zirkel has written more than 1,500 publications on various aspects of school law, with an emphasis on legal issues in special education. He writes a regular column for NAESP’s Principal magazine and NASP’s Communiqué newsletter, and he did so previously for Phi Delta Kappan and Teaching Exceptional Children.
Evan has worked with the Prader-Willi Syndrome Association (USA) since 2007 primarily as a Crisis Intervention and Family Support Counselor. Evans works with parents and schools to foster strong collaborative relationships and appropriate educational environments for students with PWS.
Dr. Amy McTighe is the PWS Program Manager and Inpatient Teacher at the Center for Prader-Willi Syndrome at the Children’s Institute of Pittsburgh. She graduated from Duquesne University receiving her Bachelor’s and Master’s degree in Education with a focus on elementary education, special education, and language arts.
Staci Zimmerman works for Prader-Willi Syndrome Association of Colorado as an Individualized Education Program (IEP) consultant. Staci collaborates with the PWS multi-disciplinary clinic at the Children’s Hospital in Denver supporting families and school districts around the United States with their child’s Individual Educational Plan.
Founded in 2001, SDLC is a non-profit legal services organization dedicated to protecting and advancing the legal rights of people with disabilities throughout the South. It partners with the Southern Poverty Law Center, Protection and Advocacy (P&A) programs, Legal Services Corporations (LSC) and disability organizations on major, systemic disability rights issues involving the Individuals with Disabilities Education Act (IDEA), Americans with Disabilities Act (ADA), and the federal Medicaid Act. Recently in November 2014, Jim retired.