Hearing: Legislative Proposals to Support Patients with Rare Diseases

Subcommittee on Health (Committee on Energy and Commerce)

Thursday, February 29, 2024 (10:00 AM)

2123 RHOB
Washington, D.C.

Witnesses

Dr. Terence Flotte, MD
Provost and Dean of UMass Chan Medical School, Vice President of American Society of Gene and Cell Therapy (ASGCT)

Added 02/27/2024 at 02:24 PM
  • Witness Statement [PDF] Added 02/27/2024 at 04:54 PM
  • Questions For the Record, Transmittal Letter [PDF] Added 04/18/2024 at 05:47 PM
  • Response to Questions For the Record [PDF] Added 05/07/2024 at 03:41 PM
  • Truth in Testimony [PDF] Added 03/05/2024 at 03:29 PM
  • Curriculum Vitae [PDF] Added 02/27/2024 at 04:54 PM

Dr. Alexander Bassuk, MD, PhD
Physician-in-Chief, University of Iowa Stead Family Children’s Hospital; Chair and Professor, Stead Family Department of Pediatrics

Added 02/27/2024 at 02:24 PM
  • Witness Statement [PDF] Added 02/27/2024 at 04:54 PM
  • Questions For the Record, Transmittal Letter [PDF] Added 04/18/2024 at 05:47 PM
  • Response to Questions For the Record [PDF] Added 05/07/2024 at 03:41 PM
  • Truth in Testimony [PDF] Added 03/05/2024 at 03:29 PM
  • Curriculum Vitae [PDF] Added 02/27/2024 at 04:54 PM

Dr. Aaron Kesselheim, MD, JD, MPH
Professor of Medicine, Harvard Medical School; Director, Program On Regulation, Therapeutics, And Law (PORTAL) at Brigham and Women’s Hospital

Added 02/27/2024 at 02:24 PM
  • Witness Statement [PDF] Added 02/27/2024 at 04:54 PM
  • Questions For the Record, Transmittal Letter [PDF] Added 04/18/2024 at 05:47 PM
  • Truth in Testimony [PDF] Added 03/05/2024 at 03:29 PM
  • Curriculum Vitae [PDF] Added 02/27/2024 at 04:54 PM

Dr. Jeromie Ballreich, PhD, MHS
Associate Research Professor, Johns Hopkins Bloomberg School of Public Health

Added 02/27/2024 at 03:45 PM
  • Witness Statement [PDF] Added 02/28/2024 at 06:35 PM
  • Questions For the Record, Transmittal Letter [PDF] Added 04/18/2024 at 05:47 PM
  • Truth in Testimony [PDF] Added 03/05/2024 at 03:29 PM
  • Curriculum Vitae [PDF] Added 02/28/2024 at 06:35 PM

Dr. Alice Chen, PhD
Senior Fellow, USC Schaeffer Center for Health Policy & Economics; Associate Professor & Vice Dean for Research, USC Sol Price School of Public Policy

Added 02/27/2024 at 02:24 PM
  • Witness Statement [PDF] Added 02/27/2024 at 04:54 PM
  • Questions For the Record, Transmittal Letter [PDF] Added 04/18/2024 at 05:47 PM
  • Response to Questions For the Record [PDF] Added 05/07/2024 at 03:41 PM
  • Truth in Testimony [PDF] Added 03/05/2024 at 03:29 PM
  • Curriculum Vitae [PDF] Added 02/27/2024 at 04:54 PM

Ms. Khrystal Davis, JD
Founding President, Texas Rare Alliance

Added 02/27/2024 at 02:24 PM
  • Witness Statement [PDF] Added 02/28/2024 at 01:41 PM
  • Questions For the Record, Transmittal Letter [PDF] Added 04/18/2024 at 05:47 PM
  • Truth in Testimony [PDF] Added 03/05/2024 at 03:29 PM
  • Curriculum Vitae [PDF] Added 02/28/2024 at 09:33 AM

Text of Legislation

  • H.R. 6020, Honor Our Living Donors Act (Reps. Obernolte and DelBene) [PDF]
  • H.R. 3433, Give Kids a Chance Act (Reps. McCaul and Eshoo) [PDF]
  • H.R. 7384, Creating Hope Reauthorization Act of 2024 (Reps. McCaul and Eshoo) [PDF]
  • H.R. 7383, Retaining Access and Restoring Exclusivity (RARE) Act (Reps. Matsui and Bilirakis) [PDF]
  • H.R. 7248, FDA Modernization Act 3.0 (Reps. Carter and Barragán) [PDF]
  • H.R. 6705, Effective Screening and Testing for Tuberculosis Act (Reps. Moolenaar and Dingell) [PDF]
  • H.R. 7188, Shandra Eisenga Human Cell and Tissue Product Safety Act (Reps. Moolenaar and Dingell) [PDF]
  • H.R. 1092, Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act (Reps. Matsui and Wenstrup) [PDF]
  • H.R. 4758, Accelerating Kids Access to Care Act (Reps. Trahan and Miller-Meeks) [PDF]
  • H.R. 6465, Preserving Life-saving Access to Specialty Medicines in America (PLASMA) Act (Reps. Hudson and Davis) [PDF]
  • H.R. 5539, Optimizing Research Progress Hope And New (ORPHAN) Cures Act (Reps. Joyce and Nickel) [PDF]
  • H.R. 5547, Maintaining Investments in New Innovation (MINI) Act (Reps. Nickel and Joyce) [PDF]
  • H.R. 6094, Providing Realistic Opportunity To Equal and Comparable Treatment for (PROTECT) Rare Act (Reps. Matsui and Dunn) [PDF]
  • H.R. 6664, Innovation in Pediatric Drugs Act (Reps. Eshoo and McCaul) [PDF]
  • H.R. 5663, ALS Better Care Act (Reps. Schakowsky, Quigley, and Fitzpatrick) [PDF]
  • H.R. 7436, Antimicrobial Resistance Research Assessment Act (Rep. Griffith) [PDF]
  • H.R. ____, Patient Access Act (Rep. Guthrie) [PDF]
  • H.R. ____, Sickle Cell Disease Comprehensive Care Act (Rep. Burgess) [PDF]

Support Documents

  • Hearing Notice [PDF]
  • Public Memo [PDF] Added 02/27/2024 at 02:24 PM
  • Documents For the Record [PDF] Added 02/29/2024 at 04:27 PM

Member Statements

  • Cathy McMorris Rodgers Opening Statement [PDF] Added 02/29/2024 at 04:44 PM
  • Brett Guthrie Opening Statement [PDF] Added 02/29/2024 at 04:44 PM
  • Anna Eshoo Opening Statement [PDF] Added 03/05/2024 at 03:29 PM
  • Frank Pallone, Jr. Opening Statement [PDF] Added 03/05/2024 at 03:29 PM

Hearing Record

  • Member Attendance [PDF] Added 03/25/2024 at 08:11 PM
  • Unedited Hearing Transcript [PDF] Added 03/25/2024 at 08:11 PM

First Published: February 23, 2024 at 11:56 AM
Last Updated: May 7, 2024 at 03:41 PM