Congressional Hearing on World Rare Disease Day Looks at Slate of Bills

Rare Daily Staff

The House Energy and Commerce Committee subcommittee on Health will hold a hearing on legislative proposals to support patients with rare diseases on February 29, World Rare Disease Day.

The hearing, which will be held at 10 a.m. at 2123 Rayburn House Office Building, has not yet announced a list of witnesses.

The hearing is expected to touch on 18 pieces of legislation that have been introduced into Congress. They include:

H.R. 1092, Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act, a bill designed to strengthen the use of patient-experience data within the benefit-risk framework for approval of new drugs.

H.R. 3433, Give Kids a Chance Act, which would amend the Federal Food, Drug, and Cosmetic Act with respect to molecularly targeted pediatric cancer investigations, and for other purposes.

H.R. 4758, Accelerating Kids Access to Care Act, a bill that would amend title XIX of the Social Security Act to streamline enrollment under the Medicaid program of certain providers across State lines, and for other purposes.

H.R. 5539, Optimizing Research Progress Hope and New (ORPHAN) Cures Act, a bill that would amend title XI of the Social Security Act to expand and clarify the exclusion for orphan drugs under the Drug Price Negotiation Program.

H.R. 5547, Maintaining Investments in New Innovation (MINI) Act, which would amend title XI of the Social Security Act to protect access to genetically targeted technologies.

H.R. 5663, ALS Better Care Act, a bill that would amend title XVIII of the Social Security Act to provide coverage of ALS-related services under the Medicare program for individuals diagnosed with amyotrophic lateral sclerosis, and for other purposes

H.R. 6020, Honor Our Living Donors Act, a bill that would amend the Public Health Service Act to eliminate consideration of the income of organ recipients in providing reimbursement of expenses to donating individuals, and for other purposes.

H.R. 6094, Providing Realistic Opportunity to Equal and Comparable Treatment for (PROTECT) Rare Act, which would amend titles XVIII and XIX of the Social Security Act and title XXVII of the Public Health Service Act to refine the set of information sources for determining coverage of certain drugs and biologicals used in the treatment or management of a rare disease or condition, and for other purposes.

H.R. 6465, Preserving Life-saving Access to Specialty Medicines in America (PLASMA) Act, which would amend title XVIII of the Social Security Act to provide a phase-in for plasma-derived products under the manufacturer discount program. H.R. 6664, Innovation in Pediatric Drugs Act.

H.R. 6705, Effective Screening and Testing for Tuberculosis Act, a bill that would require the Secretary of Health and Human Services to treat certain tests for tuberculosis as breakthrough devices eligible for expedited development and priority review, to require certain establishments that perform donor screening or testing to screen or test for active and latent tuberculosis, and for other purposes.

H.R. 7188, Shandra Eisenga Human Cell and Tissue Product Safety Act, which would require the Secretary of Health and Human Services to conduct a national, evidence-based education campaign to increase public and health care provider awareness regarding the potential risks and benefits of human cell and tissue products transplants, and for other purposes.

H.R. 7248, FDA Modernization Act 3.0, which would amend the Federal Food, Drug, and Cosmetic Act to establish a process for the qualification of nonclinical testing methods to reduce and replace the use of animals in nonclinical research, improve the predictivity of nonclinical testing methods, and reduce development time for a biological product or other drug, and for other purposes.

H.R. 7383, Retaining Access and Restoring Exclusivity (RARE) Act, which amend the Federal Food, Drug, and Cosmetic Act to set forth limitations on exclusive approval or licensure of drugs designated for rare diseases or conditions.

H.R. 7384, Creating Hope Reauthorization Act of 2024, would amend the Federal Food, Drug, and Cosmetic Act to extend the authority of the Secretary of Health and Human Services to issue priority review vouchers to encourage treatments for rare pediatric diseases.

H.R. ____, Antimicrobial Resistance Research Assessment Act, legislation that would direct the Comptroller General of the United States to prepare a report on Federal efforts to address antimicrobial resistance.

H.R. ____, Patient Access Act, which would amend title XI of the Social Security Act to exclude from antikickback and other sanctions certain travel and lodging arrangements between manufacturers of drugs and individuals being administered such drugs, and for other purposes.

H.R. ____, Sickle Cell Disease Comprehensive Care Act, which would amend title XIX of the Social Security Act to enable State Medicaid programs to provide comprehensive, coordinated care through a health home to individuals with sickle cell disease.

The hearing will be open to the public and press and will be live streamed online at https://energycommerce.house.gov/. The question is if any of these bills will continue to grab the attention of lawmakers beyond World Rare Disease day.