2023 ASH Advocacy Efforts Related to Sickle Cell Disease

In 2023, ASH was actively advocating for issues impacting hematology research and practice, including research and public health funding, access to quality care for patients, physician payment and coverage for hematologists, and policy issues related to sickle cell disease (SCD). The following overview highlights ASH’s advocacy in 2023 to help enhance and expand government activities in SCD.

SCD Advocacy on Capitol Hill

• The Sickle Cell Disease (SCD) Comprehensive Care Act (H.R. 1672/S. 996) was reintroduced in a bipartisan fashion in both houses of Congress in late March by Representatives Michael Burgess (R-TX) and Danny Davis (D-IL) and Senators Cory Booker (D-NJ) and Tim Scott (R-SC). ASH worked closely with these congressional champions to ensure that the legislation was reintroduced in the 118th Congress.
  • ASH issued a statement in support of the legislation, and a quote from ASH President Dr. Robert Brodsky was included in the statement issued by Senator Booker upon reintroduction.
  • In 2023, ASH staff and members of the Society convened nearly 150 in-person and virtual meetings with congressional offices to encourage co-sponsorship of this legislation.
  • ASH also worked with SCD partners, including community groups, to provide resources for their respective virtual Capitol Hill Days. In September, ASH, the Sickle Cell Disease Association of America, and Sick Cells co-hosted a congressional briefing focused on the need for comprehensive care in a time of therapeutic innovation and the importance of this bill.
  • Congressional staff on the House Energy and Commerce Committee have indicated that a potential path forward for this legislation may be the creation of a Health Home for SCD as it may be more palatable for members of Congress to support an enhancement of current law (i.e., the Affordable Care Act) than to create a new program such as the SCD Comprehensive Care Act. Representative Burgess’ staff asked ASH staff to review and provide comments on draft legislative text that would create an SCD Health Home.
• During 2023, ASH continued to be highly visible in advocacy for federal support of biomedical research and public health funding, including funding for the National Institutes of Health (NIH), the Centers for Disease Control and Prevention (CDC), and the Health Resources and Services Administration (HRSA)—this included focused advocacy to increase funding for the following sickle cell programs.
  • In the spring, ASH submitted testimony to the House appropriators and a similar statement to Senate appropriators on fiscal year (FY) 2024 funding issues of importance to ASH, including a request for at least $10 million for the CDC’s Sickle Cell Data Collection program, and funding for sickle cell disease programs within HRSA.
  • As a direct result of ASH’s ongoing advocacy to support the CDC’s Sickle Cell Data Collection Program, it was recently expanded from 11 to 16 states (AL, AZ, CA, CO, FL, GA, IN, MI, MN, MO, NJ, NC, RI, TN, TX, WI), now covering approximately 50% of the U.S. SCD population. Funding, which was doubled in FY 2023, will support the first year of a five-year effort to collect essential population-based data on sickle cell disease (SCD) to analyze SCD-related health care and health outcomes across states and population groups.
• In mid-May, the Sickle Cell Care Expansion Act (H.R. 3100 /S.1423) was reintroduced in both the House and Senate. ASH President Dr. Robert Brodsky’s statement supporting the legislation was included in the congressional co-sponsors’ press release on it. This legislation enables the National Health Service Corps to provide educational incentives, such as loan repayment assistance and scholarships, for those studying classical hematology, with a focus on those who engage in research or care for individuals with SCD. This bill also provides funding grants to hospitals that offer comprehensive SCD care to further fund support services for young adults aged 18–29 transitioning from pediatric to adult care.
• On June 14, former ASH President Alexis A. Thompson, MD, MPH, testified at a House Energy and Commerce Subcommittee on Health hearing on legislative proposals to provide access to care for patients and support research for rare diseases. Among the proposals discussed was the Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2023 (H.R. 3884), which reauthorizes the Sickle Cell Disease Treatment Demonstration Program administered by HRSA.

SCD Advocacy with Federal Agencies

• ASH continues to have discussions with the Centers for Medicare and Medicaid Services (CMS) as it develops its Cell and Gene Therapy Payment Model for SCD; ASH learned that the model is on track to be implemented in 2025, a year ahead of schedule. As a follow up to discussions, the Society sent a letter building on ASH’s advocacy for comprehensive care and outlined a thorough and inclusive “Wishlist” for treatment considerations that are crucial to the success of this model.
  • In December, ASH submitted comments on CMS’ Sickle Cell Disease Action Plan, the objective of which is to eliminate barriers to care, reduce health disparities, and improve health outcomes for individuals with SCD. ASH’s letter encourages CMS to support the delivery of comprehensive care to individuals living with SCD across Medicaid and Medicare.
  • On September 11, ASH submitted comments on CMS’ Calendar Year 2024 Physician Fee Schedule. ASH supported several changes to Medicare payment for dental services, including coverage for patients receiving CAR-T therapy and chemotherapy, and also recommended dental coverage for individuals who have SCD.  Additionally, ASH supported the CMS proposal to create new services to address health-related social needs, including community health integration, social determinants of health risk assessment, and principal illness navigation services. Telehealth extensions granted under the pandemic and authorized by Congress for patients receiving care in their homes was also a provision supported by ASH.
• Over the past year, ASH and HHS co-hosted a series of three Roundtables with federal and non-federal data registry leaders, and participants prioritized opportunities to collaborate on harmonizing data elements and definitions across programs and establishing methods to reduce redundant data capture without compromising data quality. During the final Roundtable in June, HHS Secretary Xavier Becerra and Assistant Secretary for Health Admiral Rachel Levine, MD, provided the keynote talk showing HHS’ highest level of support and commitment to SCD.
• In late March, representatives from the ASH Research Collaborative (RC) and the Innovative Genomics Institute (IGI) briefed nearly 400 FDA leaders and staff on reports from the ASH RC-IGI Real-World Evidence Initiative – Accelerating Genomic Therapies with Real-World Evidence.
• ASH, in collaboration the Centers for Disease Control and Prevention (CDC), Foundation for Women & Girls with Blood Disorders (FWGBD), and Sickle Cell Reproductive Health Education Directive (SC RED) released new fact sheets on preconception care, prenatal care, and postpartum care for women living with SCD. The fact sheets are also available in Spanish.

Additional Non-Federal SCD Advocacy

• In mid-May, ASH submitted comments to the Institute for Clinical and Economic Review (ICER) on their draft evidence report assessing the comparative clinical effectiveness and value of two gene therapies for SCD.