2023 ASH Advocacy Efforts to Ensure Patient Access to Care

During 2023, ASH continued to be highly visible in our advocacy on issues that ensure patient access to affordable health care, including blood donor diversity, maternal health and hematology, drug shortage issues, and access to cell and gene therapies. The following is a summary of advocacy efforts that ASH pursued with the Federal Administration and the U.S. Congress throughout 2023.

Blood Donor Diversity Issues

• ASH commended the U.S. Food and Drug Administration (FDA) for releasing an evidenced-based safe and inclusive draft guidance recommending that blood donor eligibility be determined based on individual assessment, regardless of gender identity or sexual orientation—an important issue for which ASH has engaged the FDA for several years. ASH also applauded the FDA when the Agency released its final Recommendations for Evaluating Donor Eligibility Using Individual Risk-Based Questions to Reduce the Risk of Human Immunodeficiency Virus Transmission by Blood and Blood Products

Maternal Health and Hematology Advocacy

• ASH sent letters of support to members of Congress who have introduced legislation consistent with ASH’s policy statement on the ASH sent letters of support to members of Congress who have introduced legislation consistent with ASH’s policy statement on the ASH sent letters of support to members of Congress who have introduced legislation consistent with ASH’s policy statement on the ASH sent letters of support to members of Congress who have introduced legislation consistent with ASH’s policy statement on the Right to Maternal Health and Hematology. During the last Congressional session, two bills were introduced: The Women’s Health Protection Act, and The Let Doctors Provide Reproductive Health Care Act. Both pieces of legislation would protect the patient–provider relationship and give physicians, including hematologists, freedom to make evidence-based and lifesaving decisions for patients without government interference. In November, ASH sent a letter of support to the cosponsors of the Let Doctors Provide Reproductive Health Care Act, applauding the reintroduction of that legislation. 
• In April, ASH joined 33 patient and provider groups in a statement regarding the Alliance for Hippocratic Medicine et al. v. FDA et al., which calls into question the FDA’s approval of mifepristone. The joint statement expressed serious concern regarding legal interference with the federal agency’s ability and authority to review and approve drugs that are critical to health care access for all Americans.
• In response to a proposed rule on reproductive healthcare privacy issued by the U.S. Department of Health and Human Services Office of Civil Rights, ASH submitted comments on June 14, stating its support for the proposal to strengthen patient–provider confidentiality and prohibit the disclosure of protected health information, among other issues.
• In October, ASH joined another amicus brief to urge the U.S. Supreme Court to accept a case to protect access to FDA-approved drugs for patients (Alliance for Hippocratic Medicine et al. v. FDA).
• ASH joined the American Cancer Society, the American Society for Clinical Oncology, and the Leukemia and Lymphoma Society in a policy brief focused on medication access for pregnant patients with cancer and fertility preservation in cancer care. ASH is currently drafting a policy statement entitled, Fertility Management in Hematologic Care.
• ASH, in collaboration with the Centers for Disease Control and Prevention (CDC), the Foundation for Women & Girls with Blood Disorders, and Sickle Cell Reproductive Health Education Directive, released new fact sheets on preconception care, prenatal care, and postpartum care for women living with sickle cell disease.
• In December, ASH supported the Iron Deficiency Education and Awareness (IDEA) Act (H.R. 6746), introduced by Representative Yadira Caraveo (D-CO), which seeks to raise awareness of iron deficiency among women and young children. A quote from ASH President Dr. Robert Brodsky was included in the statement issued by Representative Caraveo upon introduction of the legislation.

Engaging with FDA to Ensure Access to Safe and Effective Hematologic Therapies

• Staff from the U.S. Food and Drug Administration’s (FDA) Office of Prescription Drug Promotion presented at the Committee on Government Affairs meeting about the Agency’s role in reviewing prescription drug advertising and promotional labeling, the processes used for the reviews and monitoring, and highlighted how providers can report potentially false or misleading prescription drug promotion to the FDA’s Bad Ad Program.
• As drug shortages continue to reach an all-time high in the United States, especially for hematologic therapies, ASH continues to update the Hematologic Drug Shortage Information webpage to keep members apprised of shortages and actively work with Congress, federal agencies, and other stakeholders to ensure access to safe and effective hematologic drugs.
  • In late May, the lead from FDA’s Office of Drug Shortages met with the Committee on Government Affairs to discuss the impact of ongoing hematologic drug shortages, FDA’s efforts to prevent and mitigate shortages, and how ASH could help support the Agency.
  • Over the summer, the Society activated the ASH Grassroots Network to contact their elected officials about the devastating impact of the shortages and encourage action.
  • In late August, ASH sent a letter to Representative Cathy McMorris Rodgers (R-WA-5), Chair of the House Energy & Commerce Committee, recommending changes to draft legislation entitled Stop Drug Shortages Act. ASH also sent a letter to Congressional leaders in early September about the need for timely and comprehensive action on drug shortages, including those that impact hematology care.
• In early October, ASH hosted the second annual ASH/FDA Collaboration: A Workshop on Regulatory Science in Hematology to facilitate information sharing between ASH members and FDA representatives. The workshop is for ASH members in academic medicine who focus on clinical or translational research, drug development, or regulatory medicine. ASH was pleased to welcome the workshop co-chairs, Drs. Grzegorz Nowakowski (Chair, ASH Subcommittee on Clinical Trials, Mayo Clinic) and Richard Pazdur (Director, FDA Oncology Center of Excellence) and over 50 FDA staff to educate 20 ASH members who were selected from a pool of 60 strong applicants.
• Over the past year, ASH submitted feedback to the FDA on various draft guidance documents that intersect with hematology and are directly related to the Society’s priorities, leading to better patient access to therapies.
  • Letter re: Pediatric Drug Development Under the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BCPA): Scientific Considerations
  • Letter re: Decentralized Clinical Trials for Drugs, Biological Products, and Devices
  • Letter and Presentation to FDA re Capturing Post-Approval Efficacy Data on Cell and Gene Therapies
  • Letter to FDA Draft on Considerations re Accelerated Approval of Oncology Therapeutics
  • Letter to FDA and International Conference on Harmonization re Draft Guideline for Good Clinical Practice
  • Letter to FDA re: Draft Guidance on Graft-versus-Host Diseases: Developing Drugs, Biological Products, and Certain Devices for Prevention or Treatment

Access to Safe and Effective Drugs and Therapies

• ASH continues to have discussions with the Centers for Medicare and Medicaid Services (CMS) as it develops its Cell and Gene Therapy Access 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 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.
• As part of the Coalition to Improve Access to Cancer Care (CIACC), ASH continues to advocate for the Cancer Drug Parity Act (S. 2039/H.R. 6301), which was reintroduced in the Senate in June and in the House in early November. This legislation seeks to ensure that cancer patients have equal insurance coverage and access to all approved anticancer regimens by requiring any health plan that provides coverage for cancer chemotherapy treatment to provide coverage for orally administered and self-injectable anticancer medications at an out-of-pocket cost no less favorable than that of IV, port administered, or injected anticancer medications.
  • ASH also joined several patient and provider organizations to support legislation in the state of Michigan to help address the financial barriers to cancer care that occur when undue cost sharing requirements are imposed upon individuals and families for oral and self-administered anticancer medications. The legislation passed the state legislature and was signed into law in October.
• In early October, ASH was invited to join a series of Amicus Briefs in support of the Medicare Drug Negotiation Program that is aimed at lowering the cost of Medicare Part D drugs. ASH has joined 5 briefs to date. (Janssen Pharmaceuticals, Inc. v. Becerra et al., Bristol Myers Squibb Co. v. Becerra et al., AstraZeneca Pharmaceuticals LP et al. v. Becerra et al., Dayton Area Chamber of Commerce et al. v Becerra et al., and Boehringer Ingelheim Pharmaceuticals, Inc. v. U.S. Department of Health and Human Services et al.)