CHAPTER 1 Access to Genetic Therapies for People with Hemophilia  

 

EXPERTS:  Aaron Blocker1, Nathan Schaefer1, Amy Dunn 2,3. 1.  National Bleeding Disorders Foundation,2.  Nationwide Children’s Hospital,  3. The Ohio State University College of Medicine.

1(Institution, City, State, Country)

 

TOPIC 1: Access to gene-based  therapies for hemophilia; alignment of payer policy with FDA-approved indications and evidence-based clinical practice.

 

BACKGROUND

  • AAV-based gene therapy represents a significant advancement in the treatment of hemophilia, offering the potential for sustained endogenous clotting factor expression and long-term reductions in bleeding rates, factor utilization, and treatment burden 1,5,8.
  • FDA-approved gene-based  therapies for hemophilia have clearly defined labeled indications developed through rigorous clinical evaluation.1,8 These indications are intentionally broad to reflect the diversity of bleeding phenotypes, treatment needs, and clinical presentations within the hemophilia community. 
  • However, coverage barriers have emerged across payers, largely stemming from the adoption of clinical trial inclusion criteria in place of FDA-approved labeling. Examples include restricting access based on baseline factor activity, prophylaxis status, or other eligibility criteria that were necessary for trial design but are not part of the label indication5,1.
  • These misaligned requirements have resulted in coverage denials, treatment delays, and inequitable access for individuals who otherwise meet the FDA-approved indication and are deemed appropriate candidates by their hemophilia treatment center (HTC).
  • Independent economic analyses have shown that gene therapy for hemophilia may be cost-effective when long-term clinical and economic outcomes are considered.3 Furthermore, real-world insurance continuity studies demonstrate that individuals with hemophilia often remain with the same insurer for multiple years, countering payer assumptions about short member tenure4. 
  • Ensuring alignment between payer coverage and FDA labeling is critical to allow appropriate candidates access to gene therapy.

RECOMMENDATION 1.1 Coverage Should Align with FDA-Approved  Indications and Current Standards of Care

  • MASAC recommends that all payers provide coverage for FDA-approved gene therapies for hemophilia in accordance with the full scope of their labeled indications and the clinical judgement of providers. Payers should not impose additional restrictions derived from clinical trial protocols or non-evidence-based criteria that are not included in product labeling.
  • REMARK: Coverage criteria should cite FDA-labeled indications. Trial eligibility parameters such as baseline factor levels, prophylaxis requirements, or specific screening conditions, should not be used as de facto coverage requirements unless explicitly stated in the labeling.
  • REMARK: Appeal reviews should involve clinicians with expertise in treatment of people with  hemophilia and gene therapy, ensuring decisions are consistent with scientific and clinical standards. 
  • REMARK: Transparency in medical policies is essential; payers should publish clear, up-to-date criteria reflecting current labeling and standards of care. 

 

FUTURE RESEARCH NEEDS:

  • Studies examining the impact of insurance coverage variability on timing of therapy, access to care and clinical outcomes. 
  • Economic analyses assessing payer risk, long-term cost offsets, and the impact of coverage alignment with labeled indications. 
  • Evaluation of access disparities and identification of policy interventions to ensure equitable access among all eligible individuals with hemophilia. 

 

 

REFERENCES

  1.  U.S. Food and Drug Administration. HEMGENIX (etranacogene dezaparvovec-drlb) Prescribing Information. 2022.
  2.  Pipe SW et al. Gene therapy with etranacogene dezaparvovec for hemophilia B. N Engl J Med. 2023;388(8):706-718.
  3.  Institute for Clinical and Economic Review (ICER). Valoctocogene roxaparvovec and etranacogene dezaparvovec for hemophilia A and B: Evidence Report. Boston, MA; 2022.
  4.  He C et al. Health insurance coverage and switching among people with hemophilia A in the U.S. J Manag Care Spec Pharm. 2022;28(12):1274-1282.

  5. ClinicalTrials.gov. HOPE-B Study (NCT03569891). 2020.

  6. Ar MC, Balkan C, Kavaklı K. Extended Half-Life Coagulation Factors: A New Era in the Management of Hemophilia Patients. Turk J Haematol. 2019 Aug 2;36(3):141-154. doi: 10.4274/tjh.galenos.2019.2018.0393. Epub 2019 May 15. PMID: 31088040; PMCID: PMC6682782.

  7. Margaretos NM, Patel AM, Panzer AD, Lai RC, Whiteley J, Chambers JD. Variation in access to hemophilia A treatments in the United States. J Med Econ. 2021 Jan-Dec;24(1):1143-1148. doi: 10.1080/13696998.2021.1982225. PMID: 34538215.

  8. U.S. Food and Drug Administration. FDA approves first gene therapy for adults with severe hemophilia. FDA News Release. November 22, 2022.

 

KEYWORDS: Gene Therapy, Hemophilia A, Hemophilia B

MASAC Documents
MASAC Document 297 - Access to Genetic Therapies for People with Hemophilia