Why It Is Important to Ensure the Price Is Right: Access Restrictions for Therapies Treating Duchenne Muscular Dystrophy
Author(s)
Desai A, Lee K, Brooks K
Red Nucleus, San Francisco, CA, USA
Presentation Documents
OBJECTIVES: Therapeutic options for Duchenne muscular dystrophy (DMD) have been increasing and developing at a rapid rate. Despite the severity and rarity of DMD, some health plans are choosing not to cover these life-prolonging therapies. This research aims to evaluate trends in price and coverage outcomes of DMD therapies in the United States.
METHODS: FDA databases were used to identify therapies approved in DMD. Therapies’ annual Wholesale Acquisition Costs (WACs) were calculated and analyzed together to understand the differences between options in the DMD space. An ICER report was used to analyze perception of price-value alignment. Finally, publicly available coverage policies at 10 of the largest insurance plans by lives covered were used to analyze the accessibility of DMD therapies.
RESULTS: Prices of current DMD therapies vary greatly from several hundreds of thousands to millions of dollars. While most of the top plans by lives covered allow the use of most DMD therapies, some smaller plans do not cover therapies such as Elevidys or Viltepso. ICER analysis found two DMD therapies (Emflaza and Exondys 51) providing low long-term value but still recommend payers to cover the therapies with prior authorizations. Most large plans do cover DMD therapies with prior authorizations based on trial criteria.
CONCLUSIONS: As the DMD disease landscape continues to evolve, manufacturers must ensure price is supported by value and data, regardless of being indicated in a rare space. This importance only intensifies as more therapies become available. While most plans currently cover DMD therapies, if value and price are perceived to be misaligned, stakeholders can attempt to restrict access to who they determine as the most appropriate patients for treatment (i.e., population studied in the trial) or only approve on a case-by-case basis (i.e., non-formulary).
Conference/Value in Health Info
Value in Health, Volume 27, Issue 6, S1 (June 2024)
Code
HPR95
Topic
Health Policy & Regulatory
Topic Subcategory
Coverage with Evidence Development & Adaptive Pathways, Insurance Systems & National Health Care, Pricing Policy & Schemes, Reimbursement & Access Policy
Disease
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Rare & Orphan Diseases