Why It Is Still Important to Ensure the Price is Right: Updates to Access Restrictions for Therapies Treating Duchenne Muscular Dystrophy
Author(s)
Kevin Brooks, MBA, Ayush Desai, BS, Kayla Lee, BA.
Red Nucleus, San Francisco, CA, USA.
Red Nucleus, San Francisco, CA, USA.
Presentation Documents
OBJECTIVES: Duchenne muscular dystrophy (DMD) is a rare and highly burdensome condition, yet some insurers elect to not cover these life-changing therapies. With increasing therapeutic competition, additional barriers may impact existing treatments. This research aims to provide an updated evaluation of trends between price and coverage outcomes for DMD therapies in the United States compared to a 2024 analysis.
METHODS: FDA databases were used to identify targeted therapies approved in DMD. Corticosteroids (Emflaza and Amagree) are excluded as they have significantly different value and price considerations. Publicly available coverage policies at 10 of the largest commercial plans (by covered lives) were analyzed to assess the access of DMD therapies, and subsequently compared to 2024 assessment. Therapies’ annual Wholesale Acquisition Costs were calculated and analyzed versus access and annual product sales to understand differences.
RESULTS: Most of the top plans by lives covered allow the use of DMD therapies with minimal or no changes to coverage policies. Generally, prior authorizations are aligned to trial criteria, including new entrant Duvyzat. Select plans have not updated criteria to include indication expansions and/or new product launches. Additionally, some plan(s) have updated criteria to allow coverage of Elevidys in ambulatory patients following regular FDA approval, but not for its non-ambulatory indication via accelerated approval.
CONCLUSIONS: Coverage criteria aligned to trial criteria is to ensure appropriate use. Duvyzat’s parity access to existing agents is likely a product of similar price-value alignment. If clinical benefit is unclear and price is high (e.g., non-ambulatory Elevidys), there is a prevailing risk of stakeholders adding restrictions (e.g., step edits) or only approving on a case-by-case basis (i.e., non-formulary). With an evolving treatment landscape, ensuring price and value are aligned continues to be a key consideration for manufacturers. For new entrants to avoid unnecessary commercialization hurdles, early market access and value strategy planning is essential.
METHODS: FDA databases were used to identify targeted therapies approved in DMD. Corticosteroids (Emflaza and Amagree) are excluded as they have significantly different value and price considerations. Publicly available coverage policies at 10 of the largest commercial plans (by covered lives) were analyzed to assess the access of DMD therapies, and subsequently compared to 2024 assessment. Therapies’ annual Wholesale Acquisition Costs were calculated and analyzed versus access and annual product sales to understand differences.
RESULTS: Most of the top plans by lives covered allow the use of DMD therapies with minimal or no changes to coverage policies. Generally, prior authorizations are aligned to trial criteria, including new entrant Duvyzat. Select plans have not updated criteria to include indication expansions and/or new product launches. Additionally, some plan(s) have updated criteria to allow coverage of Elevidys in ambulatory patients following regular FDA approval, but not for its non-ambulatory indication via accelerated approval.
CONCLUSIONS: Coverage criteria aligned to trial criteria is to ensure appropriate use. Duvyzat’s parity access to existing agents is likely a product of similar price-value alignment. If clinical benefit is unclear and price is high (e.g., non-ambulatory Elevidys), there is a prevailing risk of stakeholders adding restrictions (e.g., step edits) or only approving on a case-by-case basis (i.e., non-formulary). With an evolving treatment landscape, ensuring price and value are aligned continues to be a key consideration for manufacturers. For new entrants to avoid unnecessary commercialization hurdles, early market access and value strategy planning is essential.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR48
Topic
Health Policy & Regulatory
Topic Subcategory
Coverage with Evidence Development & Adaptive Pathways, Insurance Systems & National Health Care, Pricing Policy & Schemes, Reimbursement & Access Policy
Disease
SDC: Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), SDC: Rare & Orphan Diseases