SPENDING ON GENE THERAPIES FOR RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA
Author(s)
Adam J. Raymakers, BSc, MSc, PhD1, Aaron S. Kesselheim, MD, MPH, JD2, William B. Feldman, MD, DPhil, MPH3;
1Division of Pharmacoepidemiology and Pharmacoeconomics, Harvard Medical School & Brigham and Women's, Boston, MA, USA, 2Division of Pharmacoepidemiology and Pharmacoeconomics, Harvard Medical School & Brigham and Women's, Program on Regulation, Therapeutics, and Law, Boston, MA, USA, 3David Geffen School of Medicine at UCLA, Los Angeles, CA, USA
1Division of Pharmacoepidemiology and Pharmacoeconomics, Harvard Medical School & Brigham and Women's, Boston, MA, USA, 2Division of Pharmacoepidemiology and Pharmacoeconomics, Harvard Medical School & Brigham and Women's, Program on Regulation, Therapeutics, and Law, Boston, MA, USA, 3David Geffen School of Medicine at UCLA, Los Angeles, CA, USA
OBJECTIVES: The Food and Drug Administration (FDA) has recently approved two gene therapies for recessive dystrophic epidermolysis bullosa (RDEB): beremagene geperpavec (B-VEC; Vyjuvek), a once-weekly drug with a yearly cost of $657,000 (approved in 2023), and prademagene zamikeracel (PZ-CEL; Zevaskyn), a one-time therapy costing $3.1 million (approved in 2025). The differing costs and dosing schedules of these two therapies will present payers with difficult decisions about coverage. This analysis sought to estimate spending on each therapy under a variety of potential scenarios.
METHODS: We estimated spending on treatment for RDEB after the introduction of PZ-CEL for the treatment of patients based previous analysis estimating US health care system spending on B-VEC for both RDEB and DDEB. The projected costs of treatment were calculated based on the current list price of B-VEC ($657,000 per year) and PZ-CEL ($3.1 million). In the primary analysis, we estimated the 5-year aggregate costs of B-VEC versus PZ-CEL. We further estimated potential lifetime costs associated with each therapy.
RESULTS: In year one, if all patients receive either B-VEC or PZ-CEL, estimated spending would be $290 million and $1.4 billion, respectively. By year five, spending for the PZ-CEL and B-VEC converges at $1.5 billion due to ongoing B-VEC therapy. The estimated lifetime treatment costs per patient would range from $32.8M to $49.2M for those receiving B-VEC, depending on improvements in life expectancy.
CONCLUSIONS: This analysis quantifies the trade-offs that payers face when balancing up-front costs with those accruing over longer durations for high-priced gene therapies in RDEB and may consider alternative payment mechanisms or new negotiation models.
METHODS: We estimated spending on treatment for RDEB after the introduction of PZ-CEL for the treatment of patients based previous analysis estimating US health care system spending on B-VEC for both RDEB and DDEB. The projected costs of treatment were calculated based on the current list price of B-VEC ($657,000 per year) and PZ-CEL ($3.1 million). In the primary analysis, we estimated the 5-year aggregate costs of B-VEC versus PZ-CEL. We further estimated potential lifetime costs associated with each therapy.
RESULTS: In year one, if all patients receive either B-VEC or PZ-CEL, estimated spending would be $290 million and $1.4 billion, respectively. By year five, spending for the PZ-CEL and B-VEC converges at $1.5 billion due to ongoing B-VEC therapy. The estimated lifetime treatment costs per patient would range from $32.8M to $49.2M for those receiving B-VEC, depending on improvements in life expectancy.
CONCLUSIONS: This analysis quantifies the trade-offs that payers face when balancing up-front costs with those accruing over longer durations for high-priced gene therapies in RDEB and may consider alternative payment mechanisms or new negotiation models.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE486
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Rare & Orphan Diseases, SDC: Sensory System Disorders (Ear, Eye, Dental, Skin), STA: Genetic, Regenerative & Curative Therapies