PRICE ANALYSIS OF GENE THERAPIES AUTHORIZED BY THE U.S. FOOD AND DRUG ADMINISTRATION
Author(s)
Anna Pelc, MS, MBA, MPH1, Mana Rahimian, PharmD, MS1, Adeola S. Bakare, BPharm, MS1, Lawrence M. Brown, PharmD, PhD, FAPhA1, Marc L. Fleming, BSPharm, MPH, MS, PhD1, Vaishali Shukla, MSc, MSRA, MS, PhD1, Rosa Rodriguez-monguio, PhD, MS2, Enrique Seoane-Vazquez, PhD1;
1Chapman University School of Pharmacy, Department of Pharmaceutical Economic and Policy, Irvine, CA, USA, 2University of California San Francisco, Department of Clinical Pharmacy, School of Pharmacy, San Francisco, CA, USA
1Chapman University School of Pharmacy, Department of Pharmaceutical Economic and Policy, Irvine, CA, USA, 2University of California San Francisco, Department of Clinical Pharmacy, School of Pharmacy, San Francisco, CA, USA
OBJECTIVES: The high cost of gene therapies threatens their accessibility. This study evaluated the prices of gene therapies approved in the US.
METHODS: We collected the Wholesale Acquisition Cost (WAC) (Red Book) and the Average Sales Price (ASP) (Centers for Medicare & Medicaid Services (CMS)) at market entry for all gene therapies authorized by the FDA as of January 1, 2026. Prices of therapies for chronic use were converted to WAC per year. Prices were adjusted for inflation using the Consumer Price Index (CPI). Data were analyzed descriptively.
RESULTS: The WAC was available for 26 (96.3%) therapies and the ASP for 11 (40.7%). The inflation-adjusted WAC (adj-WAC) ranged from $253,121 (revakinagene taroretcel) to $4,395,213 (atidarsagene autotemcel). Twelve (46.2%) therapies were priced under $1 million; two (7.7%) between $1-2 million; three (11.5%) between $2-3 million; seven (26.9%) between $3-4 million; and two (7.7%) over $4 million. The median adj-WAC was $1,227,477 (IQR $2,680,780). Tisagenlecleucel had different prices for two indications and onasemnogene abeparvovec for two routes of administration/patient populations. Alimentary tract and metabolism had the highest median adj-WAC (n=3; $4,020,785; IQR $562,773) and antineoplastic and immunomodulating agents had the lowest (11; $510,210; IQR $96,340). The median adj-WAC was $510,210 (15; IQR $213,863) for adult patients; $3,269,667 (3; IQR $850,312) for pediatric patients and $2,825,349 (10; IQR $1,650,752) for adult/pediatric patients. The median adj-WAC was $626,477 (3; IQR $546,403) for chronic use therapies and $2,316,863 (25; IQR $2,754,461) for single-use therapies. The prices increased an average of 2.22%±2.70% (unadjusted) and decreased by 0.37%±2.27% (inflation-adjusted) post-market. On average, the ASP was 0.84%±4.34% (range -9.02% to 10.19%) lower than the WAC.
CONCLUSIONS: When adjusted for inflation, prices remained relatively constant over time. Prices varied depending on therapeutic class, patient population, and duration of therapy. Gene therapies had similar WAC and ASP.
METHODS: We collected the Wholesale Acquisition Cost (WAC) (Red Book) and the Average Sales Price (ASP) (Centers for Medicare & Medicaid Services (CMS)) at market entry for all gene therapies authorized by the FDA as of January 1, 2026. Prices of therapies for chronic use were converted to WAC per year. Prices were adjusted for inflation using the Consumer Price Index (CPI). Data were analyzed descriptively.
RESULTS: The WAC was available for 26 (96.3%) therapies and the ASP for 11 (40.7%). The inflation-adjusted WAC (adj-WAC) ranged from $253,121 (revakinagene taroretcel) to $4,395,213 (atidarsagene autotemcel). Twelve (46.2%) therapies were priced under $1 million; two (7.7%) between $1-2 million; three (11.5%) between $2-3 million; seven (26.9%) between $3-4 million; and two (7.7%) over $4 million. The median adj-WAC was $1,227,477 (IQR $2,680,780). Tisagenlecleucel had different prices for two indications and onasemnogene abeparvovec for two routes of administration/patient populations. Alimentary tract and metabolism had the highest median adj-WAC (n=3; $4,020,785; IQR $562,773) and antineoplastic and immunomodulating agents had the lowest (11; $510,210; IQR $96,340). The median adj-WAC was $510,210 (15; IQR $213,863) for adult patients; $3,269,667 (3; IQR $850,312) for pediatric patients and $2,825,349 (10; IQR $1,650,752) for adult/pediatric patients. The median adj-WAC was $626,477 (3; IQR $546,403) for chronic use therapies and $2,316,863 (25; IQR $2,754,461) for single-use therapies. The prices increased an average of 2.22%±2.70% (unadjusted) and decreased by 0.37%±2.27% (inflation-adjusted) post-market. On average, the ASP was 0.84%±4.34% (range -9.02% to 10.19%) lower than the WAC.
CONCLUSIONS: When adjusted for inflation, prices remained relatively constant over time. Prices varied depending on therapeutic class, patient population, and duration of therapy. Gene therapies had similar WAC and ASP.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE382
Topic
Economic Evaluation
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Rare & Orphan Diseases