ALIGNMENT OF ESTIMATED ACQUISITION COSTS IN U.S. GENE THERAPY COST EFFECTIVENESS ANALYSES WITH STANDARD PRICE BENCHMARKS
Author(s)
Adeola S. Bakare, BPharm, MS1, Anna Pelc, MS, MBA, MPH2, Mana Rahimian, PharmD, MS2, Lawrence M. Brown, PharmD, PhD; FAPhA2, Marc Fleming, BS, MPH, RPh, PhD2, Vaishali Shukla, MSc, MSRA, MS, PhD2, Rosa Rodriguez-monguio, MS, PhD3, Enrique Seoane-Vazquez, PhD2;
1Chapman University School of Pharmacy, Pharmaceutical Economic and Policy, Irvine, CA, USA, 2Chapman university School of Pharmacy, Pharmaceutical Economics and Policy, Irvine, CA, USA, 3University of California San Francisco, Clinical Pharmacy, School of Pharmacy, San Francisco, CA, USA
1Chapman University School of Pharmacy, Pharmaceutical Economic and Policy, Irvine, CA, USA, 2Chapman university School of Pharmacy, Pharmaceutical Economics and Policy, Irvine, CA, USA, 3University of California San Francisco, Clinical Pharmacy, School of Pharmacy, San Francisco, CA, USA
OBJECTIVES: Cost-effectiveness analysis (CEA) guides coverage and reimbursement of gene therapies (GT). The use of price benchmarks as estimated acquisition costs (EACs) remains unclear. This study assessed the alignment of EACs used in U.S. gene therapy CEA studies with available price benchmarks.
METHODS: We extracted EACs from articles assessing the cost-effectiveness of GT in the U.S. identified in PubMed on November 4, 2025. The EAC for each GT was compared with the wholesale acquisition cost (WAC) and average wholesale price (AWP) from RedBook, and the average sales price (ASP) reported by the Centers for Medicare and Medicaid Services. Results were descriptively summarized.
RESULTS: There were 79 CEA studies listed in PubMed, of which 34 (54.4%) were original research and reported the EAC for 43 GT. Seven studies assessed multiple therapies. There were 14 GT (51.9% of 27 FDA-authorized GT) with a published CEA. Axicabtagene was evaluated in 13 CEA, and 7 GT were evaluated in only one CEA.The EAC represented a mean±standard deviation of 97.4%±11.9% (range 69.7%-119.7%) of the WAC and 81.3%±9.8% (range 59.1%-99.7%) of the AWP. The WAC reported by manufacturers was fixed at 83.3% of the AWP. There were 13 (30.2%) EACs lower, 17 (39.5%) equal, and 13 (30.2%) higher than the WAC. All EACs were below the AWP. ASP date was unavailable for inclusion as an EAC in CEAs. As of December 31, 2025, the ASP was available for 6 therapies and represented 96.2%±2.5% of the WAC and 86.8%±3.0% of the AWP.
CONCLUSIONS: Considering the high cost of GT, small percentage differences in EACs may result in changes in CEA results. The estimate acquisition costs of GT were generally similar to the WAC. The ASP is the preferred price for establishing the EAC of GT; however, given its limited availability, the use of WAC is recommended.
METHODS: We extracted EACs from articles assessing the cost-effectiveness of GT in the U.S. identified in PubMed on November 4, 2025. The EAC for each GT was compared with the wholesale acquisition cost (WAC) and average wholesale price (AWP) from RedBook, and the average sales price (ASP) reported by the Centers for Medicare and Medicaid Services. Results were descriptively summarized.
RESULTS: There were 79 CEA studies listed in PubMed, of which 34 (54.4%) were original research and reported the EAC for 43 GT. Seven studies assessed multiple therapies. There were 14 GT (51.9% of 27 FDA-authorized GT) with a published CEA. Axicabtagene was evaluated in 13 CEA, and 7 GT were evaluated in only one CEA.The EAC represented a mean±standard deviation of 97.4%±11.9% (range 69.7%-119.7%) of the WAC and 81.3%±9.8% (range 59.1%-99.7%) of the AWP. The WAC reported by manufacturers was fixed at 83.3% of the AWP. There were 13 (30.2%) EACs lower, 17 (39.5%) equal, and 13 (30.2%) higher than the WAC. All EACs were below the AWP. ASP date was unavailable for inclusion as an EAC in CEAs. As of December 31, 2025, the ASP was available for 6 therapies and represented 96.2%±2.5% of the WAC and 86.8%±3.0% of the AWP.
CONCLUSIONS: Considering the high cost of GT, small percentage differences in EACs may result in changes in CEA results. The estimate acquisition costs of GT were generally similar to the WAC. The ASP is the preferred price for establishing the EAC of GT; however, given its limited availability, the use of WAC is recommended.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE64
Topic
Economic Evaluation
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Oncology, SDC: Rare & Orphan Diseases, STA: Genetic, Regenerative & Curative Therapies, STA: Personalized & Precision Medicine