Expert assessment of the attributes of high-cost medications: a case study of CAR T-cell therapy in the gulf cooperation council.

BACKGROUND

The influx of high-cost advanced therapies, particularly CAR T-cell therapies, poses profound economic and operational challenges for healthcare systems in the Gulf Cooperation Council (GCC). As the region transitions from volume-based procurement to Value-Based Healthcare (VBHC), decision-makers must balance the urgent need for innovative curative treatments with fiscal sustainability and limited local health economics data.

METHODS

This study utilized a quantitative expert preference rating approach, using a rating-based Conjoint Analysis to assess the utility and relative importance of key value drivers for CAR T-cell therapies. Eighteen Key Opinion Leaders (KOLs) evaluated four hypothetical CAR T-cell product profiles. Individual ratings for sequentially presented attributes were collected from participants and subsequently analyzed using an ordinary least squares (OLS) transformation regression model based on Multi-Attribute Utility Theory (MAUT) to estimate part-worth utilities.

RESULTS

The analysis identified labeled indications and outcome-based agreements as the dominant drivers of decision-making utility, with markedly higher estimated utility values than upfront price and individual efficacy or safety metrics. Specifically, a cohort-level Outcome-Based Agreements model, where reimbursement is conditional on the response rate of a treated patient population, generated the single highest utility value (U = 24.07). In terms of structural attributes, experts demonstrated a statistically significant preference for comprehensive Patient Support Programs (PSPs) and Technology Transfer commitments (local manufacturing) over simple scientific guidance or basic discounts.

CONCLUSION

Decision-makers in the GCC prioritize risk-sharing mechanisms and broad therapeutic indications over traditional cost-containment strategies when evaluating high-cost CAR T-cell therapies. These findings suggest that to ensure equitable and sustainable patient access, policymakers must establish unified regulatory frameworks that facilitate performance-linked reimbursement models and incentivize the localization of advanced therapy infrastructure.