Application of a Value Framework to Determine the Value of Prophylaxis Versus On-Demand Treatment in Adults with Hemophilia A in China.

INTRODUCTION

This study aimed to establish and apply a multicriteria value framework to determine the value of prophylaxis versus on‑demand treatment in adult patients with hemophilia A in China, which could enhance evidence-based care decisions.

METHODS

The framework was developed using key literature to identify dimensions and indicators for assessing the value of hemophilia A. We interviewed 21 stakeholders-including clinical experts, medical insurance experts, health economics and outcomes research (HEOR) experts, charity organization representatives, and patient advocacy organization representatives-to evaluate the relative importance of indicators. The interviewees also assessed the value of prophylaxis and on-demand treatments for adults, providing justification for their ratings. The analytic hierarchy process (AHP) was employed to calculate the weight of each indicator based on stakeholder ratings. A linear additive value function was used to calculate total value scores. The main outcomes of the study include the weighted indicators of the value framework and the comprehensive value scores for different hemophilia A care strategies.

RESULTS

The primary indicators in the value framework were clinical value, economic value, patient value, and social value. These were further broken down into nine secondary indicators. Overall, interviewees rated patient value highest (32.88%), followed by clinical value (30.08%), social value (22.25%), and economic value (14.79%). The adult prophylaxis strategy scored higher than on-demand treatment in all four primary value categories, with the largest difference observed in patient value. The total value score for adult prophylaxis (8.42) was higher than that for on-demand treatment (5.90), with an absolute difference of 2.52 (95% confidence interval [CI] 1.68-3.36).

CONCLUSION

The hemophilia value framework affirmed value of prophylaxis for adult patients with hemophilia A versus on-demand treatment, with benefit in terms of clinical value, economic value, patient value, and social value. The study also demonstrates that the value framework is an excellent tool for assisting stakeholders in decision-making that is grounded in patient-centered value in China.