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

IF 3.4 3区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Advances in Therapy Pub Date : 2025-02-28 DOI:10.1007/s12325-025-03131-9

Baoying Tan, Ailing Lin, Rong Han, Lu Bai, Jing Sun, Shanlian Hu, Jianwei Xuan

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Abstract

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.

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来源期刊

Advances in Therapy 医学-药学

CiteScore

7.20

自引率

2.60%

发文量

353

审稿时长

6-12 weeks

期刊介绍： Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.