Evidence map›Paper›PMID 40016439›Full record

ArticleAdvances in therapy2025

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

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

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Article in Advances in therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

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3 · Its place in the literature

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4 · The record

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5 · Who and what money

Authors and funding

7 authors.

Baoying TanHealth Economic Research Institute, School of Pharmacy, Sun Yat-Sen University, Guangzhou, 510006, China.ORCID 0000-0001-7339-5101
Ailing LinHealth Economic Research Institute, School of Pharmacy, Sun Yat-Sen University, Guangzhou, 510006, China.ORCID 0000-0002-7667-8294
Rong HanPharmaceuticals, Bayer Healthcare Company Ltd, Beijing, 100020, China.ORCID 0009-0004-1589-6636
Lu BaiPharmaceuticals, Bayer Healthcare Company Ltd, Beijing, 100020, China.ORCID 0009-0006-1911-4245
Jing SunDepartment of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, 510515, China.ORCID 0000-0002-5130-4835
Shanlian HuSchool of Public Health, Fudan University, Shanghai, 200032, China.ORCID 0000-0002-6240-695X
Jianwei XuanHealth Economic Research Institute, School of Pharmacy, Sun Yat-Sen University, Guangzhou, 510006, China. xuanjw3@mail.sysu.edu.cn.ORCID 0000-0003-4800-4935

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionThis 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.

methodsThe 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.

resultsThe 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).

conclusionThe 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.

Indexed as

Hemophilia AAdultChinaCost-Benefit AnalysisHumansMaleAdultChinaHemophiliaProphylaxisRecombinant factor VIIIValue framework

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.