Evidence map›Paper›PMID 42824885›Full record

ArticleJournal of pharmaceutical policy and practice2026

Cost-effectiveness and budget impact of Luspatercept in Thai adults with transfusion-dependent beta-thalassaemia: a multi-state Markov model based on national claims data.

Khachen Kongpakwattana, Peerapon Wong, Nattiya Teawtrakul, Pimlak Charoenkwan, Nilawan Upakdee

Abstract read
In one paragraph

Article in Journal of pharmaceutical policy and practice, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
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

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors.

Khachen KongpakwattanaDepartment of Social and Administrative Pharmacy, Faculty of Pharmaceutical Sciences, Chulalongkorn University, Bangkok, Thailand.ORCID https://orcid.org/0000-0001-8030-8096
Peerapon WongThalassemia Research Unit, Naresuan University Hospital, Faculty of Medicine, Naresuan University, Phitsanulok, Thailand.
Nattiya TeawtrakulDepartment of Internal Medicine, Faculty of Medicine, Khon Kaen University, Khon Kaen, Thailand.
Pimlak CharoenkwanDepartment of Internal Medicine, Faculty of Medicine, Chiang Mai University, Chiang Mai, Thailand.
Nilawan UpakdeeDepartment of Pharmacy Practice, Faculty of Pharmaceutical Sciences, Naresuan University, Phitsanulok, Thailand.ORCID https://orcid.org/0009-0009-8640-0614

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Luspatercept is a first-in-class erythroid maturation agent that reduces transfusion burden in adults with transfusion-dependent beta-thalassaemia (TDT). However, its cost-effectiveness has not been evaluated in middle-income settings. This study aimed to evaluate the cost-effectiveness and budget impact of Luspatercept for TDT in Thai adults. Methods: A hybrid decision tree and lifetime Markov cohort model was developed to compare Luspatercept plus best supportive care (BSC) with BSC alone from a societal perspective. Age-specific 12-week transition probabilities across three transfusion burden states (low, medium, and high) and death were derived from a multi-state Markov survival model fitted to national administrative claims data. Treatment efficacy was based on the phase 3 BELIEVE trial. Three treatment eligibility scenarios were evaluated: universal (all patients), targeted (medium and high transfusion burden), and restricted (high transfusion burden only). Deterministic, probabilistic, threshold price, and budget impact analyses were performed. Results: In the universal scenario, Luspatercept produced an incremental gain of 0.205 QALYs and an additional cost of 955,053 THB (29,048 US$) per patient, resulting in an incremental cost-effectiveness ratio (ICER) of 4,650,427 THB/QALY (141,442 US$/QALY). This value exceeds the Thai willingness-to-pay threshold of 160,000 THB/QALY by approximately 29-fold. Restricting eligibility to patients with higher transfusion burden did not improve cost-effectiveness; in the restricted scenario, Luspatercept was dominated. A price reduction of 90.5% would be required for Luspatercept to become cost-effective, which would also reduce the five-year cumulative budget impact from 2,505 million THB to 296 million THB. Conclusion: Luspatercept is not cost-effective for TDT in Thailand at its current price across all scenarios evaluated. A substantial price reduction would be necessary, though this would bring the budget impact to a manageable level. These findings may help inform pricing and reimbursement decisions in Thailand and other middle-income countries.

Indexed as

beta-thalassaemiabudget impactcost-effectivenessLuspaterceptThailand

Identifiers

PMID42824885
PMCPMC13629779

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Registered trials

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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.