Is mass screening for coeliac disease a wise use of resources? A health economic evaluation

Abstract Background Living with undiagnosed symptomatic coeliac disease is connected with deteriorated health, and persons with coeliac disease often wait a long time for their diagnosis. A mass screening would lower the delay, but its cost-effectiveness is still unclear. Our aim was to determine the cost-effectiveness of a coeliac disease mass screening at 12 years of age, taking a life course perspective on future benefits and drawbacks. Methods The cost-effectiveness was derived as cost per quality-adjusted life-year (QALY) using a Markov model. As a basis for our assumptions, we mainly used information from the Exploring the Iceberg of Celiacs in Sweden (ETICS) study, a school-based screening conducted in 2005/2006 and 2009/2010, where 13,279 12-year-old children participated and 240 were diagnosed with coeliac disease, and a study involving members of the Swedish Coeliac Association with 1031 adult participants. Results The cost for coeliac disease screening was 40,105 Euro per gained QALY. Sensitivity analyses support screening based on high compliance to a gluten-free diet, rapid progression from symptom-free coeliac disease to coeliac disease with symptoms, long delay from celiac disease with symptoms to diagnosis, and a low QALY score for undiagnosed coeliac disease cases. Conclusions A coeliac disease mass screening is cost-effective based on the commonly used threshold of 50,000 Euro per gained QALY. However, this is based on many assumptions, especially regarding the natural history of coeliac disease and the effects on long-term health for individuals with coeliac disease still eating gluten.

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Cladribine tablets are a cost-effective strategy in high-disease activity relapsing multiple sclerosis patients in Iran

Current Journal of Neurology ◽

10.18502/cjn.v20i3.7690 ◽

2021 ◽

Author(s):

Nayyereh Ayati ◽

Lora Fleifel ◽

Mohammad Ali Sahraian ◽

Shekoufeh Nikfar

Keyword(s):

Multiple Sclerosis ◽

Cost Effectiveness ◽

Disease Activity ◽

Cost Effective ◽

High Disease Activity ◽

Sensitivity Analyses ◽

Life Years ◽

Multiple Sclerosis Patients ◽

The Cost ◽

Relapsing Multiple Sclerosis

Background: Cladribine tablets are the foremost oral immune-reconstitution therapy for high disease activity relapsing multiple sclerosis (HDA-RMS). We aimed to assess the cost-effectiveness of cladribine tablets compared to natalizumab in patients with HDA-RMS in Iran. Methods: A 5-year cohort-based Markov model was developed with 11 expanded disability status score (EDSS) health states, including patients with HDA-RMS as on and off-treatment. All costs were identified from the literature and expert opinion and were measured in Iranian Rial rates, changed to the 2020 USD rate and were discounted by 7.2%. Quality adjusted life years (QALY), discounted by 3.5%, and life years gained (LYG) were adopted to measure efficacy. The final results were presented as incremental cost-effectiveness ratio that was compared to a national willingness to pay (WTP) threshold of 1 to 3 gross domestic product (GDP) per capita. Deterministic and probabilistic sensitivity analyses (D/PSA) were employed to evaluate uncertainty. Results: Cladribine tablets dominated natalizumab and yielded 6,607 USD cost-saving and 0.003 additional QALYs per patient. LYG was comparable. The main cost component was drug acquisition cost in both arms. DSA indicated the sensitivity of the results to the cost discount rates and also the patients’ body weight; while they were less sensitive to the main clinical variables. PSA indicated that cladribine tablets were cost-effective in Iran, with a probability of 57.5% and 58.6% at lower and higher limits of threshold, respectively. Conclusion: Cladribine tablets yielded higher QALYs and lower costs compared to natalizumab, in patients with HDA-RMS in Iran.

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First-Line Pembrolizumab Plus Chemotherapy for Extensive-Stage Small-Cell Lung Cancer: A Cost-Effectiveness Analysis

10.21203/rs.3.rs-725799/v1 ◽

2021 ◽

Author(s):

Youwen Zhu ◽

Huabin Hu ◽

Dong Ding ◽

Shuosha Li ◽

Mengting Liao ◽

...

Keyword(s):

Cost Effectiveness ◽

Cost Effective ◽

Small Cell ◽

Sensitivity Analyses ◽

Small Cell Lung ◽

First Line ◽

The Us ◽

Extensive Stage ◽

The Cost ◽

First Line Treatment

Abstract Background：The phase III clinical trial Keynote-604 indicated that pembrolizumab plus chemotherapy could generate clinical benefits in Extensive-Stage Small-Cell Lung Cancer (ES-SCLC). We aim to evaluate the cost-effectiveness of pembrolizumab plus chemotherapy as the first-line treatment of ES-SCLC from the United States (US) payers’ perspective.Methods: A synthetical Markov model was used to evaluate cost and effectiveness of pembrolizumab plus platinum-etoposide (EP) versus EP in first-line therapy for ES-SCLC from the data of Keynote-604. Lifetime costs life-years (LYs), quality adjusted LYs (QALYs), and incremental cost-effectiveness ratios (ICERs) were estimated. One-way and probabilistic sensitivity analyses were performed. In addition, We also considered subgroup cost-effectiveness.Results: Pembrolizumab plus EP resulted in additional 0.18 QALYs (0.32 LYs) and corresponding incremental costs $113,625, resulting an ICER of $647,509 per QALY versus EP. The most influential factor in this model was the cost of pembrolizumab. Probabilistic sensitivity analysis showed there was 0% probability that pembrolizumab combination chemotherapy was cost-effective at willingness-to-pay (WTP) values of $150,000 per QALY in the US. The results of subgroup probabilistic sensitivity analyses suggested that all subgroups were not cost-effective.Conclusion: From the perspective of the US payer, pembrolizumab plus EP is not a cost-effective option as first-line treatment for patients with ES-SCLC at a WTP threshold of $150,000 per QALY.

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P14.41 Cost-effectiveness of FET PET for early treatment response assessment in glioma patients following adjuvant temozolomide chemotherapy

Neuro-Oncology ◽

10.1093/neuonc/noab180.158 ◽

2021 ◽

Vol 23 (Supplement_2) ◽

pp. ii46-ii46

Author(s):

J Rosen ◽

G Ceccon ◽

E K Bauer ◽

J M Werner ◽

C Kabbasch ◽

...

Keyword(s):

Cost Effectiveness ◽

Cost Effective ◽

Response Assessment ◽

Sensitivity Analyses ◽

Tree Model ◽

Fet Pet ◽

Conventional Mri ◽

Adjuvant Temozolomide ◽

The Cost ◽

Temozolomide Chemotherapy

Abstract BACKGROUND In light of increasing healthcare costs, higher medical expenses should be justified socio-economically. Therefore, we calculated the effectiveness and cost-effectiveness of PET using the radiolabeled amino acid O-(2-[18F]-fluoroethyl)-L-tyrosine (FET) compared to conventional MRI for early identification of responders to adjuvant temozolomide chemotherapy. A recent study in IDH-wildtype glioma patients suggested that after two cycles, FET-PET parameter changes predicted a significantly longer survival while MRI changes were not significant. MATERIALS AND METHODS To determine the effectiveness and cost-effectiveness of serial FET-PET imaging, we analyzed published clinical data and calculated the associated costs in the context of the German healthcare system.Based on a decision-tree model, FET-PET and MRI’s effectiveness was calculated, i.e., the probability to correctly identify a responder as defined by an overall survival ≥15 months. To determine the cost-effectiveness, the incremental cost-effectiveness ratio (ICER) was calculated, i.e., the cost for each additionally identified responder by FET-PET who would have remained undetected by MRI. The robustness of the results was tested by deterministic and probabilistic (Monte Carlo simulation) sensitivity analyses. RESULTS Compared to MRI, FET-PET increases the rate of correctly identified responders to chemotherapy by 26%; thus, four patients need to be examined by FET-PET to identify one additional responder. Considering the respective cost for serial FET-PET and MRI, the ICER resulted in €4,396.83 for each additional correctly identified responder by FET-PET. The sensitivity analyses confirmed the robustness of the results. CONCLUSION In contrast to conventional MRI, the model suggests that FET PET is cost-effective in terms of ICER values. Concerning the high cost of temozolomide, the integration of FET-PET has the potential to avoid premature chemotherapy discontinuation at a reasonable cost.

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Modelling the cost-effectiveness of person-centred care for patients with acute coronary syndrome

The European Journal of Health Economics ◽

10.1007/s10198-020-01230-8 ◽

2020 ◽

Vol 21 (9) ◽

pp. 1317-1327 ◽

Cited By ~ 1

Author(s):

Laura Pirhonen ◽

Hanna Gyllensten ◽

Andreas Fors ◽

Kristian Bolin

Keyword(s):

Monte Carlo ◽

Acute Coronary Syndrome ◽

Cost Effectiveness ◽

Usual Care ◽

Time Perspective ◽

Controlled Trial ◽

Cost Effective ◽

Sensitivity Analyses ◽

Coronary Syndrome ◽

The Cost

Abstract Background Person-centred care has been shown to be cost-effective compared to usual care for several diseases, including acute coronary syndrome, in a short-term time perspective (< 2 years). The cost-effectiveness of person-centred care in a longer time perspective is largely unknown. Objectives To estimate the mid-term cost-effectiveness of person-centred care compared to usual care for patients (< 65) with acute coronary syndrome, using a 2-year and a 5-year time perspective. Methods The mid-term cost-effectiveness of person-centred care compared to usual care was estimated by projecting the outcomes observed in a randomized-controlled trial together with data from health registers and data from the scientific literature, 3 years beyond the 2-year follow-up, using the developed simulation model. Probabilistic sensitivity analyses were performed using Monte Carlo simulation. Results Person-centred care entails lower costs and improved effectiveness as compared to usual care, for a 2-year time and a 5-year perspective. Monte Carlo simulations suggest that the likelihoods of the person-centred care being cost-effective compared to usual care were between 80 and 99% and between 75 and 90% for a 2-year and a 5-year time perspective (using a 500,000 SEK/QALY willingness-to-pay threshold). Conclusions Person-centred care was less costly and more effective compared to usual care in a 2-year and a 5-year time perspective for patients with acute coronary syndrome under the age of 65.

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A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK

International Journal of Neonatal Screening ◽

10.3390/ijns5030028 ◽

2019 ◽

Vol 5 (3) ◽

pp. 28 ◽

Cited By ~ 2

Author(s):

Alice Bessey ◽

James Chilcott ◽

Joanna Leaviss ◽

Carmen de la Cruz ◽

Ruth Wong

Keyword(s):

Cost Effectiveness ◽

Severe Combined Immunodeficiency ◽

Cost Effective ◽

Sensitivity Analyses ◽

Combined Immunodeficiency ◽

Tree Model ◽

Life Years ◽

The Uk ◽

The Cost ◽

The Impact

Severe combined immunodeficiency (SCID) can be detected through newborn bloodspot screening. In the UK, the National Screening Committee (NSC) requires screening programmes to be cost-effective at standard UK thresholds. To assess the cost-effectiveness of SCID screening for the NSC, a decision-tree model with lifetable estimates of outcomes was built. Model structure and parameterisation were informed by systematic review and expert clinical judgment. A public service perspective was used and lifetime costs and quality-adjusted life years (QALYs) were discounted at 3.5%. Probabilistic, one-way sensitivity analyses and an exploratory disbenefit analysis for the identification of non-SCID patients were conducted. Screening for SCID was estimated to result in an incremental cost-effectiveness ratio (ICER) of £18,222 with a reduction in SCID mortality from 8.1 (5–12) to 1.7 (0.6–4.0) cases per year of screening. Results were sensitive to a number of parameters, including the cost of the screening test, the incidence of SCID and the disbenefit to the healthy at birth and false-positive cases. Screening for SCID is likely to be cost-effective at £20,000 per QALY, key uncertainties relate to the impact on false positives and the impact on the identification of children with non-SCID T Cell lymphopenia.

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Meta-analysis and cost-effectiveness of second-line antiepileptic drugs for status epilepticus

Neurology ◽

10.1212/wnl.0000000000007503 ◽

2019 ◽

Vol 92 (20) ◽

pp. e2339-e2348 ◽

Cited By ~ 13

Author(s):

Iván Sánchez Fernández ◽

Marina Gaínza-Lein ◽

Nathan Lamb ◽

Tobias Loddenkemper

Keyword(s):

Status Epilepticus ◽

Cost Effectiveness ◽

Antiepileptic Drugs ◽

Meta Analysis ◽

Cost Effective ◽

Sensitivity Analyses ◽

Convulsive Status Epilepticus ◽

Decision Analysis Model ◽

Analysis Model ◽

The Cost

ObjectiveCompare the cost and effectiveness of nonbenzodiazepine antiepileptic drugs (non-BZD AEDs) for treatment of BZD-resistant convulsive status epilepticus (SE).MethodsDecision analysis model populated with effectiveness data from a systematic review and meta-analysis of the literature, and cost data from publicly available prices. The primary outcome was cost per seizure stopped ($/SS). Sensitivity analyses evaluated the robustness of the results across a wide variation of the input parameters.ResultsWe included 24 studies with 1,185 SE episodes. The most effective non-BZD AED was phenobarbital (PB) with a probability of SS of 0.8 (95% confidence interval [CI]: 0.69–0.88), followed by valproate (VPA) (0.71 [95% CI: 0.61–0.79]), lacosamide (0.66 [95% CI: 0.51–0.79]), levetiracetam (LEV) (0.62 [95% CI: 0.5–0.73]), and phenytoin/fosphenytoin (PHT) (0.53 [95% CI: 0.39–0.67]). In pairwise comparisons, PB was more effective than PHT (p = 0.002), VPA was more effective than PHT (p = 0.043), and PB was more effective than LEV (p = 0.018). The most cost-effective non-BZD AED was LEV (incremental cost-effectiveness ratio [ICER]: $18.55/SS), followed by VPA (ICER: $94.44/SS), and lastly PB (ICER: $847.22/SS). PHT and lacosamide were not cost-effective compared to the other options. Sensitivity analyses showed marked overlap in cost-effectiveness, but PHT was consistently less cost-effective than LEV, VPA, and PB.ConclusionVPA and PB were more effective than PHT for SE. There is substantial overlap in the cost-effectiveness of non-BZD AEDs for SE, but available evidence does not support the preeminence of PHT, neither in terms of effectiveness nor in terms of cost-effectiveness.

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P0434COST-EFFECTIVENESS OF MAINTANCE THERAPY WITH AZATHIOPRIME VERSUS RITUXIMAB (TAILORED OR FIXED-SCHEDULE) IN ADULTS WITH GENERALIZED ANCA VASCULITIS IN COLOMBIA

Nephrology Dialysis Transplantation ◽

10.1093/ndt/gfaa142.p0434 ◽

2020 ◽

Vol 35 (Supplement_3) ◽

Author(s):

Kateir Mariel Contreras ◽

Viviana Orozco Ortiz ◽

Eduardo José Puche ◽

Paola Karina Garcia ◽

Camilo Alberto Gonzalez ◽

...

Keyword(s):

Cost Effectiveness ◽

Lower Cost ◽

Transition Probabilities ◽

Cost Effective ◽

Sensitivity Analyses ◽

Fixed Dose ◽

National Guidelines ◽

Anca Vasculitis ◽

Life Years ◽

The Cost

Abstract Background and Aims Azathioprine has been for decades the drug of choice for maintenance therapy in patients with generalized ANCA vasculitis in remission. However, recent studies show that rituximab, a high-cost biological agent, which can be administrated in two different schedules, might be more effective, so it is necessary to know the cost- effectiveness. Our goal was to compare the cost-effectiveness of the 3 maintenance schemes: standard therapy with azathioprine; fixed-dose rituximab and rituximab tailored according to CD19 lymphocyte level and ANCA titres, from the perspective of the Colombian healthcare system. Method We designed a 5-year annual cycle Markov model with the following stages: remission, minor relapse, mayor relapse and death. Transition probabilities were obtained from a systematic review of the literature (Scopus and Pubmed). Following national guidelines for economic studies, costs (in 2018, 1 euro = 3489 Colombian pesos) were estimated based on national drug registries, and official tariff manuals for procedures and other resources. Main outcome was quality-adjusted life years (QALY), using lupus nephropathy as a proxy; values were obtained from Tufts CEA Registry and validated by local expert panel through a modified Delphi technique. Cost-effectiveness threshold was three-times per capita GDP (16.872 euros). Discount rate was 5%. Univariate and probabilistic sensitivity analyses were performed. Results Overall discounted 5-years costs were € 1149 for azathioprine; € 4025 for tailored rituximab and € 5221 for fixed rituximab. QALY gains were 2.94, 3.63 and 3.64, respectively. Both tailored and fixed rituximab were cost-effective (cost per QALY gained: € 4168 and € 5817 respectively), but tailored dosing was preferable due to its lower cost. Sensitivity analyses did not modify these results significantly. Conclusion To our knowledge this is the first economic evaluation that compare azathioprine with tailored and fixed rituximab regimens as a vasculitis maintenance treatment in adults with ANCA generalized. Due to its lower effectiveness azathioprine should not be the first line of treatment. Tailored rituximab should be a better option than fixed schedule due to its lower cost with similar effectiveness.

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P14.12 The cost-effectiveness of tumor-treating fields in patients with newly diagnosed glioblastoma

Neuro-Oncology ◽

10.1093/neuonc/noz126.247 ◽

2019 ◽

Vol 21 (Supplement_3) ◽

pp. iii68-iii69

Author(s):

X Armoiry ◽

P Auguste ◽

C Dussart ◽

J Guyotat ◽

M Connock

Keyword(s):

Cost Effectiveness ◽

Cost Effective ◽

Survival Model ◽

Sensitivity Analyses ◽

Base Case ◽

Tumor Treating Fields ◽

Kaplan Meier ◽

Life Years ◽

Secondary Analyses ◽

The Cost

Abstract BACKGROUND The addition of novel therapy “Tumor-Treating fields” (TTF) to standard radio-chemotherapy with Temozolomide (TMZ) has recently shown superiority over conventional TMZ regimen in patients with glioblastoma. Despite the clinical benefit of TTF, there is a strong concern regarding the cost of this new treatment. A first cost-effectiveness analysis, which was published in 2016, was based on effectiveness outcomes from an interim analysis of the pivotal trial and used a “standard” Markov model. Here, we aimed to update the cost-effectiveness evaluation using a partitioned survival model design and using the latest effectiveness data. MATERIAL AND METHODS A partitioned survival model was developed with three mutually exclusive health states: stable disease, progressive disease, and dead. Parametric models were fitted to the Kaplan-Meier data for overall and progression-free survival. These generated clinically plausible extrapolations beyond the observed data. The perspective of the French national health insurance was adopted and the time horizon was 20 years. Base case results were expressed as cost/life-years (LY) gained (LYG). Secondary analyses were undertaken, with the results presented as cost/per quality adjusted life years (QALY) gained. Last, we undertook deterministic and probabilistic sensitivity analyses. RESULTS After applying 4% annual discounting of benefits and costs, the base case model generated incremental benefit of 0.507 LY at a incremental cost of €258,695 yielding an incremental cost effectiveness ratio (ICER) of €510,273 / LYG. Secondary analyses yielded an ICER of €667,173/QALY. Sensitivity analyses and bootstrapping methods showed the model was relatively robust. The model was sensitive to TTF device costs and the parametric model fitted to the Kaplan-Meier data for overall survival. The cost-effectiveness acceptability curve showed TTF has 0% of being cost-effective under conventional thresholds. CONCLUSION Using a partitioned survival model, uprated costs and more mature survival outcomes, TTF when compared to standard radio-chemotherapy with TMZ is not likely to be cost-effective. This has major implications in terms of access of newly eligible patients

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Cost-effectiveness of an insertable cardiac monitor in a high-risk population in the UK

Open Heart ◽

10.1136/openhrt-2019-001037 ◽

2019 ◽

Vol 6 (1) ◽

pp. e001037 ◽

Cited By ~ 1

Author(s):

Claudia I Rinciog ◽

Laura M Sawyer ◽

Alexander Diamantopoulos ◽

Mitchell S V Elkind ◽

Matthew Reynolds ◽

...

Keyword(s):

Cost Effectiveness ◽

High Risk ◽

Oral Anticoagulants ◽

Cost Effective ◽

Sensitivity Analyses ◽

High Risk Population ◽

Risk Population ◽

Life Years ◽

The Uk ◽

The Cost

ObjectiveTo evaluate the cost-effectiveness of insertable cardiac monitors (ICMs) compared with standard of care (SoC) for detecting atrial fibrillation (AF) in patients at high risk of stroke (CHADS2 >2), using a UK National Health Service (NHS) perspective.MethodsUsing patient characteristics and clinical data from the REVEAL AF trial, a Markov model assessed the cost-effectiveness of detecting AF with an ICM compared with SoC. Costs and benefits were extrapolated across modelled patient lifetime. Ischaemic and haemorrhagic strokes, intracranial and extracranial haemorrhages and minor bleeds were modelled. Diagnostic and device costs were included, plus costs of treating stroke and bleeding events and costs of oral anticoagulants (OACs). Costs and health outcomes, measured as quality-adjusted life years (QALYs), were discounted at 3.5% per annum. One-way deterministic and probabilistic sensitivity analyses (PSA) were undertaken.ResultsThe total per-patient cost for ICM was £13 360 versus £11 936 for SoC (namely, annual 24 hours Holter monitoring). ICMs generated a total of 6.50 QALYs versus 6.30 for SoC. The incremental cost-effectiveness ratio (ICER) was £7140/QALY gained, below the £20 000/QALY acceptability threshold. ICMs were cost-effective in 77.4% of PSA simulations. The number of ICMs needed to prevent one stroke was 21 and to cause a major bleed was 37. ICERs were sensitive to assumed proportions of patients initiating or discontinuing OAC after AF diagnosis, type of OAC used and how intense the traditional monitoring was assumed to be under SoC.ConclusionsThe use of ICMs to identify AF in a high-risk population is cost-effective for the UK NHS.

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Cost-effectiveness analysis of tranexamic acid for the treatment of traumatic brain injury, based on the results of the CRASH-3 randomised trial: a decision modelling approach

BMJ Global Health ◽

10.1136/bmjgh-2020-002716 ◽

2020 ◽

Vol 5 (9) ◽

pp. e002716

Author(s):

Jack Williams ◽

Ian Roberts ◽

Haleema Shakur-Still ◽

Fiona E Lecky ◽

Rizwana Chaudhri ◽

...

Keyword(s):

Cost Effectiveness ◽

Tranexamic Acid ◽

Randomised Trial ◽

Cost Effective ◽

Sensitivity Analyses ◽

Risk Of Death ◽

Life Years ◽

Markov Decision Model ◽

The Uk ◽

The Cost

IntroductionAn estimated 69 million traumatic brain injuries (TBI) occur each year worldwide, with most in low-income and middle-income countries. The CRASH-3 randomised trial found that intravenous administration of tranexamic acid within 3 hours of injury reduces head injury deaths in patients sustaining a mild or moderate TBI. We examined the cost-effectiveness of tranexamic acid treatment for TBI.MethodsA Markov decision model was developed to assess the cost-effectiveness of treatment with and without tranexamic acid, in addition to current practice. We modelled the decision in the UK and Pakistan from a health service perspective, over a lifetime time horizon. We used data from the CRASH-3 trial for the risk of death during the trial period (28 days) and patient quality of life, and data from the literature to estimate costs and long-term outcomes post-TBI. We present outcomes as quality-adjusted life years (QALYs) and 2018 costs in pounds for the UK, and US dollars for Pakistan. Incremental cost-effectiveness ratios (ICER) per QALY gained were estimated, and compared with country specific cost-effective thresholds. Deterministic and probabilistic sensitivity analyses were also performed.ResultsTranexamic acid was highly cost-effective for patients with mild TBI and intracranial bleeding or patients with moderate TBI, at £4288 per QALY in the UK, and US$24 per QALY in Pakistan. Tranexamic acid was 99% and 98% cost-effective at the cost-effectiveness thresholds for the UK and Pakistan, respectively, and remained cost-effective across all deterministic sensitivity analyses. Tranexamic acid was even more cost-effective with earlier treatment administration. The cost-effectiveness for those with severe TBI was uncertain.ConclusionEarly administration of tranexamic acid is highly cost-effective for patients with mild or moderate TBI in the UK and Pakistan, relative to the cost-effectiveness thresholds used. The estimated ICERs suggest treatment is likely to be cost-effective across all income settings globally.

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