Clinical and economic impact of current ALK rearrangement testing in Spain compared with a hypothetical no-testing scenario

Abstract Background Currently biomarkers play an essential role in diagnosis, treatment, and management of cancer. In non-small cell lung cancer (NSCLC) determination of biomarkers such as ALK, EGFR, ROS1 or PD-L1 is mandatory for an adequate treatment decision. The aim of this study is to determine the clinical and economic impact of current anaplastic lymphoma kinase testing scenario in Spain. Methods A joint model, composed by decision-tree and Markov models, was developed to estimate the long-term health outcomes and costs of NSCLC patients, by comparing the current testing scenario for ALK in Spain vs a hypothetical no-testing. The current distribution of testing strategies for ALK determination and their sensitivity and specificity data were obtained from the literature. Treatment allocation based on the molecular testing result were defined by a panel of Spanish experts. To assess long-term effects of each treatment, 3-states Markov models were developed, where progression-free survival and overall survival curves were extrapolated using exponential models. Medical direct costs (expressed in €, 2019) were included. A lifetime horizon was used and a discount rate of 3% was applied for both costs and health effects. Several sensitivity analyses, both deterministic and probabilistic, were performed in order test the robustness of the analysis. Results We estimated a target population of 7628 NSCLC patients, including those with non-squamous histology and those with squamous carcinomas who were never smokers. Over the lifetime horizon, the current ALK testing scenario produced additional 5060 and 3906 life-years and quality-adjusted life-years (QALY), respectively, compared with the no-testing scenario. Total direct costs were increased up to € 51,319,053 for testing scenario. The incremental cost-effectiveness ratio was 10,142 €/QALY. The sensitivity analyses carried out confirmed the robustness of the base-case results, being the treatment allocation and the test accuracy (sensitivity and specificity data) the key drivers of the model. Conclusions ALK testing in advanced NSCLC patients, non-squamous and never-smoker squamous, provides more than 3000 QALYs in Spain over a lifetime horizon. Comparing this gain in health outcomes with the incremental costs, the resulting incremental cost-effectiveness ratio reinforces that testing non-squamous and never-smoker squamous NSCLC is a cost-effective strategy in Spain.

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Economic Evaluation of Posaconazole Versus Standard Azole Therapy as Prophylaxis against Invasive Fungal Infections in Patients with Prolonged Neutropenia in Canada

Canadian Journal of Infectious Diseases and Medical Microbiology ◽

10.1155/2012/583630 ◽

2012 ◽

Vol 23 (2) ◽

pp. 59-64 ◽

Cited By ~ 8

Author(s):

Amir A Tahami Monfared ◽

Amy K O’Sullivan ◽

Coleman Rotstein ◽

George Papadopoulos

Keyword(s):

Cost Effectiveness ◽

High Risk ◽

Cost Saving ◽

Incremental Cost ◽

Incremental Cost Effectiveness Ratio ◽

Cost Effectiveness Ratio ◽

Lifetime Horizon ◽

System Perspective ◽

Life Years ◽

Neutropenic Patients

INTRODUCTION: Posaconazole prophylaxis in high-risk neutropenic patients prevents invasive fungal infection (IFI). An economic model was used to assess the cost effectiveness of posaconazole from a Canadian health care system perspective.METHODS: A decision-analytic model was developed based on data from a randomized trial comparing posaconazole with standard azole (fluconazole or itraconazole) therapy. The model was extrapolated to a lifetime horizon using one-month Markov cycles; lifetime survival was specific to the underlying disease. Drug and treatment costs associated with IFI were estimated using published literature. The model was used to estimate total costs, IFIs avoided, life-years gained and the incremental cost-effectiveness ratio of posaconazole versus standard azole therapy, in 2007 Canadian dollars.RESULTS: Based on the clinical trial data, posaconazole was associated with fewer cases of IFI (0.05 versus 0.11; P=0.003), increased life-years (2.52 years versus 2.43 years) and slightly lower costs ($6,601 versus $7,045) per patient relative to standard azole therapy over a lifetime horizon. Higher acquisition costs for posaconazole were offset by IFI-associated inpatient costs for those prophylaxed with standard azoles. Probabilistic sensitivity analysis indicated a 59% probability that posaconazole was cost-saving versus standard azole therapy and a 96% probability that the incremental cost-effectiveness ratio for posaconazole was at or below the $50,000 per life-year saved threshold.DISCUSSION: In Canada, posaconazole appears to be cost-saving relative to standard azole therapy in IFI prevention among high-risk neutropenic patients.

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Economic Evaluation of Andexanet Versus Prothrombin Complex Concentrate for Reversal of Factor Xa-Associated Intracranial Hemorrhage

Stroke ◽

10.1161/strokeaha.120.031108 ◽

2021 ◽

Author(s):

Andrew Micieli ◽

Andrew M. Demchuk ◽

Harindra C. Wijeysundera

Keyword(s):

Cost Effectiveness ◽

Life Expectancy ◽

Incremental Cost ◽

Intracranial Hemorrhage ◽

Prothrombin Complex Concentrate ◽

Factor Xa ◽

Incremental Cost Effectiveness Ratio ◽

Cost Effectiveness Ratio ◽

Lifetime Horizon ◽

Life Years

Background and Purpose: Andexanet was approved by the Food and Drug Administration in 2018 for reversal of life-threatening or uncontrolled bleeding associated with factor Xa anticoagulation; however, the cost-effectiveness of Andexanet compared with standard of care (ie, prothrombin complex concentrate, PCC) in patients with factor Xa–associated intracranial hemorrhage (ICrH) is unknown. Methods: Cost-effectiveness analysis using a Markov cohort decision analytic model with a lifetime horizon was completed to determine the costs and benefits of Andexanet compared with PCC for reversal of factor Xa–associated ICrH. The population of interest was patients living in Canada on chronic factor Xa inhibitors for prevention of ischemic stroke in nonvalvular atrial fibrillation or the prevention/treatment of venous thromboembolism, presenting with an ICrH. Outcomes of interest were life expectancy (measured in years), quality-adjusted life years (QALY), costs (reported in 2020 Canadian dollars), and the incremental cost-effectiveness ratio. Results: An overall reduction in fatal ICrH and increase in thromboembolic events was associated with Andexanet compared with PCC. Andexanet had the highest discounted life expectancy of 2.53 years and a discounted QALY of 1.55. PCC had a discounted life expectancy of 2.09 years and a discounted QALY of 1.28. The average discounted lifetime costs were $237 177 Canadian dollars for Andexanet and $177 871 Canadian dollars for PCC. The strategy of Andexanet had an incremental cost-effectiveness ratio was $219 652 per QALY gained compared with the comparator of PCC. The probabilistic sensitivity analyses demonstrated that Andexanet (at its current cost) was cost-effective in 19% of simulations using a willingness-to-pay threshold of $50 000/QALY and 33% of simulations at $150 000/QALY. A 1-way sensitivity analysis found that for the incremental cost-effectiveness ratio to be <$150 000/QALY gained, Andexanet high or standard dosing would require a price reduction to <$24 000 Canadian (at current baseline efficacy). Conclusions: Based on available evidence, Andexanet represents low value for reversal of factor Xa–associated ICrH; however, there is substantial uncertainty reflecting the currently available data. Further comparative evidence and costing data will become available in the future with randomized trials of Andexanet versus PCC.

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Cost-effectiveness of prevention strategies for American tegumentary leishmaniasis in Argentina

Cadernos de Saúde Pública ◽

10.1590/0102-311x00172512 ◽

2013 ◽

Vol 29 (12) ◽

pp. 2459-2472 ◽

Cited By ~ 8

Author(s):

Pablo Wenceslao Orellano ◽

Nestor Vazquez ◽

Oscar Daniel Salomon

Keyword(s):

Cost Effectiveness ◽

Early Diagnosis ◽

Incremental Cost ◽

Cost Effective ◽

Incremental Cost Effectiveness Ratio ◽

Sensitivity Analyses ◽

Cost Effectiveness Ratio ◽

Tegumentary Leishmaniasis ◽

Life Years ◽

The Cost

The aim of this study was to estimate the cost-effectiveness of reducing tegumentary leishmaniasis transmission using insecticide-impregnated clothing and curtains, and implementing training programs for early diagnosis. A societal perspective was adopted, with outcomes assessed in terms of costs per disability adjusted life years (DALY). Simulation was structured as a Markov model and costs were expressed in American dollars (US$). The incremental cost-effectiveness ratio of each strategy was calculated. One-way and multivariate sensitivity analyses were performed. The incremental cost-effectiveness ratio for early diagnosis strategy was estimated at US$ 156.46 per DALY averted, while that of prevention of transmission with insecticide-impregnated curtains and clothing was US$ 13,155.52 per DALY averted. Both strategies were more sensitive to the natural incidence of leishmaniasis, to the effectiveness of mucocutaneous leishmaniasis treatment and to the cost of each strategy. Prevention of vectorial transmission and early diagnosis have proved to be cost-effective measures.

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First- and Second-Line Bevacizumab in Addition to Chemotherapy for Metastatic Colorectal Cancer: A United States–Based Cost-Effectiveness Analysis

Journal of Clinical Oncology ◽

10.1200/jco.2014.58.4904 ◽

2015 ◽

Vol 33 (10) ◽

pp. 1112-1118 ◽

Cited By ~ 88

Author(s):

Daniel A. Goldstein ◽

Qiushi Chen ◽

Turgay Ayer ◽

David H. Howard ◽

Joseph Lipscomb ◽

...

Keyword(s):

Colorectal Cancer ◽

Cost Effectiveness ◽

Incremental Cost ◽

Metastatic Colorectal Cancer ◽

Incremental Cost Effectiveness Ratio ◽

Sensitivity Analyses ◽

Second Line ◽

Cost Effectiveness Ratio ◽

First Line ◽

Life Years

Purpose The addition of bevacizumab to fluorouracil-based chemotherapy is a standard of care for previously untreated metastatic colorectal cancer. Continuation of bevacizumab beyond progression is an accepted standard of care based on a 1.4-month increase in median overall survival observed in a randomized trial. No United States–based cost-effectiveness modeling analyses are currently available addressing the use of bevacizumab in metastatic colorectal cancer. Our objective was to determine the cost effectiveness of bevacizumab in the first-line setting and when continued beyond progression from the perspective of US payers. Methods We developed two Markov models to compare the cost and effectiveness of fluorouracil, leucovorin, and oxaliplatin with or without bevacizumab in the first-line treatment and subsequent fluorouracil, leucovorin, and irinotecan with or without bevacizumab in the second-line treatment of metastatic colorectal cancer. Model robustness was addressed by univariable and probabilistic sensitivity analyses. Health outcomes were measured in life-years and quality-adjusted life-years (QALYs). Results Using bevacizumab in first-line therapy provided an additional 0.10 QALYs (0.14 life-years) at a cost of $59,361. The incremental cost-effectiveness ratio was $571,240 per QALY. Continuing bevacizumab beyond progression provided an additional 0.11 QALYs (0.16 life-years) at a cost of $39,209. The incremental cost-effectiveness ratio was $364,083 per QALY. In univariable sensitivity analyses, the variables with the greatest influence on the incremental cost-effectiveness ratio were bevacizumab cost, overall survival, and utility. Conclusion Bevacizumab provides minimal incremental benefit at high incremental cost per QALY in both the first- and second-line settings of metastatic colorectal cancer treatment.

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Cost-effectiveness of sorafenib versus best supportive care in advanced renal cell carcinoma

Journal of Clinical Oncology ◽

10.1200/jco.2006.24.18_suppl.4604 ◽

2006 ◽

Vol 24 (18_suppl) ◽

pp. 4604-4604 ◽

Cited By ~ 7

Author(s):

X. Gao ◽

P. Reddy ◽

R. Dhanda ◽

K. Gondek ◽

Y. C. Yeh ◽

...

Keyword(s):

Renal Cell Carcinoma ◽

Cost Effectiveness ◽

Supportive Care ◽

Renal Cell ◽

Best Supportive Care ◽

Incremental Cost Effectiveness Ratio ◽

Sensitivity Analyses ◽

Cost Effectiveness Ratio ◽

Disease States ◽

Life Years

4604 Background: Results from the Phase III TARGETs study showed that sorafenib plus best supportive care (BSC) significantly prolonged progression-free survival (PFS) compared with BSC alone (p < 0.000001) in patients with advanced renal cell carcinoma (RCC). In addition, at a planned interim analysis, overall survival was numerically longer with sorafenib than BSC with a hazard ratio of 0.72. The objective of this study was to evaluate the cost-effectiveness of sorafenib + BSC versus BSC alone in advanced RCC from a US payer perspective. Methods: A Markov model was developed to project the lifetime survival and costs associated with sorafenib + BSC and BSC alone. The model tracked patients with advanced RCC through three disease states - PFS, progression, and death. Transition probabilities between disease states varied for each 3-month period and were obtained from the TARGETs study. Life-years gained were used as a measure of treatment effectiveness. Resource utilization included drug, administration, physician visits, monitoring, and adverse events. Costs and survival benefits were discounted annually at 3%. All costs were adjusted to 2004 US dollars. Scenario sensitivity analyses were conducted. Results: The lifetime per patient costs were $85,571 and $36,634 for sorafenib + BSC and BSC alone, respectively. The life-years gained were higher for sorafenib relative to BSC. The incremental cost-effectiveness ratio (ICER) of sorafenib + BSC versus BSC alone was $75,354 per life-year gained. The key drivers of the model results were survival after progression and PFS probabilities for both treatment groups. Sensitivity analyses showed that the model results were robust to variance in sorafenib and BSC treatment costs. Conclusions: The incremental cost-effectiveness ratio was within the established threshold that society is willing to pay (i.e., $50,000-$100,000). Therefore, sorafenib + BSC appears to be cost-effective in the management of advanced RCC. [Table: see text]

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Anticoagulation Therapy for Atrial Fibrillation in Patients With Alzheimer’s Disease

Stroke ◽

10.1161/strokeaha.118.022596 ◽

2018 ◽

Vol 49 (12) ◽

pp. 2844-2850

Author(s):

Estefanía Ruiz Vargas ◽

Luciano A. Sposato ◽

Spencer A. W. Lee ◽

Vladimir Hachinski ◽

Lauren E. Cipriano

Keyword(s):

Cost Effectiveness ◽

Incremental Cost ◽

Care Setting ◽

Anticoagulation Therapy ◽

Cost Effective ◽

Incremental Cost Effectiveness Ratio ◽

Cost Effectiveness Ratio ◽

Term Care ◽

Life Years

Background and Purpose— Direct oral anticoagulants (DOACs) are safer, at least equally efficacious, and cost-effective compared to warfarin for stroke prevention in atrial fibrillation (AF) but they remain underused, particularly in demented patients. We estimated the cost-effectiveness of DOACs compared with warfarin in patients with AF and Alzheimer’s disease (AD). Methods— We constructed a microsimulation model to estimate the lifetime costs, quality-adjusted life-years (QALYs), and cost-effectiveness of anticoagulation therapy (adjusted-dose warfarin and various DOACs) in 70-year-old patients with AF and AD from a US societal perspective. We stratified patient cohorts based on stage of AD and care setting. Model parameters were estimated from secondary sources. Health benefits were measured in the number of acute health events, life-years, and QALYs gained. We classified alternatives as cost-effective using a willingness-to-pay threshold of $100 000 per QALY gained. Results— For patients with AF and AD, compared with warfarin, DOACs increase costs but also increase QALYs by reducing the risk of stroke. For mild-AD patients living in the community, edoxaban increased lifetime costs by $6603 and increased QALYs by 0.076 compared to warfarin, yielding an incremental cost-effectiveness ratio of $86 882/QALY gained. Even though DOACs increased QALYs compared with warfarin for all patient groups (ranging from 0.019 to 0.085 additional QALYs), no DOAC treatment alternative had an incremental cost-effectiveness ratio <$150 000/QALY gained for patients with moderate to severe AD. For patients living in a long-term care facility with mild AD, the DOAC with the lowest incremental cost-effectiveness ratio (rivaroxaban) costs $150 169 per QALY gained; for patients with more severe AD, the incremental cost-effectiveness ratios were higher. Conclusions— For patients with AF and mild AD living in the community, edoxaban is cost-effective compared with warfarin. Even though patients with moderate and severe AD living in the community and patients with any stage of AD living in a long-term care setting may obtain positive clinical benefits from anticoagulation treatment, DOACs are not cost-effective compared with warfarin for these populations. Compared to aspirin, no oral anticoagulation (warfarin or any DOAC) is cost effective in patients with AF and AD.

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Cost-Effectiveness Analysis of Dabrafenib Plus Trametinib and Vemurafenib as First-Line Treatment in Patients with BRAF V600 Mutation-Positive Unresectable or Metastatic Melanoma in China

International Journal of Environmental Research and Public Health ◽

10.3390/ijerph18126194 ◽

2021 ◽

Vol 18 (12) ◽

pp. 6194

Author(s):

Tianfu Gao ◽

Jia Liu ◽

Jing Wu

Keyword(s):

Cost Effectiveness ◽

Incremental Cost ◽

Cost Effective ◽

Incremental Cost Effectiveness Ratio ◽

Sensitivity Analyses ◽

Cost Effectiveness Ratio ◽

First Line ◽

Health States ◽

Life Years ◽

Braf V600 Mutation

Objective: To evaluate the cost-effectiveness of dabrafenib plus trametinib combination therapy versus vemurafenib as first-line treatment in patients with BRAF V600 mutation-positive unresectable or metastatic melanoma from a healthcare system perspective in China. Methods: This study employed a partitioned survival model with three health states (progression-free survival, post-progression survival and dead) to parameterize the data derived from Combi-v trial and extrapolated to 30 years. Health states’ utilities were measured by EQ-5D-3L, also sourced from the Combi-v trial. Costs including drug acquisition costs, disease management costs and adverse event costs were based on the Chinese Drug Bidding Database and physician survey in China. The primary outcomes of the model were lifetime costs, life-years (LYs), quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratio (ICER). Deterministic and probabilistic sensitivity analyses were conducted, respectively. Result: Dabrafenib plus trametinib is projected to increase a patient’s life expectancy by 0.95 life-years over vemurafenib (3.03 vs. 2.08) and 1.09 QALY gains (2.48 vs. 1.39) with an incremental cost of $3833. The incremental cost-effectiveness ratio (ICER) was $3511 per QALY. In the probabilistic sensitivity analyses, at a threshold of $33,357 per QALY (three times the gross domestic product (GDP) per capita in China in 2020), the probability of dabrafenib plus trametinib being cost-effective was 90%. In the deterministic sensitivity analyses, the results were most sensitive to the dabrafenib plus trametinib drug costs, vemurafenib drug costs and discount rate of cost. Conclusion: Dabrafenib plus trametinib therapy yields more clinical benefits than vemurafenib. Using a threshold of $33,357 per QALY, dabrafenib plus trametinib is very cost-effective as compared with vemurafenib in China.

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Cost-utility Analysis of Second-generation Direct-acting Antivirals for Hepatitis C

Hepatitis Monthly ◽

10.5812/hepatmon118646 ◽

2021 ◽

Vol 21 (8) ◽

Author(s):

Abdollah Poursamad ◽

Zahra Goudarzi ◽

Iman Karimzadeh ◽

Nahid Jallaly ◽

Khosro Keshavarz ◽

...

Keyword(s):

Cost Effectiveness ◽

Hepatitis C ◽

Cost Effective ◽

Sensitivity Analyses ◽

Genotype 1 ◽

Medication Regimen ◽

Lifetime Horizon ◽

Medication Therapy ◽

Life Years ◽

Study Results

Background: Hepatitis C virus (HCV) can lead to increased mortality, disability, and liver transplantation if left untreated, and it is associated with a possible increase in disease burden in the future, all of which would surely have a significant impact on the health system. New antiviral regimens are effective in the treatment of the disease yet expensive. Objectives: The purpose of the present study was to assess the cost-effectiveness of three medication regimens, namely, ledipasvir/sofosbuvir (LDV/SOF), velpatasvir/sofosbuvir, and daclatasvir/sofosbuvir (DCV/SOF) for HCV patients with genotype 1 in Iran. Methods: A Markov model with a lifetime horizon was developed to predict the costs and outcomes of the three mentioned medication therapy strategies. The final outcome of the study was quality-adjusted life-years (QALYs), which was obtained using the previously published studies. The study was conducted from the perspective of the Health Ministry; therefore, only direct medical costs were estimated. The results were provided as the incremental cost-effectiveness ratio (ICER) per QALY. Ultimately, the one-way and probabilistic sensitivity analyses were used to measure the strength of study results. Results: The results showed that the QALYs for LDV/SOF, DCV/SOF, and VEL/SOF were 13.25, 13.94, and 14.61, and the costs were 4,807, 7,716, and 4,546$, respectively. The VEL/SOF regimen had lower costs and higher effectiveness than the LDV/SOF and DCV/SOF regimens, making it a dominant strategy. The tornado diagram results showed that the study results had the highest sensitivity to chronic hepatitis C (CHC) and compensated cirrhosis (CC) state costs. Moreover, the scatter plots showed that the VEL/SOF was the dominant therapeutic strategy in 73% of the simulations compared to LDV/SOF and 66% of the simulations compared to DCV/SOF; moreover, it was in the acceptable region in 92% of the simulations and below the threshold. Therefore, it was considered the most cost-effective strategy. Moreover, the results showed that DCV/SOF was in the acceptable region below the threshold in 69% of the simulations compared to LDV/SOF. Therefore, the DCV/SOF regimen was more cost-effective than LDV/SOF. Conclusions: According to the present study results, it is suggested that the VEL/SOF regimen be used as the first line of therapy in patients with HCV genotype 1. Moreover, DCV/SOF can be the second-line medication regimen.

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Pharmacokinetic Parameter Driven Outcomes Model Predicts a Reduction in Bleeding Events Associated with BAY 81-8973 Versus Antihemophilic Factor (Recombinant) Plasma/Albumin- Free Method in a Chinese Healthcare Setting

10.21203/rs.3.rs-1159955/v1 ◽

2022 ◽

Author(s):

Rong Chen ◽

Dmitry Gultyaev ◽

Johanna Lister ◽

Rong Han ◽

Nan Hu ◽

...

Keyword(s):

Hemophilia A ◽

Cost Savings ◽

Standard Of Care ◽

Healthcare Setting ◽

Bleeding Events ◽

Plasma Albumin ◽

Lifetime Horizon ◽

Life Years ◽

Antihemophilic Factor

Abstract Background: Long-term prophylactic therapy is considered the standard of care for hemophilia A patients. This study models the long-term clinical and cost outcomes of two factor VIII (FVIII) products using a pharmacokinetic (PK) simulation model in a Chinese population. Methods: Head-to-head PK profile data of BAY 81-8973 (KOVALTRY®) and antihemophilic factor (recombinant) plasma/albumin-free method (rAHF-PFM, ADVATE®) were applied to a two-state (alive and dead) Markov model to simulate blood FVIII concentrations at a steady state in prophylactically-treated patients with hemophilia A. Worsening of the Pettersson score was simulated and decline was associated with the probability of having orthopaedic surgery. The only difference between the compounds was FVIII concentration at a given time; each subject was treated with 25 IU/kg every 3 days. The model used a lifetime horizon, with cycle lengths of 1 year. Results: Cumulative bleeding events, joint bleeding events, and major bleeding events were reduced by 19.3%, 9.3% and 19.3%, respectively for BAY 81-8973 compared to rAHF-PFM. Hospitalizations and hospitalization days were also reduced by 19.3% for BAY 81-8973 compared to rAHF-PFM. BAY 81-8973 resulted in both cost savings and a gain in quality adjusted life years (QALYs) compared to rAHF-PFM. Conclusion: Based on modeled head-to-head comparisons, differences in PK-properties between BAY 81-8973 and rAHF-PFM result in a reduced number of bleeding events, leading to reduced costs and increased quality of life for BAY 81-8973. These results should be used to inform clinical practice in China when caring for patients with severe hemophilia A.

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Newborn screening for spinal muscular atrophy with disease-modifying therapies: a cost-effectiveness analysis

Journal of Neurology Neurosurgery & Psychiatry ◽

10.1136/jnnp-2021-326344 ◽

2021 ◽

pp. jnnp-2021-326344

Author(s):

Sophy TF Shih ◽

Michelle Anne Farrar ◽

Veronica Wiley ◽

Georgina Chambers

Keyword(s):

Gene Therapy ◽

Cost Effectiveness ◽

Spinal Muscular Atrophy ◽

Newborn Screening ◽

Markov Models ◽

Muscular Atrophy ◽

Early Gene ◽

Sensitivity Analyses ◽

Life Years ◽

Late Treatment

ObjectiveTo assess cost-effectiveness of newborn screening (NBS) for spinal muscular atrophy (SMA) and early treatment with nusinersen or onasemnogene abeparvovec (gene therapy), compared with nusinersen without SMA screening.MethodsInformed by an Australian state-wide SMA NBS programme, a decision analytical model nested with Markov models was constructed to evaluate costs and quality-adjusted life-years (QALYs) from a societal perspective with sensitivity analyses.ResultsBy treating one presymptomatic SMA infant with nusinersen or gene therapy, an additional 9.93 QALYs were gained over 60 years compared with late treatment in clinically diagnosed SMA. The societal cost was $9.8 million for early nusinersen treatment, $4.4 million for early gene therapy and $4.8 million for late nusinersen treatment. Compared with late nusinersen treatment, early gene therapy would be dominant, gaining 9.93 QALYs while saving $360 000; whereas early nusinersen treatment would result in a discounted incremental cost-effectiveness ratio (ICER) of $507 000/QALY.At a population level, compared with no screening and late treatment with nusinersen, NBS and early gene therapy resulted in 0.00085 QALY gained over 60 years and saving $24 per infant screened (85 QALYs gained and $2.4 million saving per 100 000 infants screened). More than three quarters of simulated ICERs by probability sensitivity analyses showed NBS and gene therapy would be dominant or less than $50 000/QALY, compared with no screening and late nusinersen treatment.ConclusionNBS coupled with gene therapy improves the quality and length of life for infants with SMA and would be considered value-for-money from an Australian clinical and policy context.

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