scholarly journals Conflicting findings between the Mitra-Fr and the Coapt trials: Implications regarding the cost-effectiveness of percutaneous repair for heart failure patients with severe secondary mitral regurgitation

PLoS ONE ◽  
2020 ◽  
Vol 15 (11) ◽  
pp. e0241361
Author(s):  
Xavier Armoiry ◽  
Jean-François Obadia ◽  
Peter Auguste ◽  
Martin Connock
Keyword(s):  
Heart Failure ◽  
Cost Effectiveness ◽  
Mitral Regurgitation ◽  
Cost Effective ◽  
Base Case ◽  
Published Data ◽  
Parametric Modelling ◽  
Percutaneous Repair ◽  
The Cost ◽  
The Impact

Purpose Two randomized controlled trials (RCTs), Mitra-Fr and Coapt, evaluating the benefit of percutaneous repair (PR) for heart failure (HF) patients with severe mitral regurgitation, have led to conflicting results. We aimed to evaluate the impact of these trial results on the cost-effectiveness of PR using effectiveness inputs from the two RCTs. Methods We developed a time varying Markov type model with three mutually exclusive health states: alive without HF hospitalisation, alive with HF hospitalisation, and dead. Clinically plausible extrapolations beyond observed data were obtained by developing parametric modelling for overall survival and HF hospitalisations using published data from each trial. We adopted the perspective of the French Health System and used a 30-year time horizon. Results were expressed as € / quality-adjusted life year (QALY) gained using utility inputs from literature. Findings Results are presented using treatment efficacy measures from Mitra-F and Coapt trials respectively. With the Mitra-Fr data, after annual discounting, the base case model generated an incremental 0.00387 QALY at a cost of €25,010, yielding an incremental cost effectiveness ratio (ICER) of €6,467,032 / QALY. The model was sensitive to changes made to model inputs. There was no potential of PR being cost-effective. With the Coapt data, the model generated 1.19 QALY gain at a cost of €26,130 yielding an ICER of €21,918 / QALY and at a threshold of >€50,000/QALY PR had a probability of 1 of being cost-effective. Implications Cost effectiveness results were conflicting; reconciling differences between trials is a priority and could promote optimal cost effectiveness analyses and resource allocation.

Cost-effectiveness of a home telemonitoring system and a diagnostic algorithm in the management of heart failure in the Netherlands (Preprint)

2021 ◽  
Author(s):  
Fernando Albuquerque de Almeida ◽  
Isaac Corro Ramos ◽  
Maiwenn Al ◽  
Maureen Rutten-van Mölken
Keyword(s):  
Heart Failure ◽  
Cost Effectiveness ◽  
The Netherlands ◽  
Nyha Class ◽  
Cost Effective ◽  
Health Concern ◽  
Base Case ◽  
Home Telemonitoring ◽  
Wide Range ◽  
The Cost

BACKGROUND Heart failure (HF) is a major health concern associated with significant morbidity, mortality, and reduced quality of life for patients. Home telemonitoring (HTM) facilitates frequent or continuous assessment of disease signs and symptoms, while it has been shown to improve compliance by involving patients in their own care and to prevent emergency admissions by facilitating early detection of clinically significant changes. Diagnostic algorithms (DAs) are predictive mathematical relationships that make use of a wide range of collected data for calculating the likelihood of a particular event happening and utilise this output for prioritising patients with regards to their treatment. OBJECTIVE Assessing the cost-effectiveness of HTM and a DA in the management of heart failure in the Netherlands. Three interventions were analysed: usual care (UC), HTM, and HTM+DA. METHODS A previously published discrete event simulation model was used. The base-case analysis was performed according to the Dutch guidelines for economic evaluation. Sensitivity, scenario, and value of information analyses were performed. Particular attention was given to the cost-effectiveness of the DA at various levels of diagnostic accuracy of event prediction and to different patient subgroups. RESULTS HTM+DA extendedly dominates HTM and it has a deterministic incremental cost-effectiveness ratio versus UC of €27,712 per quality-adjusted life year (QALY). The model showed robustness in the sensitivity and scenario analyses. HTM+DA had a 96.0% probability of being cost-effective at a €80,000/QALY threshold. An optimal point for the threshold value for the alarm of the DA in terms of its cost-effectiveness was estimated. NYHA class IV patients were the subgroup with the worst cost-effectiveness results versus UC, while HTM+DA was found to be the most cost-effective for patients <65 years-old and for patients in NYHA class I. CONCLUSIONS Although increased costs of adopting HTM and DA in the management of HF may seemingly be an additional strain on scarce health care resources, the results of this study demonstrate that, by increasing patient life expectancy by 1.28 years and reducing their hospitalisation rate by 23% when compared to UC, the use of these technologies may be seen as an investment, as HTM+DA extendedly dominates HTM and is cost-effective versus UC at normally accepted thresholds in the Netherlands.

Therapeutic options in metastatic castration-resistant prostate cancer (mCRPC): A cost-effectiveness analysis.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. e15154-e15154 ◽  
Author(s):  
Lixian Zhong ◽  
Sandy Srinivas ◽  
Vickie Pon ◽  
Nicole Nguyen ◽  
Meghan Frear ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Cost Effectiveness ◽  
Side Effect ◽  
Wholesale Price ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Published Data ◽  
Baseline Severity ◽  
The Cost

e15154 Background: Initial therapy for metastatic prostate cancer is androgen deprivation therapy. Uniformly progression of disease occurs despite castration. Docetaxel is then the standard of care. Abiraterone (Abi) and cabazitaxel (Cab) are approved for mCRPC post docetaxel with improvements in survival We seek to evaluate the cost-effectiveness of Abi and Cab compared to mitoxantrone (Mit) and prednisone for mCRPC. Methods: A decision tree model was constructed comparing four treatments for mCRPC patients over an 18 month period: 1) Abi, 2) Cab, 3) Mit, and 4) prednisone only. Chance nodes included baseline pain as a severity indicator, grade III & IV adverse events (AE) (neutropenia and cardiac ) and survival at 18mos. Survival rates and probabilities were from published data. Cost of drug regimens were based on 2010 US dollars and average wholesale price (AWP). Model cost inputs included drug-costs, administration costs, management of AE’s, radiation for pain, and costs associated with death. Health utilities for baseline pain, bone pain, neutropenia, cardiac events, and radiation were obtained from published data. Sensitivity analyses were conducted focused on baseline severity and costs of drug. Results: Base case estimates suggest that Cab and Abi are most effective. The incremental cost effectiveness ratio (ICER) for Mit vs. placebo is $110K/QALY, Abi vs. Mit is $76K/QALY and for Cab vs. Abi, the ICER is $925K/QALY. Model-sensitive parameters include Abi AWP and cost of Mit side effect management; Abi becomes less cost-effective as its AWP increases, or if the cost of Mit side effect management decreases. When baseline severity increases, Mit becomes cost-effective over placebo and Abi remains cost-effective over Mit. Cab remains not cost-effective at tolerable thresholds. Conclusions: Compared to Cab, Mit, and placebo, Abi is the most cost-effective option in docetaxel-refractory mCRPC patients due to its ability to prolong survival and maintain a willingness-to-pay threshold (WTP) of $100K. Despite higher survival with Cab, it is not cost-effective because of costs associated with treatment of neutropenia. In situations where illness is more severe, Abi remains the cost-effective choice.

Financial implications of dialysis modalities in the developing world: A Chinese perspective

2020 ◽  
Vol 40 (2) ◽  
pp. 193-201
Author(s):  
Jing Liu ◽  
David W Hutton ◽  
Yonghong Gu ◽  
Yao Hu ◽  
Yi Li ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Direct Cost ◽  
Cost Effective ◽  
The United States ◽  
Direct Costs ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Published Data ◽  
Life Years ◽  
The Cost

Background: End-stage renal disease has been imposing a heavy economic burden on public health; however, few studies have been performed on the cost-effectiveness of dialysis modalities. We aim to estimate the cost-effectiveness of different dialysis modalities in China. Methods: Cost-effectiveness analyses were performed using Markov models based on published data of hemodialysis (HD) and peritoneal dialysis (PD) modalities in China. Sensitivity analyses were conducted to identify key variables influencing the results. Results: Over a 10-year time horizon, the base-case cost-effectiveness analysis indicated that PD-first absolutely dominated the HD-first option by gaining 0.13 more quality-adjusted life years (QALYs) and costing RMB ¥81,081 less. When using reported mortality of HD and PD from the United States, the PD-first option still dominated HD-first with higher QALYs and lower costs. Sensitivity analyses revealed that the results were more sensitive to the direct cost of HD, utility of HD, utility of PD, direct cost of PD, PD mortality, and HD mortality, while less sensitive to the indirect costs and transition probabilities. The HD utility needed to be at least 0.148 higher than PD utility for HD to be cost-effective. PD was about 72% likely to be considered cost-effective compared with HD, regardless of the willingness-to-pay for QALYs. Conclusion: PD appears to be more cost-effective than HD in China, and the major influential factors on the cost-effectiveness are the direct costs of HD, utility of HD, utility of PD, direct costs of PD, PD mortality, and HD mortality.

Abstract 15981: Cost-effectiveness of Dapagliflozin in Heart Failure With Reduced Ejection Fraction

Circulation ◽  
2020 ◽  
Vol 142 (Suppl_3) ◽  
Author(s):  
Nicolas Isaza ◽  
Paola Calvachi ◽  
Inbar Raber ◽  
Changyu Shen ◽  
Michael C Gavin ◽  
...  
Keyword(s):  
Heart Failure ◽  
Cost Effectiveness ◽  
Ejection Fraction ◽  
Incremental Cost ◽  
Cost Effective ◽  
Healthcare Sector ◽  
Base Case ◽  
Reduced Ejection Fraction ◽  
Life Years ◽  
The Cost

Introduction: In May 2020, the US FDA approved the use of dapagliflozin, an SGLT2 inhibitor, for the reduction of cardiovascular death and heart failure hospitalization in patients with heart failure with reduced ejection fraction (HFrEF). We examined the cost-effectiveness of adding dapagliflozin to guideline-directed medical therapy (GDMT) in patients with or without diabetes. Methods: We developed a state-transition Markov model with inputs from the DAPA-HF trial, FDA review documents, published literature, and nationally representative datasets (Panel A). The model was calibrated to event rates observed in DAPA-HF; survival was extrapolated using non-parametric approaches. The main outcomes were quality-adjusted life years (QALYs) and incremental cost effectiveness ratio (ICER) of dapagliflozin + GDMT compared with GDMT alone, from a healthcare sector perspective and a lifetime analytic horizon. We applied a discount rate of 3% per year for future costs and benefits, and assumed a willingness-to-pay threshold of $100,000 per QALY gained. In sensitivity analyses, we examined subgroups with or without diabetes, and varied the cost of dapagliflozin (base case = $6,188 per year). This analysis was independent of the trial sponsor. Results: Compared with GDMT alone, adding dapagliflozin produced 0.57 additional QALYs at an incremental cost of $56,650, producing an ICER of $98,700 per QALY gained (Panel B). In subgroup analyses, dapagliflozin produced 0.71 additional QALYs in patients with diabetes at an ICER of $89,100 per QALY gained, and 0.48 additional QALYs in patients without diabetes at an ICER of $108,800 per QALY (Panels C and D). A 9% price reduction (to $5,613 per year) would make dapagliflozin cost-effective in patients without diabetes. Conclusions: Adding dapagliflozin to GDMT in patients with HFrEF is cost-effective and has the potential to improve long-term outcomes. Scalable strategies to improve access and uptake are urgently required.

scholarly journals Cost-effectiveness and feasibility of conditional economic incentives and motivational interviewing to improve HIV health outcomes of adolescents living with HIV in Anambra State, Nigeria

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Obinna Ikechukwu Ekwunife ◽  
Chinelo Janefrances Ofomata ◽  
Charles Ebuka Okafor ◽  
Maureen Ugonwa Anetoh ◽  
Stephen Okorafor Kalu ◽  
...  
Keyword(s):  
Viral Load ◽  
Cost Effectiveness ◽  
Service Delivery ◽  
Motivational Interviewing ◽  
Cost Effective ◽  
Economic Incentives ◽  
Additional Patient ◽  
Incentive Scheme ◽  
The Cost ◽  
The Impact

Abstract Background In sub-Saharan Africa, there is increasing mortality and morbidity of adolescents due to poor linkage, retention in HIV care and adherence to antiretroviral therapy (ART). This is a result of limited adolescent-centred service delivery interventions. This cost-effectiveness and feasibility study were piggybacked on a cluster-randomized trial that assessed the impact of an adolescent-centred service delivery intervention. The service delivery intervention examined the impact of an incentive scheme consisting of conditional economic incentives and motivational interviewing on the health outcomes of adolescents living with HIV in Nigeria. Method A cost-effectiveness analysis from the healthcare provider’s perspective was performed to assess the cost per additional patient achieving undetected viral load through the proposed intervention. The cost-effectiveness of the incentive scheme over routine care was estimated using the incremental cost-effectiveness ratio (ICER), expressed as cost/patient who achieved an undetectable viral load. We performed a univariate sensitivity analysis to examine the effect of key parameters on the ICER. An in-depth interview was conducted on the healthcare personnel in the intervention arm to explore the feasibility of implementing the service delivery intervention in HIV treatment hospitals in Nigeria. Result The ICER of the Incentive Scheme intervention compared to routine care was US$1419 per additional patient with undetectable viral load. Going by the cost-effectiveness threshold of US$1137 per quality-adjusted life-years suggested by Woods et al., 2016, the intervention was not cost-effective. The sensitivity test showed that the intervention will be cost-effective if the frequency of CD4 count and viral load tests are reduced from quarterly to triannually. Healthcare professionals reported that patients’ acceptance of the intervention was very high. Conclusion The conditional economic incentives and motivational interviewing was not cost-effective, but can become cost-effective if the frequency of HIV quality of life indicator tests are performed 1–3 times per annum. Patients’ acceptance of the intervention was very high. However, healthcare professionals believed that sustaining the intervention may be difficult unless factors such as government commitment and healthcare provider diligence are duly addressed. Trial registration This trial is registered in the WHO International Clinical Trials Registry through the WHO International Registry Network (PACTR201806003040425).

scholarly journals Addition of Flucytosine to Fluconazole for the Treatment of Cryptococcal Meningitis in Africa: A Multicountry Cost-effectiveness Analysis

2019 ◽  
Vol 70 (1) ◽  
pp. 26-29 ◽  
Author(s):  
Tinevimbo Shiri ◽  
Angela Loyse ◽  
Lawrence Mwenge ◽  
Tao Chen ◽  
Shabir Lakhi ◽  
...  
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Cryptococcal Meningitis ◽  
Cost Effective ◽  
Immunodeficiency Virus ◽  
The Mean ◽  
The Cost ◽  
The Impact ◽  
Costing Analysis ◽  
Very High

Abstract Background Mortality from cryptococcal meningitis remains very high in Africa. In the Advancing Cryptococcal Meningitis Treatment for Africa (ACTA) trial, 2 weeks of fluconazole (FLU) plus flucytosine (5FC) was as effective and less costly than 2 weeks of amphotericin-based regimens. However, many African settings treat with FLU monotherapy, and the cost-effectiveness of adding 5FC to FLU is uncertain. Methods The effectiveness and costs of FLU+5FC were taken from ACTA, which included a costing analysis at the Zambian site. The effectiveness of FLU was derived from cohorts of consecutively enrolled patients, managed in respects other than drug therapy, as were participants in ACTA. FLU costs were derived from costs of FLU+5FC in ACTA, by subtracting 5FC drug and monitoring costs. The cost-effectiveness of FLU+5FC vs FLU alone was measured as the incremental cost-effectiveness ratio (ICER). A probabilistic sensitivity analysis assessed uncertainties and a bivariate deterministic sensitivity analysis examined the impact of varying mortality and 5FC drug costs on the ICER. Results The mean costs per patient were US $847 (95% confidence interval [CI] $776–927) for FLU+5FC, and US $628 (95% CI $557–709) for FLU. The 10-week mortality rate was 35.1% (95% CI 28.9–41.7%) with FLU+5FC and 53.8% (95% CI 43.1–64.1%) with FLU. At the current 5FC price of US $1.30 per 500 mg tablet, the ICER of 5FC+FLU versus FLU alone was US $65 (95% CI $28–208) per life-year saved. Reducing the 5FC cost to between US $0.80 and US $0.40 per 500 mg resulted in an ICER between US $44 and US $28 per life-year saved. Conclusions The addition of 5FC to FLU is cost-effective for cryptococcal meningitis treatment in Africa and, if made available widely, could substantially reduce mortality rates among human immunodeficiency virus–infected persons in Africa.

scholarly journals Cytomegalovirus Screening in Pregnancy: A Cost-Effectiveness and Threshold Analysis

2018 ◽  
Vol 36 (07) ◽  
pp. 678-687 ◽  
Author(s):  
Catherine M. Albright ◽  
Erika F. Werner ◽  
Brenna L. Hughes
Keyword(s):  
Cost Effectiveness ◽  
Behavioral Intervention ◽  
Universal Screening ◽  
Cost Effective ◽  
Base Case ◽  
Design Decision ◽  
Intervention Effectiveness ◽  
Behavioral Counseling ◽  
The Cost ◽  
In Pregnancy

Objective To determine threshold cytomegalovirus (CMV) infectious rates and treatment effectiveness to make universal prenatal CMV screening cost-effective. Study Design Decision analysis comparing cost-effectiveness of two strategies for the prevention and treatment of congenital CMV: universal prenatal serum screening and routine, risk-based screening. The base case assumptions were a probability of primary CMV of 1% in seronegative women, hyperimmune globulin (HIG) effectiveness of 0%, and behavioral intervention effectiveness of 85%. Screen-positive women received monthly HIG and screen-negative women received behavioral counseling to decrease CMV seroconversion. The primary outcome was the cost per maternal quality-adjusted life year (QALY) gained with a willingness to pay of $100,000 per QALY. Results In the base case, universal screening is cost-effective, costing $84,773 per maternal QALY gained. In sensitivity analyses, universal screening is cost-effective only at a primary CMV incidence of more than 0.89% and behavioral intervention effectiveness of more than 75%. If HIG is 30% effective, primary CMV incidence can be 0.82% for universal screening to be cost-effective. Conclusion The cost-effectiveness of universal maternal screening for CMV is highly dependent on the incidence of primary CMV in pregnancy. If efficacious, HIG and behavioral counseling allow universal screening to be cost-effective at lower primary CMV rates.

The Cost-Effectiveness of an RCT to Establish Whether 5 or 10 Years of Bisphosphonate Treatment Is the Better Duration for Women With a Prior Fracture

2009 ◽  
Vol 29 (6) ◽  
pp. 678-689 ◽  
Author(s):  
Matt D. Stevenson ◽  
Jeremy E. Oakley ◽  
Myfawny Lloyd Jones ◽  
Alan Brennan ◽  
Juliet E. Compston ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Cost Effective ◽  
Fracture Prevention ◽  
Published Data ◽  
Bisphosphonate Treatment ◽  
Net Benefit ◽  
England And Wales ◽  
Term Efficacy ◽  
The Cost

Purpose. Five years of bisphosphonate treatment have proven efficacy in reducing fractures. Concerns exist that long-term bisphosphonate treatment may actually result in an increased number of fractures. This study evaluates, in the context of England and Wales, whether it is cost-effective to conduct a randomized controlled trial (RCT) and what sample size may be optimal to estimate the efficacy of bisphosphonates in fracture prevention beyond 5 years. Method. An osteoporosis model was constructed to evaluate the cost-effectiveness of extending bisphosphonate treatment from 5 years to 10 years. Two scenarios were run. The 1st uses long-term efficacy data from published literature, and the 2nd uses distributions elicited from clinical experts. Results of a proposed RCT were simulated. The expected value of sample information technique was applied to calculate the expected net benefit of sampling from conducting such an RCT at varying levels of participants per arm and to compare this with proposed trial costs. Results. Without further information, the better duration of bisphosphonate treatment was estimated to be 5 years using the published data but 10 years using the elicited expert opinions, although in both cases uncertainty was substantial. The net benefit of sampling was consistently high when between 2000 and 5000 participants per arm were recruited. Conclusions. An RCT to evaluate the long-term efficacy of bisphosphonates in fracture prevention appears to be cost-effective for informing decision making in England and Wales.

Abstract 200: Cost-Effectiveness of Newer Anticoagulants for Stroke Prevention in Atrial Fibrillation

2013 ◽  
Vol 6 (suppl_1) ◽  
Author(s):  
Brendan L Limone ◽  
William L Baker ◽  
Craig I Coleman
Keyword(s):  
Atrial Fibrillation ◽  
Cost Effectiveness ◽  
Stroke Prevention ◽  
Indirect Comparison ◽  
Cost Effective ◽  
The United States ◽  
Base Case ◽  
Treatment Comparison ◽  
Newer Anticoagulants ◽  
The Cost

Background: A number of new anticoagulants for stroke prevention in atrial fibrillation (SPAF) have gained regulatory approval or are in late-stage development. We sought to conduct a systematic review of economic models of dabigatran, rivaroxaban and apixaban for SPAF. Methods: We searched the Medline, Embase, National Health Service Economic Evaluation Database and Health Technology Assessment database along with the Tuft’s Registry through October 10, 2012. Included models assessed the cost-effectiveness of dabigatran (150mg, 110mg, sequential), rivaroxaban or apixaban for SPAF using a Markov model or discrete event simulation and were published in English. Results: Eighteen models were identified. All models utilized a lone randomized trial (or an indirect comparison utilizing a single study for any given direct comparison), and these trials were clinically and methodologically heterogeneous. Dabigatran 150mg was assessed in 9 of models, dabigatran 110mg in 8, sequential dabigatran in 9, rivaroxaban in 4 and apixaban in 4. Adjusted-dose warfarin (either trial-like, real-world prescribing or genotype-dosed) was a potential first-line therapy in 94% of models. Models were conducted from the perspective of the United States (44%), European countries (39%) and Canada (17%). In base-case analyses, patients typically were at moderate-risk of ischemic stroke, initiated anticoagulation between 65 and 73 years of age, and were followed for or near a lifetime. All models reported cost/quality-adjusted life-year (QALY) gained, and while 22% of models reported using a societal perspective, no model included costs of lost productivity. Four models reported an incremental cost-effectiveness ratio (ICER) for a newer anticoagulant (dabigatran 110mg (n=4)/150mg (n=2); rivaroxaban (n=1)) vs. warfarin above commonly reported willingness-to-pay thresholds. ICERs (in 2012US$) vs. warfarin ranged from $3,547-$86,000 for dabigatran 150mg, $20,713-$150,000 for dabigatran 110mg, $4,084-$21,466 for sequentially-dosed dabigatran and $23,065-$57,470 for rivaroxaban. In addition, apixaban was demonstrated to be an economically dominant strategy compared to aspirin and to be dominant or cost-effective ($11,400-$25,059) vs. warfarin. Based on separate indirect treatment comparison meta-analyses, 3 models compared the cost-effectiveness of these new agents and reported conflicting results. Conclusions: Cost-effectiveness models of newer anticoagulants for SPAF have been extensively published. Models have frequently found newer anticoagulants to be cost-effective, but due to the lack of head-to-head trial comparisons and heterogeneity in clinical characteristic of underlying trials and modeling methods, it is currently unclear which of these newer agents is most cost-effective.

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

2019 ◽  
Vol 5 (3) ◽  
pp. 28 ◽  
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.

Sign in / Sign up

Export Citation Format

Share Document