Targeted Therapies Compared to Dacarbazine for Treatment of BRAFV600EMetastatic Melanoma: A Cost-Effectiveness Analysis

Purpose. Two BRAFV600Etargeted therapies, dabrafenib and vemurafenib, have received US approval for treatment of metastatic melanoma in BRAFV600Epatients, a mutation that affects ~50% of patients. We evaluated the cost-effectiveness of BRAF inhibitors and traditional chemotherapy for treatment of metastatic melanoma.Methods. A Markov model was developed using a societal perspective. Transition probabilities were derived from two Phase III registration trials comparing each BRAF inhibitor against dacarbazine. Costs were obtained from literature, national databases, and Medicare fee schedules. Utilities were obtained from published literature. Deterministic and probabilistic sensitivity analyses were run to test the impact of uncertainties.Results. The incremental cost-effectiveness ratio of dabrafenib was $149,035/QALY compared to dacarbazine. Vemurafenib was dominated by dabrafenib. Probabilistic sensitivity analysis showed that, at a willingness-to-pay (WTP) threshold of ≤$100,000/QALY, dacarbazine was the optimal treatment in ~85% of simulations. At a WTP threshold of ≥$150,000/QALY, dabrafenib was the optimal treatment.Conclusion. Compared with dacarbazine, dabrafenib and vemurafenib were not cost-effective at a willingness-to-pay threshold of $100,000/QALY. Dabrafenib is more efficient compared to vemurafenib. With few treatment options, dabrafenib is an option for qualifying patients if the overall cost of dabrafenib is reduced to $30,000–$31,000 or a WTP threshold of ≥$150,000/QALY is considered. More comparative data is needed.

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Cost-effectiveness of rivaroxaban compared with enoxaparin plus warfarin for the treatment of hospitalised acute deep vein thrombosis in China

BMJ Open ◽

10.1136/bmjopen-2020-038433 ◽

2020 ◽

Vol 10 (7) ◽

pp. e038433

Author(s):

Li Yang ◽

Jingjing Wu

Keyword(s):

Deep Vein Thrombosis ◽

Cost Effectiveness ◽

Cost Saving ◽

Transition Probabilities ◽

Sensitivity Analyses ◽

Vein Thrombosis ◽

Life Years ◽

Acute Deep Vein Thrombosis ◽

Deep Vein ◽

The Impact

ObjectiveLimited economic evaluation data for rivaroxaban compared with standard of care (SoC) exists in China. The objective of this analysis was to evaluate the cost-effectiveness of rivaroxaban compared with current SoC (enoxaparin overlapped with warfarin) for the treatment of acute deep vein thrombosis (DVT) in China.MethodsA Markov model was adapted from a payer’s perspective to evaluate the costs and quality-adjusted life years (QALYs) of patients with DVT treated with rivaroxaban or enoxaparin/warfarin. Clinical data from the EINSTEIN-DVT trial were obtained to estimate the transition probabilities. Data on Chinese health resource use, unit costs and utility parameters were collected from previously published literature and used to estimate the total costs and QALYs. The time horizon was set at 5 years and a 3-month cycle length was used in the model. A 5% discount rate was applied to the projected costs. One-way sensitivity analyses and probabilistic sensitivity analyses were undertaken to assess the impact of uncertainty on results.ResultsRivaroxaban therapy resulted in an increase of 0.008 QALYs and was associated with lower total costs compared with enoxaparin/warfarin (US$4744.4 vs US$5572.4, respectively), demonstrating it to be a cost-saving treatment strategy. The results were mainly sensitive to length of hospitalisation due to DVT on enoxaparin/warfarin, cost per day of hospitalisation and the difference in length of stay of rivaroxaban-treated and enoxaparin/warfarin-treated patients.ConclusionRivaroxaban therapy resulted in a cost saving compared with enoxaparin/warfarin for the anticoagulation treatment of patients with hospitalised acute DVT in China.Trial registration numberNCT00440193; Post-results.

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Cost-effectiveness Analysis of Denosumab in the Prevention of Skeletal-related Events in Patients with Prostate Cancer in Kazakhstan

Central Asian Journal of Global Health ◽

10.5195/cajgh.2014.154 ◽

2014 ◽

Vol 3 ◽

Author(s):

Carina Bektur ◽

Talgat Nurgozhin

Keyword(s):

Prostate Cancer ◽

Cost Effectiveness ◽

Transition Probabilities ◽

Direct Costs ◽

Phase Iii ◽

Sensitivity Analyses ◽

Tree Age ◽

Bone Mass Loss ◽

Skeletal Related Events ◽

The Cost

Introduction. Bone mass loss (BML) is one of the adverse effects of oncological chemotherapy, especially in cases of hormonal types of cancer, such as a prostate cancer (PC). BML is strongly associated with skeletal-related events (SREs), therefore decreasing the quality of patient’s life. Denosumab shows an advantage over zoledronic acid (ZA) in delaying the first onset of SREs and subsequent SREs in adults with PC in several phase III clinical trials. Since generic ZA recently became available, the purpose of the present study was to assess the cost-effectiveness of denosumab vs. brand or generic ZA in the prevention of SREs in Kazakhstani patients with PC.Methods. A Markov model was constructed in Tree-Age Pro 2013 software program with 4-week model cycles to analyze the cost-effectiveness of the treatments from the perspective of Ministry of Health (MoH) over a 10-year PC cohort. Direct costs (in Kazakhstani monetary units “tenge” in 2014) included costs of drug, SRE (pathologic fracture, surgery to bone, radiation to bone, spinal cord compression), and adverse events treatment. All costs were discounted for 3% per year. Effectiveness was appraised based on the number of SREs. Health states were defined according to SRE occurrence, SRE history, and death. The model assumed that a maximum of 1 SRE could occur in each cycle. Transition probabilities were derived from the relevant phase III trials. Results were present in the incremental total cost per SRE avoided. One-way sensitivity analyses were performed to examine the robustness of the model.Results. Over the 10-year period, denosumab incurred 103,091 tenge higher costs than brand ZA, 677,133 tenge higher costs than generic ZA, and 0.58 fewer SREs per patient with PC. The estimated incremental total direct costs per SRE avoided with the use of denosumab were 177,743 tenge (instead of brand ZA) and 1,167,470 tenge (instead of generic ZA). Results were robust to one-way sensitivity analyses.Conclusions.With the assumption that brand and generic ZAs are equally effective in the prevention of SREs in PC patients, denosumab seems to be a cost-effective alternative for brand ZA (insignificant difference in costs – less than 5%) and a costly alternative for generic ZA from the perspective of MoH of Kazakhstan.

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Cost-effectiveness analysis of transarterial chemoembolization combined with stereotactic body radiation therapy versus transarterial chemoembolization for inoperable hepatocellular carcinoma: A markov modelling study

10.21203/rs.3.rs-16644/v1 ◽

2020 ◽

Author(s):

haiyin wang ◽

Vijay Gc ◽

Sheng Zhang ◽

chunlin Jin

Keyword(s):

Hepatocellular Carcinoma ◽

Radiation Therapy ◽

Cost Effectiveness ◽

Willingness To Pay ◽

Transarterial Chemoembolization ◽

Stereotactic Body Radiation Therapy ◽

Transition Probabilities ◽

Sensitivity Analyses ◽

Stereotactic Body Radiation ◽

Life Years

Abstract Background Transarterial chemoembolization (TACE) and Stereotactic body radiation therapy (SBRT) might both provide survival benefits for inoperable hepatocellular carcinoma (HCC). Adopting combined therapy as a solution carries major cost and resource implications. We aimed to estimate the cost-effectiveness of TACE plus SBRT and TACE alone in inoperable HCC. Methods A Markov model was constructed in a hypothetical cohort of patients aged 60 years with inoperable HCC and Child-Pugh A/B cirrhosis over a lifetime frame. Two strategies (TACE plus SBRT and TACE) were compared. Transition probabilities, utility and costs were extracted from published literature. Incremental cost-effectiveness ratios (ICER) were measured. Deterministic and probabilistic sensitivity analyses were conducted to assess the robustness of the findings. Results TACE plus SBRT and TACE respectively produced 12.26 and 9.67 quality-adjusted life years (QALYs). The ICER of TACE plus SBRT versus TACE was $3,133/QALY. One-way sensitivity analysis revealed that the utility of TACE combined with SBRT progress survival, probability of death from progress survival in TACE, and the initial cost of TACE combined with SBRT were the most sensitive parameters. The Monte-Carlo simulation demonstrated that the probability of cost-effectiveness at a willingness to pay threshold of US$ 29,440 per QALY was 95% and 5% for TACE plus SBRT and TACE. Conclusions This study indicated that TACE plus SBRT is cost-effective compared to TACE for inoperable HCC patients at a willingness to pay threshold as defined by WHO guidelines in China.

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Cost-effectiveness of paclitaxel, doxorubicin, cyclophosphamide and trastuzumab versus docetaxel, cisplatin and trastuzumab in new adjuvant therapy of breast cancer in china

Cost Effectiveness and Resource Allocation ◽

10.1186/s12962-021-00264-w ◽

2021 ◽

Vol 19 (1) ◽

Author(s):

Qiaoping Xu ◽

Li Yuanyuan ◽

Zhu Jiejing ◽

Liu Jian ◽

Li Qingyu ◽

...

Keyword(s):

Breast Cancer ◽

Cost Effectiveness ◽

Partial Response ◽

Cancer Patients ◽

Transition Probabilities ◽

Complete Response ◽

Sensitivity Analyses ◽

Half Cycle ◽

Breast Cancer Patients ◽

Grade 3

Abstract Background Breast cancer is the most common cancer among women in China. Amplification of the Human epidermal growth factor receptor type 2 (HER2) gene is present and overexpressed in 18–20% of breast cancers and historically has been associated with inferior disease-related outcomes. There has been increasing interest in de-escalation of therapy for low-risk disease. This study analyzes the cost-effectiveness of Doxorubicin/ Cyclophosphamide/ Paclitaxel/ Trastuzumab (AC-TH) and Docetaxel/Carboplatin/Trastuzumab(TCH) from payer perspective over a 5 year time horizon. Methods A half-cycle corrected Markov model was built to simulate the process of breast cancer events and death occurred in both AC-TH and TCH armed patients. Cost data came from studies based on a Chinese hospital. One-way sensitivity analyses as well as second-order Monte Carlo and probabilistic sensitivity analyses were performed.The transition probabilities and utilities were extracted from published literature, and deterministic sensitivity analyses were conducted. Results We identified 41 breast cancer patients at Hangzhou First People’s Hospital, among whom 15 (60%) had a partial response for AC-TH treatment and 13 (81.25%) had a partial response for TCH treatment.No cardiac toxicity was observed. Hematologic grade 3 or 4 toxicities were observed in 1 of 28 patients.Nonhematologic grade 3 or 4 toxicities with a reverse pattern were observed in 6 of 29 patients. The mean QALY gain per patient compared with TCH was 0.25 with AC-TH, while the incremental costs were $US13,142. The incremental cost-effectiveness ratio (ICER) of AC-TH versus TCH was $US 52,565 per QALY gained. Conclusions This study concluded that TCH neoadjuvant chemotherapy was feasible and active in HER2-overexpressing breast cancer patients in terms of the pathological complete response, complete response, and partial response rates and manageable toxicities.

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Cost effectiveness of DPYD genotyping to screen for dihydropyrimidine dehydrogenase (DPD) deficiency prior to adjuvant chemotherapy for colon cancer.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.3_suppl.55 ◽

2021 ◽

Vol 39 (3_suppl) ◽

pp. 55-55

Author(s):

Gabriel A. Brooks ◽

Stephanie Tapp ◽

Allan T. Daly ◽

Jonathan Busam ◽

Anna N.A. Tosteson

Keyword(s):

Colon Cancer ◽

Cost Effectiveness ◽

Adjuvant Chemotherapy ◽

Transition Probabilities ◽

Dihydropyrimidine Dehydrogenase ◽

Sensitivity Analyses ◽

Dpyd Gene ◽

Cancer Screen ◽

The Cost ◽

The U.S

55 Background: Fluoropyrimidine chemotherapy agents, including 5-fluorouracil and capecitabine, are the backbone of adjuvant treatment for colon cancer, and adjuvant chemotherapy substantially reduces recurrence and mortality after surgical resection of stage 3 colon cancer. While fluoropyrimidine chemotherapy is generally safe, the risk of severe, potentially fatal chemotherapy toxicity is substantially increased for the 2-3% of U.S. patients with DPD deficiency caused by pathogenic variants in the DPYD gene. DPYD genotype testing is readily available in the U.S. but has not been widely adopted. We evaluated the cost effectiveness of DPYD genotyping prior to adjuvant chemotherapy for colon cancer in the U.S. Methods: We constructed a Markov model to simulate screening for DPD deficiency with DPYD genotyping (versus no screening) among patients receiving fluoropyrimidine-based adjuvant chemotherapy for stage 3 colon cancer. Screen-positive patients were modeled to receive dose-reduced fluoropyrimidine chemotherapy. Model transition probabilities for treatment-related toxicities were derived from published clinical trial data with annotation of DPYD genotype and chemotherapy dosing strategy. Our analysis is from the healthcare perspective, with a time horizon of five years and an annual discount rate of 3% for future costs and benefits. Direct healthcare costs and health utilities were estimated from published sources and converted to 2020 US dollars, and post-treatment survival was modeled from SEER data. The primary outcome was the incremental cost-effectiveness ratio (ICER), defined as dollars per quality-adjusted life year (QALY). We used a value of $100,000/QALY as the cost-effectiveness threshold. One-way sensitivity analyses were used to examine model uncertainty. Results: Compared with no screening, screening for DPD deficiency with DPYD genotyping increased per-patient costs by $106 and improved quality-adjusted survival by 0.0028 QALYs, leading to an ICER of $37,300/QALY. In one-way sensitivity analyses, the ICER exceeded $100,000/QALY when the carrier frequency of pathogenic DPYD gene variants was less than 1.17%, and when the specificity of DPYD genotyping was less than 98.9%. Cost-effectiveness estimates were not sensitive to the cost of DPYD genotyping, the cost of toxicity-related hospitalizations, or the health utility associated with grade 3-4 toxicity. Conclusions: Among patients receiving adjuvant chemotherapy for stage 3 colon cancer, screening for DPD deficiency with DPYD genotyping is a cost-effective strategy for preventing infrequent but severe, sometimes fatal toxicities of fluoropyrimidine chemotherapy.

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COST-EFFECTIVENESS ANALYSIS OF IVABRADINE IN TREATMENT OF PATIENTS WITH HEART FAILURE IN IRAN

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462318003598 ◽

2018 ◽

Vol 34 (6) ◽

pp. 576-583 ◽

Cited By ~ 2

Author(s):

Saeed Taheri ◽

Elham Heidari ◽

Mohammad Ali Aivazi ◽

Mehran Shams-Beyranvand ◽

Mehdi Varmaghani

Keyword(s):

Heart Failure ◽

Sensitivity Analysis ◽

Cost Effectiveness ◽

Incremental Cost ◽

Transition Probabilities ◽

Drug Acquisition ◽

Sensitivity Analyses ◽

Baseline Heart Rate ◽

Life Years ◽

The Cost

Objectives:This study aimed to assess the cost-effectiveness of ivabradine plus standard of care (SoC) in comparison with current SoC alone from the Iranian payer perspective.Methods:A cohort-based Markov model was developed to assess the incremental cost-effectiveness ratio (ICER) over a 10-year time horizon in a cohort of 1,000 patients. The baseline transition probabilities between New York Heart Association (NYHA), mortality rate, and hospitalization rate were extracted from the literature. The effect of ivabradine on mortality, hospitalization, and NYHA improvement or worsening were retrieved from the SHIFT study. The effectiveness was measured as quality-adjusted life-years (QALYs) using the utility values derived from Iranian Heart Failure Quality of Life study. Direct medical costs were obtained from hospital records and national tariffs. Deterministic and probabilistic sensitivity analyses were conducted to show the robustness of the model.Results:Ivabradine therapy was associated with an incremental cost per QALY of USD $5,437 (incremental cost of USD $2,207 and QALYs gained 0.41) versus SoC. The probabilistic sensitivity analysis showed that ivabradine is expected to have a 60 percent chance of being cost-effective accepting a threshold of USD $6,550 per QALY. Furthermore, deterministic sensitivity analysis indicated that the model is sensitive to the ivabradine drug acquisition cost.Conclusions:The cost-effectiveness model suggested that the addition of ivabradine to SoC therapy was associated with improved clinical outcomes along with increased costs. The analysis indicates that the clinical benefit of ivabradine can be achieved at a reasonable cost in eligible heart failure patients with sinus rhythm and a baseline heart rate ≥ 75 beats per minute (bpm).

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Cost-effectiveness analysis of pembrolizumab for BCG-unresponsive carcinoma in situ of the bladder.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.6_suppl.395 ◽

2021 ◽

Vol 39 (6_suppl) ◽

pp. 395-395

Author(s):

Vidit Sharma ◽

Kevin Wymer ◽

Christopher Saigal ◽

Karim Chamie ◽

Mark S. Litwin ◽

...

Keyword(s):

Sensitivity Analysis ◽

Cost Effectiveness ◽

Willingness To Pay ◽

Carcinoma In Situ ◽

Cost Effective ◽

Index Patient ◽

Price Reduction ◽

Sensitivity Analyses ◽

Effectiveness Analysis

395 Background: Patients with BCG-unresponsive carcinoma in situ (CIS) are treated with radical cystectomy (RCx) or salvage intravesical chemotherapy (SIC). Recently, the FDA approved pembrolizumab for BCG-unresponsive CIS +/- papillary tumors. Given the costs and toxicities of pembrolizumab, it remains unclear whether its benefits are sufficient to warrant widespread use for BCG-unresponsive CIS. To that end, we conducted a cost-effectiveness analysis comparing pembrolizumab with RCx and SIC (using gemcitabine-docetaxel as the prototypical regimen) for patients with BCG-unresponsive CIS. Methods: A decision-analytic Markov model compared pembrolizumab, SIC (with gemcitabine-docetaxel), and RCx for patients with BCG-unresponsive CIS +/- papillary tumors who are RCx candidates (index patient 1) or are unwilling/unable to undergo RCx (index patient 2). Each treatment option was a Markov node containing distinct variations of the following health states: surveillance, recurrence, progression to MIBC, progression to metastasis, treatment toxicity, and death. Incremental Cost-Effectiveness Ratios (ICERs) were compared using a willingness-to-pay threshold of $100,000/Quality-adjusted life year (QALY). The model used a US Medicare perspective with a 5-year time horizon for the base case. One-way and probabilistic sensitivity analyses were performed for all model parameters. Results: For index patient 1, pembrolizumab was not cost-effective vs. RCx (ICER $1,403,008) or SIC (ICER $2,011,923). One-way sensitivity analysis revealed that pembrolizumab only became cost-effective relative to RCx with a > 93% price reduction. Relative to RCx, SIC was cost-effective for time horizons < 5 years and nearly cost-effective at 5 years (ICER $118,324). One-way sensitivity analysis revealed that SIC became cost-effective relative to RCx if its risk of recurrence or metastasis at 2 years was less than 55% or 5.9%, respectively. For index patient 2, pembrolizumab required > 90% price reduction to be cost-effective vs. RCx (ICER $1,073,240). Probabilistic sensitivity analyses revealed that pembrolizumab was unlikely to be cost-effective even at high willingness-to-pay thresholds. Further sensitivity analyses found that no two-way combination of extrapolated values resulted in pembrolizumab being favored over RCx or SIC for either index patient. Conclusions: Based on decision-analytic Markov modeling of treatment options for patients with BCG-unresponsive CIS, pembrolizumab was unlikely to be cost-effective without a > 90% price reduction. While both RCx and SIC were more cost-effective than pembrolizumab, further studies may validate the cost-effectiveness of gemcitabine-docetaxel relative to RCx if the recurrence and metastasis thresholds are met. Overall, our model supports the preferential use of RCx and SIC over pembrolizumab for BCG-unresponsive CIS.

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Measuring Cost-Effectiveness in Terms of Happiness

The Origins of Happiness ◽

10.23943/princeton/9780691196336.003.0016 ◽

2019 ◽

pp. 197-210

Author(s):

Andrew E. Clark ◽

Sarah Flèche ◽

Richard Layard ◽

Nattavudh Powdthavee ◽

George Ward

Keyword(s):

Cost Effectiveness ◽

Social Science ◽

Willingness To Pay ◽

Policy Analysis ◽

Policy Change ◽

Public Services ◽

Controlled Experiments ◽

The Public ◽

The People ◽

The Impact

This chapter demonstrates that policy analysis should be based on happiness as the measure of benefit (except where traditional methods actually work). It argues that this should be generally applied throughout the public services and by nongovernment organizations (NGOs). The chapter offers four key proposals. The first is that the goal of governments should be to increase the happiness of the people and, especially, to reduce misery. Where willingness to pay is not a feasible measure of benefit, governments should develop new methods of policy analysis based on point-years of happiness as the measure of benefit. All policy change should be evaluated through controlled experiments in which the impact on happiness is routinely measured. A major objective of social science (and of its funders) should be to throw light on the causes of happiness, and how it can be enhanced—and at what cost.

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Analysis of the effect of crossover from placebo (PBO) to darolutamide (DARO) on overall survival (OS) benefit in the ARAMIS Trial.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.6_suppl.240 ◽

2021 ◽

Vol 39 (6_suppl) ◽

pp. 240-240

Author(s):

Neal D. Shore ◽

Karim Fizazi ◽

Teuvo Tammela ◽

Murilo Luz ◽

Manuel Philco Salas ◽

...

Keyword(s):

Treatment Effect ◽

Safety Profile ◽

Final Analysis ◽

Phase Iii ◽

Sensitivity Analyses ◽

Primary Analysis ◽

Open Label ◽

Double Blind ◽

Secondary Endpoints ◽

The Impact

240 Background: DARO is a structurally distinct androgen receptor inhibitor approved for the treatment of non-metastatic castration-resistant prostate cancer (nmCRPC) based on significantly prolonged metastasis-free survival compared with PBO (median 40.4 vs 18.4 months; hazard ratio [HR] 0.41; 95% confidence interval [CI] 0.34–0.50; P < 0.0001) and a favorable safety profile in the phase III ARAMIS trial. Following unblinding at the primary analysis, crossover from PBO to DARO was permitted for the subsequent open-label treatment phase. Sensitivity analyses were performed to assess the effect of PBO–DARO crossover on OS benefit. Methods: Patients (pts) with nmCRPC receiving androgen deprivation therapy were randomized 2:1 to DARO (n = 955) or PBO (n = 554). In addition to OS, secondary endpoints included times to pain progression, first cytotoxic chemotherapy, first symptomatic skeletal event, and safety. The OS analysis was planned to occur after approximately 240 deaths, and secondary endpoints were evaluated in a hierarchical order. Iterative parameter estimation (IPE) and rank-preserving structural failure time (RPSFT) analyses were performed as pre-planned sensitivity analyses to adjust for the treatment effect of PBO–DARO crossover. The IPE method used a parametric model for the survival times and iteratively determined the model parameter describing the magnitude of the treatment effect, whereas a grid search and non-parametric log-rank test were used for the RPSFT analysis. The IPE and RPSFT analyses both generated a Kaplan–Meier curve for the PBO arm that predicts what would have been observed in the absence of PBO–DARO crossover. Results: After unblinding, 170 pts (30.7% of those randomized to PBO) crossed over from PBO to DARO; median treatment duration from unblinding to the final data cut-off was 11 months. Final analysis of the combined double-blind and open label periods was conducted after 254 deaths (15.5% of DARO and 19.1% of PBO pts) and showed a statistically significant OS benefit for DARO vs PBO (HR 0.69; 95% CI 0.53–0.88; P = 0.003). Results from the IPE (HR 0.66; 95% CI 0.51–0.84; P < 0.001) and RPSFT (HR 0.68; 95% CI 0.51–0.90; P = 0.007) analyses were similar to those from the intention-to-treat population, showing that the impact of PBO–DARO crossover was small. Additional analyses accounting for the effect of PBO–DARO crossover will be presented. The safety profile of DARO continued to be favorable at the final analysis, and discontinuation rates at the end of the double-blind period remained unchanged from the primary analysis (8.9% with DARO and 8.7% with PBO). Conclusions: Early treatment with DARO in men with nmCRPC is associated with significant improvement in OS regardless of pts crossing over from PBO to DARO. The safety profile of DARO remained favorable at the final analysis. Clinical trial information: NCT02200614.

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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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