scholarly journals Cost-effectiveness of pharmacogenetic-guided treatment: are we there yet?

2016 ◽  
Author(s):  
Moira Verbelen ◽  
Michael E Weale ◽  
Cathryn M Lewis

AbstractPharmacogenetics (PGx) has the potential to personalize pharmaceutical treatments. Many relevant gene-drug associations have been discovered, but PGx guided treatment needs to be cost-effective as well as clinically beneficial to be incorporated into standard healthcare. Progress in this area can be assessed by reviewing economic evaluations to determine the cost-effectiveness of PGx testing versus standard treatment. We performed a review of economic evaluations for PGx associations listed in the US Food and Drug Administration (FDA) Table of Pharmacogenomic Biomarkers in Drug Labeling (http://www.fda.gov/Drugs/ScienceResearch/ResearchAreas/Pharmacogenetics/ucm083378.htm). We determined the proportion of evaluations that found PGx guided treatment to be cost-effective or dominant over the alternative strategies, and we estimated the impact on this proportion of removing the cost of genetic testing. Of the 130 PGx associations in the FDA table, 44 economic evaluations, relating to 10 drugs, were identified. Of these evaluations, 57% drew conclusions in favour of PGx testing, of which 30% were cost-effective and 27% were dominant (cost-saving). If genetic information was freely available, 75% of economic evaluations would support PGx guided treatment, of which 25% would be cost-effective and 50% would be dominant. Thus, PGx guided treatment can be a cost-effective and even cost-saving strategy. Having genetic information readily available in the clinical health record is a realistic future prospect, and would make more genetic tests economically worthwhile. However, few drugs with PGx associations have been studied and more economic evaluations are needed to underpin the uptake of genetic testing in clinical practice.

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Obinna Ikechukwu Ekwunife ◽  
Chinelo Janefrances Ofomata ◽  
Charles Ebuka Okafor ◽  
Maureen Ugonwa Anetoh ◽  
Stephen Okorafor Kalu ◽  
...  

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).


2019 ◽  
Vol 70 (1) ◽  
pp. 26-29 ◽  
Author(s):  
Tinevimbo Shiri ◽  
Angela Loyse ◽  
Lawrence Mwenge ◽  
Tao Chen ◽  
Shabir Lakhi ◽  
...  

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.


2021 ◽  
Author(s):  
Youwen Zhu ◽  
Huabin Hu ◽  
Dong Ding ◽  
Shuosha Li ◽  
Mengting Liao ◽  
...  

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.


2019 ◽  
Vol 5 (3) ◽  
pp. 28 ◽  
Author(s):  
Alice Bessey ◽  
James Chilcott ◽  
Joanna Leaviss ◽  
Carmen de la Cruz ◽  
Ruth Wong

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.


2020 ◽  
Vol 35 (2) ◽  
pp. 434-445 ◽  
Author(s):  
Joseph H Lipton ◽  
Mahdi Zargar ◽  
Ellen Warner ◽  
Ellen E Greenblatt ◽  
Esther Lee ◽  
...  

Abstract STUDY QUESTION Is it cost-effective to use in vitro fertilisation and preimplantation genetic testing of monogenic defects (IVT/PGT-M) to prevent transmission of BRCA1/2 mutations to second-generation new births in comparison with naturally conceived births? SUMMARY ANSWER In this cost-effectiveness analysis, we found that IVF/PGT-M is cost-effective for BRCA1 and BRCA2 mutation carriers if using a willingness to pay of $50 000 per quality-adjusted life-year (QALY). WHAT IS KNOWN ALREADY Carriers of a BRCA1 or BRCA2 mutation have a significantly increased risk of several types of cancer throughout their lifetime. The cost of risk reduction, screening and treatment of cancer in this population is high. In addition, there is a 50% chance of passing on this genetic mutation to each child. One option to avoid transmission of an inherited deleterious gene to one’s offspring involves in vitro fertilisation with preimplantation genetic testing. STUDY DESIGN, SIZE, DURATION We implemented a state transition model comparing the healthcare impact of a cohort of healthy children born after IVF/PGT-M, who have a population risk of developing cancer, to a cohort of naturally conceived live-births, half of whom are carriers of the BRCA mutation. Transition probabilities are based on published sources, a lifetime horizon and a perspective of a provincial Ministry of Health in Canada. PARTICIPANTS/MATERIALS, SETTING, METHODS The target population is the second-generation new births who have at least one parent with a known BRCA1 or BRCA2 mutation. MAIN RESULTS AND THE ROLE OF CHANCE At a willingness-to-pay threshold of $50 000 per QALY, IVF/PGT-M is a cost-effective intervention for carriers of either BRCA mutation. For BRCA1, the incremental cost-effectiveness ratio (ICER) for IVF/PGT-M is $14 242/QALY. For BRCA2, the ICER of intervention is $12 893/QALY. Probabilistic sensitivity analysis results show that IVF/PGT-M has a 98.4 and 97.3% chance of being cost-effective for BRCA1 and BRCA2 mutation carriers, respectively, at the $50 000/QALY threshold. LIMITATIONS, REASONS FOR CAUTION Our model did not include the short-term negative effect of IVF/PGT-M on the woman’s quality of life; in addition, our model did not consider any ethical issues related to post-implantation genetic testing. WIDER IMPLICATIONS OF THE FINDINGS In countries in which the healthcare of a large segment of the population is covered by a single payer system such as the government, it would be cost-effective for that payer to cover the cost of IVF/PGT-M for couples in which one member has a BRCA mutation, in order to avoid the future costs and disutility of managing offspring with an inherited BRCA mutation. STUDY FUNDING/COMPETING INTEREST(S) Dr Wong’s research program was supported by the Canadian Institutes of Health Research (CIHR), the Natural Sciences and Engineering Research Council (NSERC), the Canadian Liver Foundation and an Ontario Ministry of Research, Innovation and Science Early Researcher Award. All authors declared no conflict of interests.


2014 ◽  
Vol 30 (3) ◽  
pp. 273-281 ◽  
Author(s):  
Davide Minniti ◽  
Ottavio Davini ◽  
Maria Rosaria Gualano ◽  
Maria Michela Gianino

Objectives:The study question was whether dual-energy X-ray absorptiometry (DXA) alone is more cost-effective for identifying postmenopausal women with osteoporosis than a two-step procedure with quantitative ultrasound sonography (QUS) plus DXA. To answer this question, a systematic review was performed.Methods:Electronic databases (PubMed, INAHTA, Health Evidence Network, NIHR, the Health Technology Assessment program, the NHS Economic Evaluation Database, Research Papers in Economics, Web of Science, Scopus, and EconLit) were searched for cost-effectiveness publications. Two independent reviewers selected eligible publications based on the inclusion/exclusion criteria. Quality assessment of economic evaluations was undertaken using the Drummond checklist.Results:Seven journal articles and four reports were reviewed. The cost per true positive case diagnosed by DXA was found to be higher than that for diagnosis by QUS+DXA in two articles. In one article it was found to be lower. In three studies, the results were not conclusive. These articles were characterized by the differences in the types of devices, parameters and thresholds on the QUS and DXA tests and the unit costs of the DXA and QUS tests as well as by variability in the sensitivity and specificity of the techniques and the prevalence of osteoporosis.Conclusions:The publications reviewed did not provide clear-cut evidence for drawing conclusions about which screening test may be more cost-effective for identifying postmenopausal women with osteoporosis.


2017 ◽  
Vol 4 (suppl_1) ◽  
pp. S64-S65
Author(s):  
Emily Hyle

Abstract Background Most measles importations are due to returning US travelers infected during international travel. We projected clinical outcomes and assessed cost-effectiveness of pretravel evaluation for measles immunity and MMR vaccination among eligible adult US international travelers. Methods We designed a decision tree to investigate pretravel evaluation compared with no evaluation from the societal perspective. Data from the Global TravEpiNet Consortium and published literature informed input parameters (Figure 1). Outcomes included measles cases averted per 10 million travelers, costs, and the incremental cost-effectiveness ratio (ICER, Δcosts/Δmeasles cases averted); we considered ICERs < $100,000/measles case averted to be cost-effective. We performed sensitivity analyses to assess the impact of varying the probability of exposure based on travel destination, and the percentage of travelers with pre-existing measles immunity. Results In the base case, departure after pretravel evaluation resulted in 16 measles importations and 46 transmissions per 10 million travelers and cost $132 million, vs without pretravel evaluation (26 importations and 87 transmissions per 10 million travelers, costing $22 million). Pretravel evaluation averted 51 measles cases per 10 million travelers with an ICER of $2.2 million per case averted. Results were most sensitive to the probability of measles exposure and the traveler’s pre-existing immunity (Figure 2). Pretravel evaluation was cost-effective for travelers to Asia if pre-existing measles immunity was <80%. Evaluation was always cost-effective for travelers to Africa when pre-existing immunity was less than 100% and became cost saving when the percentage of immune travelers was lower (<70%). Travelers who were more likely to be non-immune and were visiting destinations with higher probabilities of exposure were most likely to benefit from pretravel evaluation for measles immunity at excellent economic value. Conclusion As risk of measles exposure increases and likelihood of travelers’ pre-existing immunity decreases, it can be cost-effective or cost saving to assess US international travelers’ measles immunity status and vaccinate with MMR prior to departure. Disclosures All authors: No reported disclosures.


2019 ◽  
Vol 74 (Supplement_5) ◽  
pp. v5-v16 ◽  
Author(s):  
Zoe Ward ◽  
Linda Campbell ◽  
Julian Surey ◽  
Steven Platts ◽  
Rachel Glass ◽  
...  

Abstract Background HCV disproportionately affects marginalized communities such as homeless populations and people who inject drugs (PWID), posing a challenge to traditional health services. The HepFriend initiative in London is a model of care utilizing HCV outreach screening and peer support to link vulnerable individuals to HCV treatment in secondary care. Objectives To assess the cost-effectiveness of the HepFriend initiative from a healthcare provider perspective, compared with standard-of-care pathways (consisting of testing in primary care and other static locations, including drug treatment centres, and linkage to secondary care). Methods Cost-effectiveness analysis using a dynamic HCV transmission and disease progression model among PWID and those who have ceased injecting, including housing status and drug treatment service contact. The model was parameterized using London-specific surveillance and survey data, and primary intervention cost and effectiveness data (September 2015 to June 2018). Out of 461 individuals screened, 197 were identified as HCV RNA positive, 180 attended secondary care and 89 have commenced treatment to date. The incremental cost-effectiveness ratio (ICER) was determined using a 50 year time horizon. Results For a willingness-to-pay threshold of £20000 per QALY gained, the HepFriend initiative is cost-effective, with a mean ICER of £9408/QALY, and would become cost saving at 27% (£10525 per treatment) of the current drug list price. Results are robust to variations in intervention costs and model assumptions, and if treatment rates are doubled the intervention becomes more cost-effective (£8853/QALY). Conclusions New models of care that undertake active case-finding with enhanced peer support to improve testing and treatment uptake amongst marginalized and vulnerable groups could be highly cost-effective and possibly cost saving.


2019 ◽  
Vol 4 (4) ◽  
pp. 378-387 ◽  
Author(s):  
S.S. Huang ◽  
R.R. Ruff ◽  
R. Niederman

Introduction: Current economic evaluations of school-based caries prevention programs (SCPPs) do not compare multiple types of SCPPs against each other and do not consider teeth beyond permanent first molars. Objectives: To assess the cost-effectiveness of a comprehensive SCPP relative to an SCPP focused on delivering sealants for permanent first molars only and to a default of no SCPP. Based on a societal perspective, a simulation model was used that compared the health and cost impacts on 1) permanent first molars only and 2) all posterior teeth. Methods: To calibrate the model, we used data from CariedAway, a comprehensive SCPP that used glass ionomer to prevent and arrest active decay among children. We then evaluated the incremental cost-effectiveness of implementing 3 alternate school-based approaches (comprehensive, sealant only, and no program) on only first molars and all posterior teeth. Probabilistic, 1-, and 2-way sensitivity analyses are included for robustness. Cost-effectiveness is assessed with a threshold of $54,639 per averted disability-adjusted life year (DALY). Results: We first compared the 3 programs under the assumption of treating only first molars. This assessment indicated that CariedAway was less cost-effective than school-based sealant programs (SSPs): the resulting incremental cost-effectiveness ratio (ICER) for CariedAway versus SSPs was $283,455 per averted DALY. However, when the model was extended to include CariedAway’s treatment of all posterior teeth, CariedAway was not only cost-effective but also cost-saving relative to SSPs (ICER, –$943,460.88 per averted DALY; net cost, –$261.45) and no SCPP (ICER, –$400,645.52 per averted DALY; net cost, –$239.77). Conclusions: This study finds that economic evaluations assessing only cost and health impacts on permanent first molars may underestimate the cost-effectiveness of comprehensive SCPPs 1) preventing and arresting decay and 2) treating all teeth. Hence, there is an urgent need for economic evaluations of SCPPs to assess cost and health impacts across teeth beyond only permanent first molars. Knowledge Transfer Statement: The results of this study can be used by policy makers to understand how to evaluate economic evaluations of school-based caries prevention programs and what factors to consider when deciding on what types of programs to implement.


2019 ◽  
Vol 3 (3) ◽  
Author(s):  
Iakovos Toumazis ◽  
Emily B Tsai ◽  
S Ayca Erdogan ◽  
Summer S Han ◽  
Wenshuai Wan ◽  
...  

Abstract Background Numerous health policy organizations recommend lung cancer screening, but no consensus exists on the optimal policy. Moreover, the impact of the Lung CT screening reporting and data system guidelines to manage small pulmonary nodules of unknown significance (a.k.a. indeterminate nodules) on the cost-effectiveness of lung cancer screening is not well established. Methods We assess the cost-effectiveness of 199 screening strategies that vary in terms of age and smoking eligibility criteria, using a microsimulation model. We simulate lung cancer-related events throughout the lifetime of US-representative current and former smokers. We conduct sensitivity analyses to test key model inputs and assumptions. Results The cost-effectiveness efficiency frontier consists of both annual and biennial screening strategies. Current guidelines are not on the frontier. Assuming 4% disutility associated with indeterminate findings, biennial screening for smokers aged 50–70 years with at least 40 pack-years and less than 10 years since smoking cessation is the cost-effective strategy using $100 000 willingness-to-pay threshold yielding the highest health benefit. Among all health utilities, the cost-effectiveness of screening is most sensitive to changes in the disutility of indeterminate findings. As the disutility of indeterminate findings decreases, screening eligibility criteria become less stringent and eventually annual screening for smokers aged 50–70 years with at least 30 pack-years and less than 10 years since smoking cessation is the cost-effective strategy yielding the highest health benefit. Conclusions The disutility associated with indeterminate findings impacts the cost-effectiveness of lung cancer screening. Efforts to quantify and better understand the impact of indeterminate findings on the effectiveness and cost-effectiveness of lung cancer screening are warranted.


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