scholarly journals Implementing Interventions with Varying Marginal Cost-Effectiveness: An Application in Precision Medicine

2020 ◽  
Vol 40 (7) ◽  
pp. 924-938
Author(s):  
Stuart J. Wright ◽  
Mike Paulden ◽  
Katherine Payne

Purpose. A range of barriers may constrain the effective implementation of strategies to deliver precision medicine. If the marginal costs and consequences of precision medicine vary at different levels of implementation, then such variation will have an impact on relative cost-effectiveness. This study aimed to illustrate the importance and quantify the impact of varying marginal costs and benefits on the value of implementation for a case study in precision medicine. Methods. An existing method to calculate the value of implementation was adapted to allow marginal costs and consequences of introducing precision medicine into practice to vary across differing levels of implementation. This illustrative analysis used a case study based on a published decision-analytic model-based cost-effectiveness analysis of a 70-gene recurrence score (MammaPrint) for breast cancer. The impact of allowing for varying costs and benefits for the value of the precision medicine and of implementation strategies was illustrated graphically and numerically in both static and dynamic forms. Results. The increasing returns to scale exhibited by introducing this specific example of precision medicine mean that a minimum level of implementation (51%) is required for using the 70-gene recurrence score to be cost-effective at a defined threshold of €20,000 per quality-adjusted life year. The observed variation in net monetary benefit implies that the value of implementation strategies was dependent on the initial and ending levels of implementation in addition to the magnitude of the increase in patients receiving the 70-gene recurrence score. In dynamic models, incremental losses caused by low implementation accrue over time unless implementation is improved. Conclusions. Poor implementation of approaches to deliver precision medicine, identified to be cost-effective using decision-analytic model-based cost-effectiveness analysis, can have a significant economic impact on health systems. Developing and evaluating the economic impact of strategies to improve the implementation of precision medicine will potentially realize the more cost-effective use of health care budgets.

2020 ◽  
Vol 21 (3) ◽  
pp. 437-449 ◽  
Author(s):  
Alexander Kuhlmann ◽  
Henning Krüger ◽  
Susanne Seidinger ◽  
Andreas Hahn

Abstract Background The safe use of a prosthesis in activities of daily living is key for transfemoral amputees. However, the number of falls varies significantly between different prosthetic device types. This study aims to compare medical and economic consequences of falls in transfemoral amputees who use the microprocessor-controlled knee joint C-Leg with patients who use non-microprocessor-controlled (mechanical) knee joints (NMPK). The main objectives of the analysis are to investigate the cost-effectiveness and budget impact of C-Legs in transfemoral amputees with diabetes mellitus (DM) and without DM in Germany. Methods A decision-analytic model was developed that took into account the effects of prosthesis type on the risk of falling and fall-related medical events. Cost-effectiveness and budget impact analyses were performed separately for transfemoral amputees with and without DM. The study took the perspective of the statutory health insurance (SHI). Input parameters were derived from the published literature. Univariate and probabilistic sensitivity analyses (PSA) were performed to investigate the impact of changes in individual input parameter values on model outcomes and to explore parameter uncertainty. Results C-Legs reduced the rate of fall-related hospitalizations from 134 to 20 per 1000 person years (PY) in amputees without DM and from 146 to 23 per 1000 PY in amputees with DM. In addition, the C-Leg prevented 15 or 14 fall-related death per 1000 PY. Over a time horizon of 25 years, the incremental cost-effectiveness ratio (ICER) was 16,123 Euro per quality-adjusted life years gained (QALY) for amputees without DM and 20,332 Euro per QALY gained for amputees with DM. For the period of 2020–2024, the model predicted an increase in SHI expenditures of 98 Mio Euro (53 Mio Euro in prosthesis users without DM and 45 Mio Euro in prosthesis users with DM) when all new prosthesis users received C-Legs instead of NMPKs and 50% of NMPK user whose prosthesis wore out switched to C-Legs. Results of the PSA showed moderate uncertainty and a probability of 97–99% that C-Legs are cost-effective at an ICER threshold of 40,000 Euro (≈ German GDP per capita in 2018) per QALY gained. Conclusion Results of the study suggest that the C-Leg provides substantial additional health benefits compared with NMPKs and is likely to be cost-effective in transfemoral amputees with DM as well as in amputees without DM at an ICER threshold of 40,000 Euro per QALY gained.


2012 ◽  
Vol 28 (3) ◽  
pp. 241-248 ◽  
Author(s):  
Lanting Lu ◽  
Jaime Peters ◽  
Chris Roome ◽  
Ken Stein

Objectives:The aim of this study was to evaluate the cost-effectiveness of alemtuzumab (CAMPATH-1H) compared with conventional chemotherapy in people with T-cell prolymphocytic leukemia (T-PLL).Methods:We developed a decision-analytic model to assess the costs and benefits of alemtuzumab or conventional therapy based on their effects on quality of life of patients. The main outcome was the incremental cost-effectiveness ratio incorporating costs per additional quality-adjusted life-year (QALY) gained over lifetime. Due to the limited data available, a large number of assumptions had to be made to construct the cost-utility model. One-way, multi-way, and probabilistic sensitivity analyses (PSA) were conducted to explore the impact of these uncertainties. Expected values of perfect information were also calculated for four specific scenarios.Results:Depending on different key assumptions made, the PSA suggested distinct conclusions using a willingness-to-pay threshold of 30,000 GBP per QALY gained. Using this threshold, the probability that alemtuzumab would be cost-effective varies from 0 percent to 53 percent for the four modeled scenarios. Population expected value of perfect information analysis suggests that resolving the parameter uncertainty in the analysis for people with T-PLL in the United Kingdom would have considerable value—up to 5.3 million euro.Conclusions:Alemtuzumab appears more likely to be cost-effective if used earlier in the course of T-PLL and where it replaces the use of multiple alternative therapies. However, cost-effectiveness is highly uncertain and future research is clearly justified. Nevertheless, our analysis demonstrates the feasibility of considering the cost-effectiveness of an agent despite the presence of significant uncertainty to provide appropriate assessment information to policy makers.


2021 ◽  
pp. 00790-2020
Author(s):  
Tima Mohammadi ◽  
Mohsen Sadatsafavi ◽  
Chris Carlsten

BackgroundThe demonstrable value of precision medicine, in the context of common environmental exposures, has scarcely been explored. This study evaluated the cost-effectiveness of a preventive personalised intervention to reduce the adverse effect of air pollution in the context of asthma.MethodsA decision-analytic model was used to conduct a cost-utility analysis of prevention interventions in case of acute exposure to air pollution in mild asthma. Three different strategies, as follows, were compared: no preventive intervention, precision health strategy based on information from genotype testing – followed with treating high-risk patients, and prescribing additional medication to all mild asthmatics as a preventive intervention. The costs and quality-adjusted life years (QALYs) in the base case and alternative scenarios were obtained through probabilistic analysis.ResultsThe results showed that the precision prevention intervention (anticipatory intervention for asthmatics, guided by relevant genetic abnormality, in the face of acute air pollution) is a cost-effective strategy compared to no such intervention, with an incremental cost-effectiveness ratio of $49 555 per QALY. Furthermore, this strategy is a dominant strategy compared to an intervention that prescribes medication indiscriminately to all asthmatics.ConclusionThe incorporation of genomic testing, to stratify risk of asthmatics to pollution-driven exacerbations, and then tailoring a preventive intervention accordingly, may be cost-effective relative to untailored methods. These results lend plausibility to the use of precision medicine for limiting asthma exacerbation in the context of air pollution and, potentially, other exposures.


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.


2010 ◽  
Vol 196 (5) ◽  
pp. 396-403 ◽  
Author(s):  
Djøra I. Soeteman ◽  
Roel Verheul ◽  
Jos Delimon ◽  
Anke M. M. A. Meerman ◽  
Ellen van den Eijnden ◽  
...  

BackgroundRecommendations on current clinical guidelines are informed by limited economic evidence.AimsA formal economic evaluation of three modalities of psychotherapy for patients with cluster B personality disorders.MethodA probabilistic decision-analytic model to assess the cost-effectiveness of out-patient, day hospital and in-patient psychotherapy over 5 years in terms of cost per recovered patient-year and cost per quality-adjusted life-year (QALY). Analyses were conducted from both societal and payer perspectives.ResultsFrom the societal perspective, the most cost-effective choice switched from out-patient to day hospital psychotherapy at a threshold of €12 274 per recovered patient-year; and from day hospital to in-patient psychotherapy at €113 298. In terms of cost per QALY, the optimal strategy changed at €56 325 and €286 493 per QALY respectively. From the payer perspective, the switch points were at €9895 and €155 797 per recovered patient-year, and €43 427 and €561 188 per QALY.ConclusionsOut-patient psychotherapy and day hospital psychotherapy are the optimal treatments for patients with cluster B personality disorders in terms of cost per recovered patient-year and cost per QALY.


2021 ◽  
Author(s):  
Leonardo Rojas ◽  
María Rojas-Reyes ◽  
Diego Rosselli ◽  
Andres F. Cardona

Abstract BackgroundThe best strategy to establish indication for adjuvant chemotherapy in early breast cancer (EBC) in Colombia is unknown. This study aimed to identify the cost-effectiveness of various strategies to establish the necessity of adjuvant chemotherapy.MethodsThis study used an adapted decision-analytic model to compare cost and outcomes of care that includes Oncotype DX™ (ODX) or Mammaprint™ (MMP) test with routine care without ODX or MMP tests (application of adjuvant chemotherapy for all patients) over a 5-year time horizon, and the from the perspective of the Colombian National Health System (NHS) perspective (payer). Data were obtained from published literature and clinical trial database. The study population was composed of women with EBC, hormone-receptor positive (HR+), Her2-negative, lymph-node negative (LN0), with high-risk clinical criteria for recurrence. The outcome measures were incremental cost-effectiveness ratio (ICER; 2019 United States Dollar [USD] per quality-adjusted life years [QALY] gained) and net monetary benefit (NMB).ResultsODX increases QALYs by 0.05 and MMP by 0.03 with savings of $2,445 and $570 compared with the standard strategy, respectively. The ICER for ODX was −$41,857 and that for MMP was −$18,253 per QALY; NMB was $2,821 and $771, respectively. Both tests were cost effective under defined threshold. When the two tests were compared, ODX was more cost effective than MMP. Sensitivity analysis revealed that, with a threshold of 1 GDP per capita, ODX will be cost effective in 95.5% of the cases compared with 70.2% of MMP. Probabilistic sensitivity analysis revealed that ODX was a consistently superior strategy.ConclusionsGenomic profiling using ODX or MMP tests to define the need of adjuvant chemotherapy treatment in patients with HR + and Her2 − EBC is a cost-effective strategy that allows Colombian NHS saving money.


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.


Nutrients ◽  
2019 ◽  
Vol 11 (6) ◽  
pp. 1235
Author(s):  
Anita E. Gandola ◽  
Livia Dainelli ◽  
Diane Zimmermann ◽  
Maznah Dahlui ◽  
Patrick Detzel

This study evaluated the cost-effectiveness of the consumption of a milk powder product fortified with potassium (+1050.28 mg/day) and phytosterols (+1200 mg/day) to lower systolic blood pressure and low-density lipoprotein cholesterol, respectively, and, therefore, the risk of myocardial infarction (MI) and stroke among the 35–75-year-old population in Malaysia. A Markov model was created against a do-nothing option, from a governmental perspective, and with a time horizon of 40 years. Different data sources, encompassing clinical studies, practice guidelines, grey literature, and statistical yearbooks, were used. Sensitivity analyses were performed to evaluate the impact of uncertainty on the base case estimates. With an incremental cost-effectiveness ratio equal to international dollars (int$) 22,518.03 per quality-adjusted life-years gained, the intervention can be classified as very cost-effective. If adopted nationwide, it would help prevent at least 13,400 MIs, 30,500 strokes, and more than 10,600 and 17,100 MI- and stroke-related deaths. The discounted cost savings generated for the health care system by those who consume the fortified milk powder would amount to int$8.1 per person, corresponding to 0.7% of the total yearly health expenditure per capita. Sensitivity analyses confirmed the robustness of the results. Together with other preventive interventions, the consumption of milk powder fortified with potassium and phytosterols represents a cost-effective strategy to attenuate the rapid increase in cardiovascular burden in Malaysia.


2020 ◽  
Vol 2020 ◽  
pp. 1-10 ◽  
Author(s):  
Brianna Lauren ◽  
Sassan Ostvar ◽  
Elisabeth Silver ◽  
Myles Ingram ◽  
Aaron Oh ◽  
...  

Background. The 5-year survival rate of patients with metastatic gastric cancer (GC) is only 5%. However, trials have demonstrated promising antitumor activity for targeted therapies/immunotherapies among chemorefractory metastatic GC patients. Pembrolizumab has shown particular efficacy among patients with programmed death ligand-1 (PD-L1) expression and high microsatellite instability (MSI-H). The aim of this study was to assess the effectiveness and cost-effectiveness of biomarker-guided second-line GC treatment. Methods. We constructed a Markov decision-analytic model using clinical trial data. Our model compared pembrolizumab monotherapy and ramucirumab/paclitaxel combination therapy for all patients and pembrolizumab for patients based on MSI status or PD-L1 expression. Paclitaxel monotherapy and best supportive care for all patients were additional comparators. Costs of drugs, treatment administration, follow-up, and management of adverse events were estimated from a US payer perspective. The primary outcomes were quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs) with a willingness-to-pay threshold of $100,000/QALY over 60 months. Secondary outcomes were unadjusted life years (survival) and costs. Deterministic and probabilistic sensitivity analyses were performed to evaluate model uncertainty. Results. The most effective strategy was pembrolizumab for MSI-H patients and ramucirumab/paclitaxel for all other patients, adding 3.8 months or 2.0 quality-adjusted months compared to paclitaxel. However, this strategy resulted in a prohibitively high ICER of $1,074,620/QALY. The only cost-effective strategy was paclitaxel monotherapy for all patients, with an ICER of $53,705/QALY. Conclusion. Biomarker-based treatments with targeted therapies/immunotherapies for second-line metastatic GC patients substantially improve unadjusted and quality-adjusted survival but are not cost-effective at current drug prices.


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