scholarly journals Measuring the cost-effectiveness of treatments for people with multiple sclerosis: Beyond quality-adjusted life-years

2020 ◽  
pp. 135245852095417
Author(s):  
Annie Hawton ◽  
Elizabeth Goodwin ◽  
Kate Boddy ◽  
Jennifer Freeman ◽  
Sarah Thomas ◽  
...  

Background: It is a familiar story. A promising multiple sclerosis (MS) treatment clears the three regulatory hurdles of safety, quality and efficacy, only to fall at the fourth: cost-effectiveness. This has led to concerns about the validity of the measures typically used to quantify treatment effects in cost-effectiveness analyses and in 2012, in the United Kingdom, the National Institute for Health and Care Excellence called for an improvement in the cost-effectiveness framework for assessing MS treatments. Objective and Methods: This review describes what is meant by cost-effectiveness in health/social care funding decision-making, and usual practice for assessing treatment benefits. Results: We detail the use of the quality-adjusted life-year (QALY) in resource allocation decisions, and set out limitations of this approach in the context of MS. Conclusion: We conclude by highlighting methodological and policy developments which should aid addressing these limitations.

PEDIATRICS ◽  
1993 ◽  
Vol 91 (5) ◽  
pp. 859-866
Author(s):  
Jonathan Javitt ◽  
Ronald Dei Cas ◽  
Yen-pin Chiang

Background. Retinopathy of prematurity (ROP) is the leading cause of blindness among premature infants. A recent National Eye Institute-sponsored prospective, multicenter trial investigating the use of cryotherapy for treatment of ROP demonstrates a significant reduction in blindness and low vision for patients with sight-threatening (stage 3+) ROP. Method. A microsimulation model is presented to determine the cost-effectiveness of cryotherapy for ROP. Simulations are performed for three subpopulations of premature infants with birth weights 500 through 749 g, 750 through 999 g, and 1000 through 1249 g, and for three screening strategies—weekly, biweekly, and monthly. Results. Appropriately timed screening for and treatment of ROP is predicted to result in a gain of 3899 to 4648 quality-adjusted-life-years and a net governmental budgetary savings of $38.3 to $64.9 million for each annual US birth cohort of 28 321 premature infants (500 through 1249 g). The cost per quality-adjusted-life-year gained is $2488 to $6045, depending on different screening strategies. Conclusions. Of greatest importance is the finding that properly timed screening and treatment for ROP is not only cost saving but may save approximately 320 infants per year from a lifetime of blindness.


Neurosurgery ◽  
2015 ◽  
Vol 76 (5) ◽  
pp. 552-562 ◽  
Author(s):  
Susana Esteves ◽  
Marta Alves ◽  
Marta Castel-Branco ◽  
Walter Stummer

Abstract BACKGROUND: High-grade gliomas are aggressive, incurable tumors characterized by extensive diffuse invasion of the normal brain parenchyma. Novel therapies at best prolong survival; their costs are formidable and benefit is marginal. Economic restrictions thus require knowledge of the cost-effectiveness of treatments. Here, we show the cost-effectiveness of enhanced resections in malignant glioma surgery using a well-characterized tool for intraoperative tumor visualization, 5-aminolevulinic acid (5-ALA). OBJECTIVE: To evaluate the cost-effectiveness of 5-ALA fluorescence-guided neurosurgery compared with white-light surgery in adult patients with newly diagnosed high-grade glioma, adopting the perspective of the Portuguese National Health Service. METHODS: We used a Markov model (cohort simulation). Transition probabilities were estimated with the use of data from 1 randomized clinical trial and 1 noninterventional prospective study. Utility values and resource use were obtained from published literature and expert opinion. Unit costs were taken from official Portuguese reimbursement lists (2012 values). The health outcomes considered were quality-adjusted life-years, life-years, and progression-free life-years. Extensive 1-way and probabilistic sensitivity analyses were performed. RESULTS: The incremental cost-effectiveness ratios are below &OV0556;10 000 in all evaluated outcomes, being around &OV0556;9100 per quality-adjusted life-year gained, &OV0556;6700 per life-year gained, and &OV0556;8800 per progression-free life-year gained. The probability of 5-ALA fluorescence-guided surgery cost-effectiveness at a threshold of &OV0556;20000 is 96.0% for quality-adjusted life-year, 99.6% for life-year, and 98.8% for progression-free life-year. CONCLUSION: 5-ALA fluorescence-guided surgery appears to be cost-effective in newly diagnosed high-grade gliomas compared with white-light surgery. This example demonstrates cost-effectiveness analyses for malignant glioma surgery to be feasible on the basis of existing data.


2019 ◽  
pp. 204748731987895 ◽  
Author(s):  
Kanila Perera ◽  
Zanfina Ademi ◽  
Danny Liew ◽  
Ella Zomer

Background The Comparison of Sacubitril–Valsartan versus Enalapril on Effect on NT-proBNP in Patients Stabilised from an Acute Heart Failure Episode (PIONEER-HF) trial demonstrated significant reductions in N-terminal pro-B-type natriuretic peptide. Our study explored the cost-effectiveness of the use of sacubitril-valsartan versus enalapril in acute decompensated heart failure from the Australian healthcare perspective. Methods A Markov model was designed using data from the PIONEER-HF trial to model the clinical progress and costs of patients over a lifetime time horizon. The model consisted of three health states: ‘alive and event-free’, ‘alive after non-fatal hospitalisation for acute decompensated heart failure’ or ‘dead’. Costs and utilities were estimated from published sources. The cost of sacubitril-valsartan (per the Australian pharmaceutical benefits schedule) was AU$7.08/day. Outcomes of interest were the incremental cost-effectiveness ratios in terms of cost per quality-adjusted life year gained and cost per year of life saved. Cost and benefits were discounted at 5.0% per annum. Results Compared to enalapril, sacubitril-valsartan was estimated to cost an additional AU$7464 (discounted) per person, but lead to 0.127 years of life saved (discounted) and 0.096 quality-adjusted life years gained (discounted) over a lifetime analysis. These equated to incremental cost-effectiveness ratios of AU$58,629/year of life saved (US$41,795, EU€58,629, GBP£32,001) and AU$77,889/quality-adjusted life year gained (US$55,526, EU€49,202, GBP£42,504). We have assumed a threshold of AU$50,000/quality-adjusted life year gained to suggest cost-effectiveness. Conclusions At its current acquisition price, sacubitril-valsartan in comparison to enalapril is not likely to be cost-effective in the management of acute decompensated heart failure in Australia. A price reduction of more than 25% would confer cost-effectiveness.


Immunotherapy ◽  
2021 ◽  
Author(s):  
Wei Jiang ◽  
Zhichao He ◽  
Tiantian Zhang ◽  
Chongchong Guo ◽  
Jianli Zhao ◽  
...  

Aim: To evaluate the cost–effectiveness of ribociclib plus fulvestrant versus fulvestrant in hormone receptor-positive/human EGF receptor 2-negative advanced breast cancer. Materials & methods: A three-state Markov model was developed to evaluate the costs and effectiveness over 10 years. Direct costs and utility values were obtained from previously published studies. We calculated incremental cost–effectiveness ratio to evaluate the cost–effectiveness at a willingness-to-pay threshold of $150,000 per additional quality-adjusted life year. Results: The incremental cost–effectiveness ratio was $1,073,526 per quality-adjusted life year of ribociclib plus fulvestrant versus fulvestrant. Conclusions: Ribociclib plus fulvestrant is not cost-effective versus fulvestrant in the treatment of advanced hormone receptor-positive/human EGF receptor 2-negative breast cancer. When ribociclib is at 10% of the full price, ribociclib plus fulvestrant could be cost-effective.


Author(s):  
Nayyereh Ayati ◽  
Lora Fleifel ◽  
Mohammad Ali Sahraian ◽  
Shekoufeh Nikfar

Background: Cladribine tablets are the foremost oral immune-reconstitution therapy for high disease activity relapsing multiple sclerosis (HDA-RMS). We aimed to assess the cost-effectiveness of cladribine tablets compared to natalizumab in patients with HDA-RMS in Iran. Methods: A 5-year cohort-based Markov model was developed with 11 expanded disability status score (EDSS) health states, including patients with HDA-RMS as on and off-treatment. All costs were identified from the literature and expert opinion and were measured in Iranian Rial rates, changed to the 2020 USD rate and were discounted by 7.2%. Quality adjusted life years (QALY), discounted by 3.5%, and life years gained (LYG) were adopted to measure efficacy. The final results were presented as incremental cost-effectiveness ratio that was compared to a national willingness to pay (WTP) threshold of 1 to 3 gross domestic product (GDP) per capita. Deterministic and probabilistic sensitivity analyses (D/PSA) were employed to evaluate uncertainty. Results: Cladribine tablets dominated natalizumab and yielded 6,607 USD cost-saving and 0.003 additional QALYs per patient. LYG was comparable. The main cost component was drug acquisition cost in both arms. DSA indicated the sensitivity of the results to the cost discount rates and also the patients’ body weight; while they were less sensitive to the main clinical variables. PSA indicated that cladribine tablets were cost-effective in Iran, with a probability of 57.5% and 58.6% at lower and higher limits of threshold, respectively. Conclusion: Cladribine tablets yielded higher QALYs and lower costs compared to natalizumab, in patients with HDA-RMS in Iran.


2020 ◽  
Vol 9 (8) ◽  
pp. 553-562
Author(s):  
Hongfu Cai ◽  
Longfeng Zhang ◽  
Na Li ◽  
Bin Zheng ◽  
Maobai Liu

Aim: To investigate the cost–effectiveness of lenvatinib and sorafenib in the treatment of patients with nonresected hepatocellular carcinoma in China. Materials & methods: Markov model was used to simulate the direct medical cost and quality-adjusted life years (QALY) of patients with hepatocellular carcinoma. Clinical data were derived from the Phase 3 randomized clinical trial in a Chinese population. Results: Sorafenib treatment resulted in 1.794 QALYs at a cost of $43,780.73. Lenvatinib treatment resulted in 2.916 QALYs for patients weighing <60 and ≥60 kg at a cost of $57,049.43 and $75,900.36, The incremental cost–effectiveness ratio to the sorafenib treatment group was $11,825.94/QALY and $28,627.12/QALY, respectively. Conclusion: According to WHO’s triple GDP per capita, the use of lenvatinib by providing drugs is a cost-effective strategy.


2018 ◽  
Vol 41 (2) ◽  
pp. 391-398
Author(s):  
Monica Teng ◽  
Hui Jun Zhou ◽  
Liang Lin ◽  
Pang Hung Lim ◽  
Doreen Yeo ◽  
...  

Abstract Background The study evaluated the cost-effectiveness of hydrotherapy versus land-based therapy in patients with musculoskeletal disorders (MSDs) in Singapore. Methods A decision-analytic model was constructed to compare the cost-effectiveness of hydrotherapy to land-based therapy over 3 months from societal perspective. Target population comprised patients with low back pain (LBP), osteoarthritis (OA), rheumatoid arthritis (RA), total hip replacement (THR) and total knee replacement (TKR). Subgroup analyses were carried out to determine the cost-effectiveness of hydrotherapy in individual MSDs. Relative treatment effects were obtained through a systematic review of published data. Results Compared to land-based therapy, hydrotherapy was associated with an incremental cost-effectiveness ratio (ICER) of SGD 27 471 per quality-adjusted life-year (QALY) gained, which was below the willingness-to-pay threshold of SGD 70 000 per QALY (one gross domestic product per capita in Singapore in 2015). For the respective MSDs, hydrotherapy were dominant (more effective and less costly) in THR and TKR, cost-effective for LBP and RA, and not cost-effective for OA. Treatment adherence and cost of hydrotherapy were key drivers to the ICER values. Conclusions Hydrotherapy was a cost-effective rehabilitation compared to land-based therapy for a population with MSDs in Singapore. However, the benefit of hydrotherapy was not observed in patients with OA.


Author(s):  
Annelies Boonen

Consideration of costs and budgets plays an increasingly important role in decisions on access to innovative technologies. When clinicians want to influence such decisions, it is essential to understand the information on the burden of the disease and the evidence on cost-effectiveness of technologies. This chapter provides guidance to understanding the key methodological principles of economic evaluations, and describes available evidence on these issues in axial spondyloarthritis (axSpA). In the prebiologics era, the cost-of-illness for society of ankylosing spondylitis was slightly lower than for rheumatoid arthritis, and substantially lower than chronic low back pain. Cost of sick leave and work disability accounted for up to 75% of total cost-of-illness. Treatment with biologics increased cost-of-illness substantially, but the important gain in quality-adjusted life years resulted in acceptable cost-effectiveness in patients with active disease. There remains a gap in knowledge about the cost-effectiveness of diagnosing and treating axSpA earlier.


2014 ◽  
Vol 36 (5) ◽  
pp. E3 ◽  
Author(s):  
Ian McCarthy ◽  
Michael O'Brien ◽  
Christopher Ames ◽  
Chessie Robinson ◽  
Thomas Errico ◽  
...  

Object Incremental cost-effectiveness analysis is critical to the efficient allocation of health care resources; however, the incremental cost-effectiveness ratio (ICER) of surgical versus nonsurgical treatment for adult spinal deformity (ASD) has eluded the literature, due in part to inherent empirical difficulties when comparing surgical and nonsurgical patients. Using observed preoperative health-related quality of life (HRQOL) for patients who later underwent surgery, this study builds a statistical model to predict hypothetical quality-adjusted life years (QALYs) without surgical treatment. The analysis compares predicted QALYs to observed postoperative QALYs and forms the resulting ICER. Methods This was a single-center (Baylor Scoliosis Center) retrospective analysis of consecutive patients undergoing primary surgery for ASD. Total costs (expressed in 2010 dollars) incurred by the hospital for each episode of surgical care were collected from administrative data and QALYs were calculated from the 6-dimensional Short-Form Health Survey, each discounted at 3.5% per year. Regression analysis was used to predict hypothetical QALYs without surgery based on preoperative longitudinal data for 124 crossover surgical patients with similar diagnoses, baseline HRQOL, age, and sex compared with the surgical cohort. Results were projected through 10-year follow-up, and the cost-effectiveness acceptability curve (CEAC) was estimated using nonparametric bootstrap methods. Results Three-year follow-up was available for 120 (66%) of 181 eligible patients, who were predominantly female (89%) with average age of 50. With discounting, total costs averaged $125,407, including readmissions, with average QALYs of 1.93 at 3-year follow-up. Average QALYs without surgery were predicted to be 1.6 after 3 years. At 3- and 5-year follow-up, the ICER was $375,000 and $198,000, respectively. Projecting through 10-year follow-up, the ICER was $80,000. The 10-year CEAC revealed a 40% probability that the ICER was $80,000 or less, a 90% probability that the ICER was $90,000 or less, and a 100% probability that the ICER was less than $100,000. Conclusions Based on the WHO's suggested upper threshold for cost-effectiveness (3 times per capita GDP, or $140,000 in 2010 dollars), the analysis reveals that surgical treatment for ASD is cost-effective after a 10-year period based on predicted deterioration in HRQOL without surgery. The ICER well exceeds the WHO threshold at earlier follow-up intervals, highlighting the importance of the durability of surgical treatment in assessing the value of surgical intervention. Due to the study's methodology, the results are dependent on the predicted deterioration in HRQOL without surgery. As such, the results may not extend to patients whose HRQOL would remain steady without surgery. Future research should therefore pursue a direct comparison of QALYs for surgical and nonsurgical patients to better understand the cost-effectiveness of surgery for the average ASD patient.


2008 ◽  
Vol 15 (3) ◽  
pp. 159-165 ◽  
Author(s):  
MCY Tan ◽  
NT Ayas ◽  
A Mulgrew ◽  
L Cortes ◽  
JM FitzGerald ◽  
...  

BACKGROUND: Obstructive sleep apnea-hypopnea (OSAH) is a common disorder characterized by recurrent collapse of the upper airway during sleep. Patients experience a reduced quality of life and an increased risk of motor vehicle crashes (MVCs). Continuous positive airway pressure (CPAP), which is the first-line therapy for OSAH, improves sleepiness, vigilance and quality of life.OBJECTIVE: To assess the cost-effectiveness of CPAP therapy versus no treatment for OSAH patients who are drivers.METHODS: A Markov decision analytical model with a five-year time horizon was used. The study population consisted of male and female patients, between 30 and 59 years of age, who were newly diagnosed with moderate to severe OSAH. The model evaluated the cost-effectiveness of CPAP therapy in reducing rates of MVCs and improving quality of life. Utility values were obtained from previously published studies. Rates of MVCs under the CPAP and no CPAP scenarios were calculated from Insurance Corporation of British Columbia data and a systematic review of published studies. MVCs, equipment and physician costs were obtained from the British Columbia Medical Association, published cost-of-illness studies and the price lists of established vendors of CPAP equipment in British Columbia. Findings were examined from the perspectives of a third-party payer and society.RESULTS: From the third-party payer perspective, CPAP therapy was more effective but more costly than no CPAP (incremental cost-effectiveness ratio [ICER] of $3,626 per quality-adjusted life year). From the societal perspective, the ICER was similar ($2,979 per quality-adjusted life year). The ICER was most dependent on preference elicitation method used to obtain utility values, varying almost sixfold under alternative assumptions from the base-case analysis.CONCLUSION: After considering costs and impact on quality of life, as well as the risk of MVCs in individuals with OSAH, CPAP therapy for OSAH patients is a highly efficient use of health care resources. Provincial governments who do not provide funding for CPAP therapy should reconsider.


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