scholarly journals Time-to-event analysis in economic evaluations: a comparison of modelling methods to assess the cost-effectiveness of transplanting a marginal quality kidney

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Sameera Senanayake ◽  
Nicholas Graves ◽  
Helen Healy ◽  
Keshwar Baboolal ◽  
Adrian Barnett ◽  
...  

Abstract Background Economic-evaluations using decision analytic models such as Markov-models (MM), and discrete-event-simulations (DES) are high value adds in allocating resources. The choice of modelling method is critical because an inappropriate model yields results that could lead to flawed decision making. The aim of this study was to compare cost-effectiveness when MM and DES were used to model results of transplanting a lower-quality kidney versus remaining waitlisted for a kidney. Methods Cost-effectiveness was assessed using MM and DES. We used parametric survival models to estimate the time-dependent transition probabilities of MM and distribution of time-to-event in DES. MMs were simulated in 12 and 6 monthly cycles, out to five and 20-year time horizon. Results DES model output had a close fit to the actual data. Irrespective of the modelling method, the cycle length of MM or the time horizon, transplanting a low-quality kidney as compared to remaining waitlisted was the dominant strategy. However, there were discrepancies in costs, effectiveness and net monetary benefit (NMB) among different modelling methods. The incremental NMB of the MM in the 6-months cycle lengths was a closer fit to the incremental NMB of the DES. The gap in the fit of the two cycle lengths to DES output reduced as the time horizon increased. Conclusion Different modelling methods were unlikely to influence the decision to accept a lower quality kidney transplant or remain waitlisted on dialysis. Both models produced similar results when time-dependant transition probabilities are used, most notable with shorter cycle lengths and longer time-horizons.

2020 ◽  
Vol 14 (8) ◽  
Author(s):  
Anna Parackal ◽  
Jean-Eric Tarride ◽  
Feng Xie ◽  
Gord Blackhouse ◽  
Jennifer Hoogenes ◽  
...  

Introduction: Recent health technology assessments (HTAs) of robot-assisted radical prostatectomy (RARP) in Ontario and Alberta, Canada, resulted in opposite recommendations, calling into question whether benefits of RARP offset the upfront investment. Therefore, the study objectives were to conduct a cost-utility analysis from a Canadian public payer perspective to determine the cost-effectiveness of RARP. Methods: Using a 10-year time horizon, a five-state Markov model was developed to compare RARP to open radical prostatectomy (ORP). Clinical parameters were derived from Canadian observational studies and a recently published systematic review. Costs, resource utilization, and utility values from recent Canadian sources were used to populate the model. Results were presented in terms of increment costs per quality-adjusted life years (QALYs) gained. A probabilistic analysis was conducted, and uncertainty was represented using cost-effectiveness acceptability curves (CEACs). One-way sensitivity analyses were also conducted. Future costs and QALYs were discounted at 1.5%. Results: Total cost of RARP and ORP were $47 033 and $45 332, respectively. Total estimated QALYs were 7.2047 and 7.1385 for RARP and ORP, respectively. The estimated incremental cost-utility ratio (ICUR) was $25 704 in the base-case analysis. At a willingness-to-pay threshold of $50 000 and $100 000 per QALY gained, the probability of RARP being cost-effective was 0.65 and 0.85, respectively. The model was most sensitive to the time horizon. Conclusions: The results of this analysis suggest that RARP is likely to be cost-effective in this Canadian patient population. The results are consistent with Alberta’s HTA recommendation and other economic evaluations, but challenges Ontario’s reimbursement decision.


2021 ◽  
Vol 9 (6) ◽  
Author(s):  
Liudan Tu ◽  
Ya Xie ◽  
Jieruo Gu

This review was aimed to evaluate health economic models used in evaluations of different treatment strategies in spondyloarthritis (SpA). Model-based health economic evaluation studies are increasing and complex models with short-term and long-term horizon are applied to investigate the cost-effectiveness of SpA treatments. The objective of this study was to carry out a systematic review of the evolution of health economic models used in the treatment of SpA. Electronic searches within MEDLINE and EMBASE were carried out using a predefined search strategy. Inclusion and exclusion criteria were used to select relevant studies. Data on country, intervention, evaluation perspective, type of model, time horizon, types of costs and effectiveness measurement were extracted. Eighteen models were described in 22 publications, of which 81.8% were European. Study perspectives included the societal (n=6), healthcare system and payer (n=14), or patient and government (n=1). Time horizon ranged from 52 weeks to lifetime. Markov model was the most frequently used model, only one individual patient simulation models accounting for uncertainty in multiple parameters was reported. Most studies compared different biologics (including different TNFi/biosimilar and IL-17A antibody) with conventional care (NSAIDs) because of the high prize. Only half of studies took indirect costs into account. Modeling is of importance in health economic evaluations of SpA treatment. Long-term costs especially indirect costs should be considered when comparing different treatment alternatives in order to provide more information for policy makers and clinicians.


2021 ◽  
pp. 096228022199750
Author(s):  
Zvifadzo Matsena Zingoni ◽  
Tobias F Chirwa ◽  
Jim Todd ◽  
Eustasius Musenge

There are numerous fields of science in which multistate models are used, including biomedical research and health economics. In biomedical studies, these stochastic continuous-time models are used to describe the time-to-event life history of an individual through a flexible framework for longitudinal data. The multistate framework can describe more than one possible time-to-event outcome for a single individual. The standard estimation quantities in multistate models are transition probabilities and transition rates which can be mapped through the Kolmogorov-Chapman forward equations from the Bayesian estimation perspective. Most multistate models assume the Markov property and time homogeneity; however, if these assumptions are violated, an extension to non-Markovian and time-varying transition rates is possible. This manuscript extends reviews in various types of multistate models, assumptions, methods of estimation and data features compatible with fitting multistate models. We highlight the contrast between the frequentist (maximum likelihood estimation) and the Bayesian estimation approaches in the multistate modeling framework and point out where the latter is advantageous. A partially observed and aggregated dataset from the Zimbabwe national ART program was used to illustrate the use of Kolmogorov-Chapman forward equations. The transition rates from a three-stage reversible multistate model based on viral load measurements in WinBUGS were reported.


2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Qiaoping Xu ◽  
Li Yuanyuan ◽  
Zhu Jiejing ◽  
Liu Jian ◽  
Li Qingyu ◽  
...  

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.


Author(s):  
Nadine T. Hillock ◽  
Tracy L. Merlin ◽  
Jonathan Karnon ◽  
John Turnidge ◽  
Jaklin Eliott

Abstract Background The frameworks used by Health Technology Assessment (HTA) agencies for value assessment of medicines aim to optimize healthcare resource allocation. However, they may not be effective at capturing the value of antimicrobial drugs. Objectives To analyze stakeholder perceptions regarding how antimicrobials are assessed for value for reimbursement purposes and how the Australian HTA framework accommodates the unique attributes of antimicrobials in cost-effectiveness evaluation. Methods Eighteen individuals representing the pharmaceutical industry or policy-makers were interviewed. Interviews were transcribed verbatim, coded, and thematically analyzed. Results Key emergent themes were that reimbursement decision-making should consider the antibiotic spectrum when assessing value, risk of shortages, the impact of procurement processes on low-priced comparators, and the need for methodological transparency when antimicrobials are incorporated into the economic evaluation of other treatments. Conclusions Participants agreed that the current HTA framework for antimicrobial value assessment is inadequate to properly inform funding decisions, as the contemporary definition of cost-effectiveness fails to explicitly incorporate the risk of future resistance. Policy-makers were uncertain about how to incorporate future resistance into economic evaluations without a systematic method to capture costs avoided due to good stewardship. Lacking financial reward for the benefits of narrower-spectrum antimicrobials, companies will likely focus on developing broad-spectrum agents with wider potential use. The perceived risks of shortages have influenced the funding of generic antimicrobials in Australia, with policy-makers suggesting a willingness to pay more for assured supply. Although antibiotics often underpin the effectiveness of other medicines, it is unclear how this is incorporated into economic models.


2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Bethany E. Higgins ◽  
Giovanni Montesano ◽  
Alison M. Binns ◽  
David P. Crabb

AbstractIn age-related macular degeneration (AMD) research, dark adaptation has been found to be a promising functional measurement. In more severe cases of AMD, dark adaptation cannot always be recorded within a maximum allowed time for the test (~ 20–30 min). These data are recorded either as censored data-points (data capped at the maximum test time) or as an estimated recovery time based on the trend observed from the data recorded within the maximum recording time. Therefore, dark adaptation data can have unusual attributes that may not be handled by standard statistical techniques. Here we show time-to-event analysis is a more powerful method for analysis of rod-intercept time data in measuring dark adaptation. For example, at 80% power (at α = 0.05) sample sizes were estimated to be 20 and 61 with uncapped (uncensored) and capped (censored) data using a standard t-test; these values improved to 12 and 38 when using the proposed time-to-event analysis. Our method can accommodate both skewed data and censored data points and offers the advantage of significantly reducing sample sizes when planning studies where this functional test is an outcome measure. The latter is important because designing trials and studies more efficiently equates to newer treatments likely being examined more efficiently.


Author(s):  
Jin K. Kim ◽  
Mitchell Shiff ◽  
Michael E. Chua ◽  
Fadi Zu’bi ◽  
Jessica M. Ming ◽  
...  

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