Cost-effectiveness analyses using real-world data: an overview of the literature

Bowrin, Kevin; Brière, J; Lévy, Pierre; Millier, A.; Clay, E.; Toumi, Mondher

doi:10.1080/13696998.2019.1588737

Cited by 42 publications

(58 citation statements)

References 32 publications

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“…The use of RWE is associated to many challenges, such as dealing with the selection bias, missing data, lack of accuracy related to drug exposure and outcomes, errors during the record-keeping process [43], and also to potential benefits. For example, it would enable a shift from surrogate outcomes to clinical and long-term outcomes and constitute an important source of information in the specific case of rare harms.…”

Section: Discussionmentioning

confidence: 99%

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Real-world cost-effectiveness of rivaroxaban compared with vitamin K antagonists in the context of stroke prevention in atrial fibrillation in France

et al. 2020

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ObjectiveThe objective was to assess the real-world cost-effectiveness of rivaroxaban, versus vitamin K antagonists (VKAs), for stroke prevention in patients with atrial fibrillation (AF) from a French national health insurance perspective.Funding: LF, CC and PL received honorarium from Bayer AG for critical input into study design and results analysis. Bayer Plc. provided support in the form of salaries for author KB; Bayer AG provided support in the form of salaries for author JBB. Bayer AG sponsored the study, and Bayer AG as well as Bayer Plc had a role in the study design, ConclusionsAlthough there is no official willingness-to-pay threshold in France, these results suggest that rivaroxaban is likely to be cost-effective compared to VKA in French patients with AF from a national insurance perspective. Introduction/BackgroundAtrial fibrillation (AF) is a cardiac arrhythmia with structural and/or electrophysiological abnormalities that induce remodelling in the atria; it is the most common cardiac arrhythmia [1][2][3]. Worldwide, an estimated 3% of adults aged 20 years or older suffer from AF, approximately 20.9 million men and 12.6 million women [2]. Due to the aging of the population, the worldwide prevalence is predicted to at least double in many countries during the next several decades [4,5].AF is associated with substantial morbidity and mortality [2]. Compared to otherwise healthy individuals, men and women with AF are at a 1.5-fold and 2-fold increased risk for allcause mortality, respectively [2], and the risk for strokes increased by 2-to-7-fold [4]. Strokes are associated with significant financial burden [6]; in 2015, the total costs of stroke care in the European Union (EU) was estimated to be €45 billion euros [7]. It is expected that between 2015 and 2035, there will be a 34% increase in total number of stroke events in the European Union [7].Oral anticoagulants including vitamin K antagonists (VKAs) or non-VKA oral anticoagulants (NOACs) such as rivaroxaban, dabigatran, apixaban, and edoxaban, have been established as a cornerstone of management in patients with AF and to reduce stroke incidence and mortality [2] in randomized clinical trials (RCTs) [2].Many uncertainties remain regarding the relevance of the results of RCTs in a real-world setting. Real-world evidence (RWE) may provide additional information to decision-makers [8]. Indeed, RWE sample size is not limited as it is the case of RCTs. RCTs have to respect inclusion/exclusion criteria regarding population selection. Also, RWE can offer long-term outcomes while the timeframe of RCT is usually shorter with only a few outcomes [9].A meta-analysis comparing NOACs with VKAs and reporting effectiveness, safety, and persistence using RWE has recently been published [10]. It confirmed the findings of rivaroxaban pivotal RCT [11] and concluded that rivaroxaban is a suitable alternative to VKAs in routine clinical practice. Health Technology Assessment (HTA) agencies are frequently requesting manufacturers to prove the benefits of their hea...

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Section: Discussionmentioning

confidence: 99%

“…It is expected that methodologies used to analyse and synthesize RWE will continue to evolve. However, little guidance is available on the relative merits of using RWE in the modelling context [43][44][45].…”

Section: Discussionmentioning

confidence: 99%

Real-world cost-effectiveness of rivaroxaban compared with vitamin K antagonists in the context of stroke prevention in atrial fibrillation in France

et al. 2020

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“…However, as mentioned previously, these studies have some methodological limitations, such as confounding selection, identified by the authors of the studies in this review as the main potential risk. In another review [2], the main biases were confounding and missing data. A further risk in this type of information, frequent but not detailed, is registration quality.…”

Section: Discussionmentioning

confidence: 99%

“…Nevertheless, we did not use it as, although it was complementary, it did not substitute the EE checklist and had some limitations such as not covering some aspects of statistical analysis. Moreover, this checklist did not allow a description of which specific statistical method should be prescribed to approach selection bias in analysis of cost-effectiveness [2].…”

Section: Discussionmentioning

confidence: 99%

Economic Evaluations Informed Exclusively by Real World Data: A Systematic Review

Parody-Rúa

Rubio-Valera

Guevara-Cuéllar

et al. 2020

IJERPH

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Economic evaluations using Real World Data (RWD) has been increasing in the very recent years, however, this source of information has several advantages and limitations. The aim of this review was to assess the quality of full economic evaluations (EE) developed using RWD. A systematic review was carried out through articles from the following databases: PubMed, Embase, Web of Science and Centre for Reviews and Dissemination. Included were studies that employed RWD for both costs and effectiveness. Methodological quality of the studies was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. Of the 14,011 studies identified, 93 were included. Roughly half of the studies were carried out in a hospital setting. The most frequently assessed illnesses were neoplasms while the most evaluated interventions were pharmacological. The main source of costs and effects of RWD were information systems. The most frequent clinical outcome was survival. Some 47% of studies met at least 80% of CHEERS criteria. Studies were conducted with samples of 100–1000 patients or more, were randomized, and those that reported bias controls were those that fulfilled most CHEERS criteria. In conclusion, fewer than half the studies met 80% of the CHEERS checklist criteria.

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“…Inputs are based on data obtained from randomized controlled trials (RCTs) and do not take into account the use of DPP4Is in real-world clinical practice [12,13]. Real-world evidence from sources reflecting the budget holder's perspective, such as data from insurance claims databases, is now being recognized as a valuable resource for economic studies conducted later in product life cycles [14,15]. However, without proper adjustment, comparative observational studies using these databases to evaluate the impact of DPP4Is on the cost of T2D treatment are likely to be limited by channeling bias.…”

Section: Introductionmentioning

confidence: 99%

Cost of Managing Type 2 Diabetes Before and After Initiating Dipeptidyl Peptidase 4 Inhibitor Treatment: A Longitudinal Study Using a French Public Health Insurance Database

Émery

Torreton

Dejager³

et al. 2020

Diabetes Ther

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Introduction: Diabetes is a growing epidemic that imposes a substantial economic burden on healthcare systems. This study aimed to evaluate the cost of managing type 2 diabetes (T2D) with dipeptidyl peptidase 4 inhibitors (DPP4Is) using real-world data. Method: This longitudinal study used data from the French EGB (Echantillon Généraliste des Bénéficiaires) database. The annual average direct healthcare cost of treating patients with T2D was calculated 3 years prior and 3 years after initiation of DPP4I therapy. Actual total ambulatory and hospital care expenditure for the 3 years after DPP4I initiation was compared to projected costs. The distribution of costs across all care modalities was assessed over the 6-year period. Results: Ambulatory and hospital care expenditure data for 919 patients with T2D starting DPP4I therapy alone or in combination in 2013 were analyzed. A total of 526 patients (57.2%) were still being treated with DPP4I 3 years after DPP4I initiation. Regardless of the treatment regimen, the ambulatory and hospital care costs increased above projected costs in the first year following DPP4I initiation, and then declined during the second and third years to levels in line with or below projected values for patients using DPP4Is as an add-on therapy. The increase in total expenditure in the first year following DPP4I initiation and the subsequent decline in costs in the second and third years were both associated with general trends in consumption across all aspects of patient care. Conclusion: Despite an initial increase in healthcare expenditure, concomitant with reevaluation of patient care, this study showed that initiation of DPP4Is as an add-on therapy in French patients with T2D was associated with care expenditure that was in line or below predicted values within the 3 years following treatment initiation. Additional studies are required to evaluate the economic impact of the long-term treatment benefits.

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Cost-effectiveness analyses using real-world data: an overview of the literature

Cited by 42 publications

References 32 publications

Real-world cost-effectiveness of rivaroxaban compared with vitamin K antagonists in the context of stroke prevention in atrial fibrillation in France

Real-world cost-effectiveness of rivaroxaban compared with vitamin K antagonists in the context of stroke prevention in atrial fibrillation in France

Economic Evaluations Informed Exclusively by Real World Data: A Systematic Review

Cost of Managing Type 2 Diabetes Before and After Initiating Dipeptidyl Peptidase 4 Inhibitor Treatment: A Longitudinal Study Using a French Public Health Insurance Database

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