Acknowledging Patient Heterogeneity in Economic Evaluation

Grutters, Janneke P.C.; Sculpher, Mark; Briggs, Andrew; Severens, Johan L.; Candel, Math J. J. M.; Stahl, James E.; Ruysscher, Dirk De; Boer, Albert; Ramaekers, Bram; Joore, Manuela A.

doi:10.1007/s40273-012-0015-4

Cited by 57 publications

(75 citation statements)

References 46 publications

(119 reference statements)

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“…Ignoring heterogeneity effects in parameters may influence the results of an economic evaluation. 30 Only two (6,12) reported half-cycle corrections to account for transition events occurring halfway through the cycle. Not incorporating half-cycle corrections in states may lead to overestimation or underestimation of economic evaluation outcomes.…”

Section: Discussionmentioning

confidence: 99%

Economic evaluations on centralisation of specialised healthcare services: a systematic review of methods

et al. 2016

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ObjectiveTo systematically review and appraise the quality of economic evaluations assessing centralisation of specialised healthcare services.MethodsA systematic review to identify economic evaluations on centralisation of any specialised healthcare service. Full economic evaluations comparing costs and consequences of centralisation of any specialised healthcare service were eligible for inclusion. Methodological characteristics of included studies were appraised using checklists adapted from recommended guidelines.ResultsA total of 64 full-text articles met the inclusion criteria. Two studies were conducted in the UK. Most of the studies used volume of activity as a proxy measure of centralisation. The methods used to assess centralisation were heterogeneous. Studies differed in terms of study design used and aspect of centralisation they considered. There were major limitations in studies. Only 12 studies reported the study perspective. Charges which are not true representation of costs were used by 17 studies to assess cost outcomes. Only 10 reported the detailed breakdown of the cost components used in their analysis. Discounting was necessary in 14 studies but was reported only in 7 studies. Sensitivity analyses were included by less than one-third of the studies. The applicability of the identified studies to a setting other than the one they were conducted in is questionable, given variations in the organisation of services and healthcare costs. Centralisation as a concept has also been variably and narrowly defined as activity of specific services which may not reflect the wider aspects of centralisation.ConclusionsConfounded and biased information coming from studies without standardised methods may mislead decision-makers towards making wrong decisions on centralisation. It is important to improve the methodology and reporting of economic evaluations in order to provide more robust and transferable evidence. Wider aspects of healthcare centralisation should be considered in the estimates of costs and health outcomes.

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Section: Discussionmentioning

confidence: 99%

Economic evaluations on centralisation of specialised healthcare services: a systematic review of methods

et al. 2016

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“…Regulatory authorities, healthcare organizations and clinical decision-makers should be careful about whom they assign a certain treatment and what that treatment means to that patient population. Patient heterogeneity can be described by differentiating characteristics, such as demographics, preferences and clinical characteristics [31,34,36]. HTA should give consideration to heterogeneous patient groups based on their preferences.…”

Section: Patient Heterogeneitymentioning

confidence: 99%

“…For collecting information about heterogeneity, Grutters et al [36] require policy-makers to motivate both the transparency of data and the acknowledgement of patient heterogeneity as a standard practice in economic evaluations. A problem of assessing heterogeneity is that patient heterogeneity might be neglected; for example, assessing whether excluding subgroups from specified treatment is considered unethical or controversial [31,36].…”

Section: Expert Commentarymentioning

confidence: 99%

“…A problem of assessing heterogeneity is that patient heterogeneity might be neglected; for example, assessing whether excluding subgroups from specified treatment is considered unethical or controversial [31,36]. Another problem may result from too many characteristics that might potentially influence treatment effects, leading to too many subgroups.…”

Section: Expert Commentarymentioning

confidence: 99%

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Patient-centric HTA: different strokes for different folks

Mühlbacher¹

2015

Expert Review of Pharmacoeconomics & Outcomes Research

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Patient centricity will initiate a change in healthcare decision-making paradigms. Patient-centered outcomes are a part of the solution to the challenges posed by complex decision problems due to multiple clinical and non-clinical outcomes. Multiple decision criteria require that clinical and regulatory decisions be based on the implicit value judgments of experts. Experts are under continuous pressure to ensure decisions are aligned with patient needs and result in positive performance. To fulfill this role and guarantee efficient decisions, experts must be fully informed about patient preferences; however, this assumption might not necessarily reflect reality. Health technology assessment focuses on the measurement of clinical effects, rather than the assessment of the impact of different outcomes on users' perceived value. Further evidence is needed to ensure that expert judgments are congruent with patient preferences. Therefore, patient preferences can be a valuable source of information to inform clinical and regulatory decision-makers.

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“…Structural uncertainty refers to uncertainty surrounding the structure of a decision model. Variability relates to the fact that individuals are unique and therefore vary in their outcomes, which may partly be explained by individual characteristics (heterogeneity) [9].…”

Section: Defining Uncertaintymentioning

confidence: 99%