Pros and Cons of Event Based Modelling in Economic Evaluation
Notice bibliographique
Résumé
Background: Event based models are driven by the occurrence of clinical events such as primary clinical endpoints in randomised trials or adverse events associated with treatment. Statistical analyses of individual-patient data are used to determine event rates and further statistical analyses are performed to estimate survival, costs and health-related quality of life conditional on an event having occurred. For economic evaluations of health-care programmes such an approach has several advantages as extrapolation is facilitated, it is possible to explore cost-effectiveness in different risk groups and it makes it possible to bring in relevant external evidence such as pooled treatment effect. However, event based modelling also poses methodological challenges concerning not only technical issues but also conceptual ones regarding the scientific method. The aim of this paper is to explore and discuss these methodological challenges. Methods: Published event based models were reviewed and examples from a recently developed event based model in acute coronary syndrome were used to discuss and exemplify several of the methodological issues involving event based modelling. Results: The event based modelling approach normally uses randomised evidence to determine rates of clinical events, but given that an event has occurred, life expectancy, costs and health-related quality of life are estimated conditional on the event rather than randomised treatment. Some would argue this is appropriate as treatment only affect costs and quality of life through the impact of events rates. However, others would argue that such an approach is inappropriate as it adds 'structure' to the randomised evidence in terms of costs, life-expectancy and quality of life assuming conditional independence. Regarding more technical aspects, several methodological issues need to be considered as relatively advanced statistical models are combined in a decision-analytic framework to determine cost-effectiveness. The most important advantage of event based modelling identified in this work is that it provides a tool to estimate cost-effectiveness in a way relevant for policy, i.e. enabling the estimation of lifetime costs and health outcomes in different subgroups utilising all relevant evidence. Some of the challenges identified in this work include the choice of covariates to be included in the statistical analyses and the presentation of the results and probabilistic sensitivity analyses as much of the inputs into the cost-effectiveness model will be based on risk equations which can potentially define a very large number of subgroups. Conclusion: Although there are still methodological issues that need addressing in this framework, event based modelling is a useful method for providing relevant cost-effectiveness evidence.
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Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,173 | 0,310 |
| Méta-épidémiologie (sens strict) | 0,003 | 0,002 |
| Méta-épidémiologie (sens large) | 0,005 | 0,007 |
| Bibliométrie | 0,005 | 0,009 |
| Études des sciences et des technologies | 0,001 | 0,007 |
| Communication savante | 0,011 | 0,013 |
| Science ouverte | 0,006 | 0,007 |
| Intégrité de la recherche | 0,009 | 0,010 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,007 | 0,001 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».