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Reweighting Randomized Controlled Trial Evidence to Better Reflect Real Life – A Case Study of the Innovative Medicines Initiative

Evidence from randomized controlled trials available for timely health technology assessments of new pharmacological treatments and regulatory decision making may not be generalizable to local patient populations, often resulting in decisions being made under uncertainty. In recent years, several re...

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Detalles Bibliográficos
Autores principales: Happich, Michael, Brnabic, Alan, Faries, Douglas, Abrams, Keith, Winfree, Katherine B., Girvan, Allicia, Jonsson, Pall, Johnston, Joseph, Belger, Mark
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7540324/
https://www.ncbi.nlm.nih.gov/pubmed/32301116
http://dx.doi.org/10.1002/cpt.1854
Descripción
Sumario:Evidence from randomized controlled trials available for timely health technology assessments of new pharmacological treatments and regulatory decision making may not be generalizable to local patient populations, often resulting in decisions being made under uncertainty. In recent years, several reweighting approaches have been explored to address this important question of generalizability to a target population. We present a case study of the Innovative Medicines Initiative to illustrate the inverse propensity score reweighting methodology, which may allow us to estimate the expected treatment benefit if a clinical trial had been run in a broader real‐world target population. We learned that identifying treatment effect modifiers, understanding and managing differences between patient characteristic data sets, and balancing the closeness of trial and target patient populations with effective sample size are key to successfully using this methodology and potentially mitigating some of this uncertainty around local decision making.