Reweighting Randomized Controlled Trial Evidence to Better Reflect Real Life - A Case Study of the Innovative Medicines Initiative
Open Access
- 1 October 2020
- journal article
- research article
- Published by Wiley in Clinical Pharmacology & Therapeutics
- Vol. 108 (4), 817-825
- https://doi.org/10.1002/cpt.1854
Abstract
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.Funding Information
- Eli Lilly and Company
- Seventh Framework Programme (FP7/2007‐2013)
This publication has 24 references indexed in Scilit:
- Influence of histology and biomarkers on first-line treatment of advanced non-small cell lung cancer in routine care setting: Baseline results of an observational study (FRAME)Lung Cancer, 2012
- Entropy Balancing for Causal Effects: A Multivariate Reweighting Method to Produce Balanced Samples in Observational StudiesPolitical Analysis, 2012
- The Use of Propensity Scores to Assess the Generalizability of Results from Randomized TrialsJournal of the Royal Statistical Society Series A: Statistics in Society, 2010
- Comparative Effectiveness Without Head-to-Head TrialsPharmacoEconomics, 2010
- Generalizing Evidence From Randomized Clinical Trials to Target Populations: The ACTG 320 TrialAmerican Journal of Epidemiology, 2010
- The calibration of treatment effects from clinical trials to target populationsClinical Trials, 2009
- Phase III Study Comparing Cisplatin Plus Gemcitabine With Cisplatin Plus Pemetrexed in Chemotherapy-Naive Patients With Advanced-Stage Non–Small-Cell Lung CancerJournal of Clinical Oncology, 2008
- Reconciling Decision Models With the Real WorldPharmacoEconomics, 1999
- The central role of the propensity score in observational studies for causal effectsBiometrika, 1983
- A Leisurely Look at the Bootstrap, the Jackknife, and Cross-ValidationThe American Statistician, 1983