HARmonized Protocol Template to Enhance Reproducibility of Hypothesis Evaluating Real-World Evidence Studies on Treatment Effects: A Good Practices Report of a Joint ISPE/ISPOR Task Force

作者：Shirley Wang, Anton Pottegård, William H. Crown, Peter Arlett, Darren M. Ashcroft, Eric I. Benchimol, Marc L. Berger, Gracy Crane, Wim Goettsch, Hua Wei, Shaum Kabadi, David M. Kern, Xavier Kurz, Sinéad Langan, Takahiro Nonaka, Lucinda Orsini, Susana Perez‐Gutthann, Simone Pinheiro, Nicole Pratt, Sebastian Schneeweiß, Massoud Toussi, Rebecca Williams · 发表于：Value in Health · 年份：2022 · DOI：10.1016/j.jval.2022.09.001 · 被引用次数：50 · 研究领域：Meta-analysis and systematic reviews、Advanced Causal Inference Techniques、Health Policy Implementation Science

OBJECTIVES: Ambiguity in communication of key study parameters limits the utility of real-world evidence (RWE) studies in healthcare decision-making. Clear communication about data provenance, design, analysis, and implementation is needed. This would facilitate reproducibility, replication in independent data, and assessment of potential sources of bias. METHODS: The International Society for Pharmacoepidemiology (ISPE) and ISPOR-The Professional Society for Health Economics and Outcomes Research (ISPOR) convened a joint task force, including representation from key international stakeholders, to create a harmonized protocol template for RWE studies that evaluate a treatment effect and are intended to inform decision-making. The template builds on existing efforts to improve transparency and incorporates recent insights regarding the level of detail needed to enable RWE study reproducibility. The over-arching principle was to reach for sufficient clarity regarding data, design, analysis, and implementation to achieve 3 main goals. One, to help investigators thoroughly consider, then document their choices and rationale for key study parameters that define the causal question (e.g., target estimand), two, to facilitate decision-making by enabling reviewers to readily assess potential for biases related to these choices, and three, to facilitate reproducibility. STRATEGIES TO DISSEMINATE AND FACILITATE USE: Recognizing that the impact of this harmonized template relies on upta...