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.

Autor: Wang SV; Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA., Pottegård A; University of Southern Denmark, Odense, Denmark., Crown W; Brandeis University, Waltham, Massachusetts, USA., Arlett P; European Medicines Agency, London, UK., Ashcroft DM; School of Health Sciences, University of Manchester, Manchester, UK., Benchimol EI; Child Health Evaluative Sciences, SickKids Research Institute, Toronto, Ontario, Canada.; ICES, Toronto, Ontario, Canada.; Department of Paediatrics and Institute of Health Policy, Management and Evaluation, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada., Berger ML; Independent Consultant, New York, USA., Crane G; Roche Pharmaceuticals, Camberley, UK., Goettsch W; The National Health Care Institute, Diemen, The Netherlands.; Utrecht University, Utrecht, The Netherlands., Hua W; US Food and Drug Administration, Silver Springs, Maryland, USA., Kabadi S; Sanofi-Aventis US LLC, North Potomac, Maryland, USA., Kern DM; Janssen Research & Development, LLC, Philadelphia, Pennsylvania, USA., Kurz X; European Medicines Agency, London, UK., Langan S; London School of Tropical Hygiene, London, UK., Nonaka T; Pharmaceutical and Medical Devices Agency, Japan., Orsini L; COMPASS Pathways Ltd, Skillman, UK., Perez-Gutthann S; RTI Health Solutions, Barcelona, Spain., Pinheiro S; US Food and Drug Administration, Silver Springs, Maryland, USA., Pratt N; Quality Use of Medicines and Pharmacy Research Centre, Clinical and Health Sciences, University of South Australia, South Australia, Australia., Schneeweiss S; Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA., Toussi M; IQVIA, France., Williams RJ; Bethesda, Maryland, USA.
Jazyk: angličtina
Zdroj: Pharmacoepidemiology and drug safety [Pharmacoepidemiol Drug Saf] 2023 Jan; Vol. 32 (1), pp. 44-55. Date of Electronic Publication: 2022 Oct 10.
DOI: 10.1002/pds.5507
Abstrakt: Problem: 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.
What We Did: 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 overarching 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 uptake, we have outlined a plan to introduce and pilot the template with key international stakeholders over the next 2 years.
Conclusion: The HARmonized Protocol Template to Enhance Reproducibility (HARPER) helps to create a shared understanding of intended scientific decisions through a common text, tabular and visual structure. The template provides a set of core recommendations for clear and reproducible RWE study protocols and is intended to be used as a backbone throughout the research process from developing a valid study protocol, to registration, through implementation and reporting on those implementation decisions.
(© 2022 The Authors. Pharmacoepidemiology and Drug Safety published by John Wiley & Sons Ltd.)
Databáze: MEDLINE
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