Development of a Canadian Guidance for reporting real-world evidence for regulatory and health-technology assessment (HTA) decision-making

Mina Tadrous; Theresa Aves; Christine Fahim; Jessica Riad; Nicole Mittmann; Daniel Prieto-Alhambra; Donna R Rivera; Kelvin Chan; Lisa M Lix; Seamus Kent; Dalia Dawoud; Jason Robert Guertin; James Ted McDonald; Jeff Round; Scott Klarenbach; Sanja Stanojevic; Mary A De Vera; Erin Strumpf; Robert W Platt; Farah Husein; Laurie Lambert; Kaleen N Hayes

doi:10.1016/j.jclinepi.2024.111545

Development of a Canadian Guidance for reporting real-world evidence for regulatory and health-technology assessment (HTA) decision-making

J Clin Epidemiol. 2024 Dec:176:111545. doi: 10.1016/j.jclinepi.2024.111545. Epub 2024 Sep 23.

Authors

Mina Tadrous¹, Theresa Aves², Christine Fahim³, Jessica Riad⁴, Nicole Mittmann⁵, Daniel Prieto-Alhambra⁶, Donna R Rivera⁷, Kelvin Chan⁸, Lisa M Lix⁹, Seamus Kent¹⁰, Dalia Dawoud¹¹, Jason Robert Guertin¹², James Ted McDonald¹³, Jeff Round¹⁴, Scott Klarenbach¹⁵, Sanja Stanojevic¹⁶, Mary A De Vera¹⁷, Erin Strumpf¹⁸, Robert W Platt¹⁸, Farah Husein¹⁹, Laurie Lambert¹⁹, Kaleen N Hayes²⁰

Affiliations

¹ Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada. Electronic address: [email protected].
² Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada.
³ Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada; Li Ka Shing Knowledge Institute, St. Michael's Hospital, Toronto, Canada.
⁴ Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada.
⁵ Sunnybrook Research Institute, Toronto, Canada; Canadian Agency for Drugs and Technologies in Health, Ottawa, Canada.
⁶ Department of Medical Informatics, Erasmus University Medical Center, Rotterdam, The Netherlands; Medical Informatics Department, Erasmus Universiteit Rotterdam, Rotterdam, The Netherlands.
⁷ Oncology Center of Excellence, U.S. Food and Drug Administration, Silver Spring, MD, USA.
⁸ Sunnybrook Research Institute, Toronto, Canada.
⁹ Department of Community Health Sciences, University of Manitoba, Winnipeg, Canada.
¹⁰ Erasmus School of Health Policy & Management, Erasmus University Rotterdam, The Netherlands.
¹¹ Science, Policy and Research Programme, National Institute for Health and Care Excellence, London, UK.
¹² Faculty of Medicine, Department of Social and Preventive Medicine, Université Laval, Quebec City, Quebec, Canada.
¹³ Department of Political Science, University of New Brunswick, Canada.
¹⁴ Institute of Health Economics, Edmonton, Alberta, Canada; Faculty of Medicine and Dentistry, Department of Pediatrics, University of Alberta, Edmonton, Alberta, Canada.
¹⁵ Faculty of Medicine and Dentistry, Department of Medicine, University of Alberta, Edmonton, Alberta, Canada.
¹⁶ Department of Community Health and Epidemiology, Dalhousie University, Halifax, Canada.
¹⁷ Faculty of Pharmaceutical Sciences, University of British Columbia, Vancouver, Canada.
¹⁸ Department of Epidemiology, Biostatistics and Occupational Health, McGill University, Montreal, Quebec, Canada.
¹⁹ Canadian Agency for Drugs and Technologies in Health, Ottawa, Canada.
²⁰ Department of Health Services, Policy, and Practice, Brown University School of Public Health, Providence, RI, USA.

PMID: 39322123
DOI: 10.1016/j.jclinepi.2024.111545

Abstract

Background and objective: Real-world evidence (RWE) can complement and fill knowledge gaps from randomized controlled trials to assist in health-technology assessment (HTA) for regulatory decision-making. However, the generation of RWE is an intricate process with many sequential decision points, and different methods and approaches may impact the quality and reliability of evidence. Standardization and transparency in reporting these decisions is imperative to appraise RWE and incorporate it into HTA decision-making. A partnership between Canadian health system stakeholders, namely, Health Canada and Canada's Drug Agency (formerly the Canadian Agency for Drugs and Technologies in Health), was established to develop guidance for the standardization of reporting of RWE for regulatory and HTA decision-making in Canada.

Study design and setting: A collaborative initiative to create structured guidance for RWE reporting in the context of regulatory and HTA decision-making.

Results: The developed guidance aims to standardize and ensure transparent reporting of RWE to improve its reliability and usefulness in regulatory and HTA processes.

Conclusion: This guidance can be adapted for other jurisdictions and will have future extensions to incorporate emerging issues with RWE and HTA decision-making.

Keywords: Epidemiologic research design; Guidelines; Health-technology assessment; Observational studies; Pharmacoepidemiology; Publishing standards; Real-world evidence; Regulators.

MeSH terms

Canada
Decision Making*
Evidence-Based Medicine / methods
Evidence-Based Medicine / standards
Guidelines as Topic
Humans
Reproducibility of Results
Technology Assessment, Biomedical* / methods
Technology Assessment, Biomedical* / standards