Suzanne M Cadarette1,2,3,4, Malcolm Maclure5, J A Chris Delaney6, Heather J Whitaker7,8, Kaleen N Hayes2, Shirley V Wang9, Mina Tadrous1,10, Joshua J Gagne9, Giulia P Consiglio1, Jesper Hallas11,12. 1. Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada. 2. Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada. 3. Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, North Carolina, USA. 4. WHO Collaborating Centre for Governance, Accountability and Transparency in the Pharmaceutical Sector, Toronto, Ontario, Canada. 5. Department of Anesthesiology, Pharmacology and Therapeutics, University of British Columbia, Vancouver, British Columbia, Canada. 6. College of Pharmacy, University of Manitoba, Winnipeg, Manitoba, Canada. 7. Department of Mathematic and Statistics, The Open University, Milton Keynes, UK. 8. Department of Statistics, Modelling and Economics, Public Health England, London, UK. 9. Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA. 10. Women's College Hospital, Toronto, Ontario, Canada. 11. Clinical Pharmacology and Pharmacy, IST, University of Southern Denmark, Odense, Denmark. 12. Department of Clinical Pharmacology and Biochemistry, Odense University Hospital, Odense, Denmark.
Abstract
PURPOSE: Consensus is needed on conceptual foundations, terminology and relationships among the various self-controlled "trigger" study designs that control for time-invariant confounding factors and target the association between transient exposures (potential triggers) and abrupt outcomes. The International Society for Pharmacoepidemiology (ISPE) funded a working group of ISPE members to develop guidance material for the application and reporting of self-controlled study designs, similar to Standards of Reporting Observational Epidemiology (STROBE). This first paper focuses on navigation between the types of self-controlled designs to permit a foundational understanding with guiding principles. METHODS: We leveraged a systematic review of applications of these designs, that we term Self-controlled Crossover Observational PharmacoEpidemiologic (SCOPE) studies. Starting from first principles and using case examples, we reviewed outcome-anchored (case-crossover [CCO], case-time control [CTC], case-case-time control [CCTC]) and exposure-anchored (self-controlled case-series [SCCS]) study designs. RESULTS: Key methodological features related to exposure, outcome and time-related concerns were clarified, and a common language and worksheet to facilitate the design of SCOPE studies is introduced. CONCLUSIONS: Consensus on conceptual foundations, terminology and relationships among SCOPE designs will facilitate understanding and critical appraisal of published studies, as well as help in the design, analysis and review of new SCOPE studies. This manuscript is endorsed by ISPE.
PURPOSE: Consensus is needed on conceptual foundations, terminology and relationships among the various self-controlled "trigger" study designs that control for time-invariant confounding factors and target the association between transient exposures (potential triggers) and abrupt outcomes. The International Society for Pharmacoepidemiology (ISPE) funded a working group of ISPE members to develop guidance material for the application and reporting of self-controlled study designs, similar to Standards of Reporting Observational Epidemiology (STROBE). This first paper focuses on navigation between the types of self-controlled designs to permit a foundational understanding with guiding principles. METHODS: We leveraged a systematic review of applications of these designs, that we term Self-controlled Crossover Observational PharmacoEpidemiologic (SCOPE) studies. Starting from first principles and using case examples, we reviewed outcome-anchored (case-crossover [CCO], case-time control [CTC], case-case-time control [CCTC]) and exposure-anchored (self-controlled case-series [SCCS]) study designs. RESULTS: Key methodological features related to exposure, outcome and time-related concerns were clarified, and a common language and worksheet to facilitate the design of SCOPE studies is introduced. CONCLUSIONS: Consensus on conceptual foundations, terminology and relationships among SCOPE designs will facilitate understanding and critical appraisal of published studies, as well as help in the design, analysis and review of new SCOPE studies. This manuscript is endorsed by ISPE.
Authors: Scott M Vouri; Earl J Morris; Silken A Usmani; Rachel Reise; Xinyi Jiang; Carl J Pepine; Todd M Manini; Daniel C Malone; Almut G Winterstein Journal: Pharmacoepidemiol Drug Saf Date: 2021-10-06 Impact factor: 2.890