A Structured Preapproval and Postapproval Comparative Study Design Framework to Generate Valid and Transparent Real‐World Evidence for Regulatory Decisions

Real‐world evidence provides important information about the effects of medicines in routine clinical practice. To engender trust that evidence generated for regulatory purposes is sufficiently valid, transparency in the reasoning that underlies study design decisions is critical. Building on existi...

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Bibliographic Details
Published in:Clinical pharmacology and therapeutics 2019-07, Vol.106 (1), p.103-115
Main Authors: Gatto, Nicolle M., Reynolds, Robert F., Campbell, Ulka B.
Format: Article
Language:English
Subjects:
Causality
Comparative Effectiveness Research - methods
Comparative Effectiveness Research - standards
Confounding Factors, Epidemiologic
Decision Making
Humans
Product Surveillance, Postmarketing - methods
Product Surveillance, Postmarketing - standards
Randomized Controlled Trials as Topic - methods
Randomized Controlled Trials as Topic - standards
Reproducibility of Results
Research Design
Reviews
State of the Art
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Summary:Real‐world evidence provides important information about the effects of medicines in routine clinical practice. To engender trust that evidence generated for regulatory purposes is sufficiently valid, transparency in the reasoning that underlies study design decisions is critical. Building on existing guidance and frameworks, we developed the Structured Preapproval and Postapproval Comparative study design framework to generate valid and transparent real‐world Evidence (SPACE) as a process for identifying design elements and minimal criteria for feasibility and validity concerns, and for documenting decisions. Starting with an articulated research question, we identify key components of the randomized controlled trial needed to maximize validity, and pragmatic choices are considered when required. A causal diagram is used to justify the variables identified for confounding control, and key decisions, assumptions, and evidence are captured in a structured way. In this way, SPACE may improve dialogue and build trust among healthcare providers, patients, regulators, and researchers.
ISSN:0009-9236
1532-6535
DOI:10.1002/cpt.1480