ArticleClinical pharmacology and therapeutics2019
A Structured Preapproval and Postapproval Comparative Study Design Framework to Generate Valid and Transparent Real-World Evidence for Regulatory Decisions.
Article in Clinical pharmacology and therapeutics, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
28 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- A systematic review of real-world evidence (RWE) supportive of new drug and biologic license application approvals in rare diseases.Orphanet journal of rare diseases · 2024Pooled it
- The Role of Real-World Evidence in FDA-Approved New Drug and Biologics License Applications.Clinical pharmacology and therapeutics · 2022Pooled it
- Beyond Missingness: Systematizing Methods for Comprehensive Data Fitness Assessment in Clinical Research.Journal of medical Internet research · 2026Article
- Evaluating Data Quality by Proxy: Can We Evaluate All Dimensions of the European Medicines Agency Data Quality Framework for Registry-Based Post-Authorization Safety Studies?Pharmacoepidemiology and drug safety · 2026Article
- Illustrating an Adaptive Prespecification Framework for Observational Research: Target Trial Emulations Comparing Immunomodulator Treatments for COVID-19.Epidemiology (Cambridge, Mass.) · 2025Article
- Navigating the Real World: A Scoping Review of Structured Frameworks to Effectively Identify, Evaluate, and Select Real-World Data Sources for Fit-for-Purpose Studies.Clinical pharmacology and therapeutics · 2025Article
- Optimizing Patient Registries for Regulatory Decision Making - Key Learnings From an HMA/EMA Multistakeholder Workshop.Clinical pharmacology and therapeutics · 2025Review
- Increasing the Utility of Real-World Data to Inform Public Health Decision Making Through a US-based Private-Public Partnership: 10 Lessons Learned from a Principled Approach to Rapid Pandemic RWE Generation.Therapeutic innovation & regulatory science · 2025Article
- Advancing Principled Pharmacoepidemiologic Research to Support Regulatory and Healthcare Decision Making: The Era of Real-World Evidence.Clinical pharmacology and therapeutics · 2025Review
- Transparency in the secondary use of health data: assessing the status quo of guidance and best practices.Royal Society open science · 2025Article
- A Standardized Guideline for Assessing Extracted Electronic Health Records Cohorts: A Scoping Review.AMIA Joint Summits on Translational Science proceedings. AMIA Joint Summits on Translational Science · 2025Article
- FOUNTAIN: a modular research platform for integrated real-world evidence generation.BMC medical research methodology · 2024Article
- Calibrating Observational Health Record Data Against a Randomized Trial.JAMA network open · 2024Article
- Navigating the unknown: how to best 'reflect' standard of care in indications without a dedicated treatment pathway in health technology assessment submissions.Journal of comparative effectiveness research · 2024Review
- Antipsychotics and Risks of Cardiovascular and Cerebrovascular Diseases and Mortality in Dwelling Community Older Adults.Pharmaceuticals (Basel, Switzerland) · 2024Article
- Important tool in our rare disease toolbox: hybrid retrospective-prospective natural history studies serve well as external comparators for rare disease studies.Frontiers in drug safety and regulation · 2024Review
- Design, implementation, and inferential issues associated with clinical trials that rely on data in electronic medical records: a narrative review.BMC medical research methodology · 2023Review
- Worldwide Trends in Registering Real-World Studies at ClinicalTrials.gov: A Cross-Sectional Analysis.International journal of general medicine · 2023Article
- A causal roadmap for generating high-quality real-world evidence.Journal of clinical and translational science · 2023Review
- Observational
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
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.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.