Good Practices for Real-World Data Studies of Treatment Effectiveness
These guidelines provide recommendations for conducting high-quality real-world data studies that assess how well treatments work in everyday clinical practice. They focus on improving trust in results by promoting transparency, study registration, and involvement of key stakeholders.
At a glance
Use when
Designing or evaluating real-world evidence studies on treatment effectiveness using observational data
Avoid when
Conducting early exploratory analyses without a predefined hypothesis or when only descriptive results are needed
Inputs
Real-world data (e.g., electronic health records, claims data, registries), research question, study protocol
Outputs
Transparent, reproducible, and stakeholder-informed real-world evidence on treatment effectiveness
How it works
Developed by a joint ISPOR-ISPE task force, these guidelines outline good procedural practices for real-world evidence (RWE) studies evaluating treatment and/or comparative effectiveness. Key areas include study registration, replicability, transparency, stakeholder engagement, and alignment with decision-making needs. The recommendations complement existing methodological standards and aim to increase confidence in RWE derived from observational datasets such as registries and electronic health records.
- HTA domains
- Clinical Effectiveness, Organisational aspects, Patient and Social Aspects
- Assumptions
- Real-world evidence can complement randomized trial data when generated using rigorous and transparent procedures; stakeholder trust increases with procedural transparency and study registration
- Strengths
- Enhances credibility and transparency of real-world studies; developed through consensus of international experts; supports decision-making in healthcare; promotes replicability and stakeholder engagement
- Limitations
- Adoption depends on incentives from regulators, funders, and journals; some recommendations may be challenging to implement in resource-limited settings
- Also known as
- ISPOR-ISPE Good Practices for RWD Studies, Good Practices for RWE Studies, Joint ISPOR-ISPE Task Force Recommendations on RWE
Questions this answers
- › How can real-world data studies be made more transparent and trustworthy?
- › What procedural steps improve the credibility of treatment effectiveness findings from observational data?
- › How should stakeholders be involved in real-world evidence research?
- › Should real-world studies be registered, and why?
- › How can real-world evidence studies be made replicable?
- › What role does peer review and consensus-building play in developing best practices for RWE?
References & sources
Related methods
- RelatedRecommendations on the use of NRs
- Used withGetReal Trial Tool
- RelatedRecommendations on statistical approaches for pragmatic trials
- RequiresTarget trial emulation demo
- RequiresCohortMethod R package
- RelatedHARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real‐world evidence studies
- RelatedISPOR Good Research Practices for Retrospective Database Analysis Checklist (Part I)
- RelatedGuidelines for Good Database Selection and Use in Pharmacoepidemiology Research
- Used withSTaRT-RWE
- RequiresFOUNTAIN
Similar by meaning
- ISPOR Prospective Observational Studies to Assess Comparative Effectiveness Good Research Practices Task Force Report
- CADTH Guidance for Reporting Real-World Evidence
- Regulatory and HTA Considerations for RWD-Derived External Controls
- REPEAT Initiative (Reproducible Evidence: Practices to Enhance and Achieve Transparency)
Beta record. Generated from the primary source via AI extraction and independent audit, pending final human review.