External Controls to Study Treatment Effects in Rare Diseases: Challenges and Future Directions
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Summary
A mini‐review of frequently used approaches to external control arms used to supplement and contextualize efficacy and safety claims of single arm clinical trials for rare disease therapies highlights some of the most important criticisms and paths forward.
- Type
- review
- Published
- 2024-09-17
- Cited by
- 6
- References
- 26
- Access
- Open access
- OpenAlex
- https://openalex.org/W4402573202
- Semantic Scholar
- https://api.semanticscholar.org/CorpusID:272699645
Keywords
Intensive care medicine, External validity, Clinical trial, Medicine, Affect (linguistics)
References
- Specifying a target trial prevents immortal time bias and other self-inflicted injuries in observational analyses
- Identifying patients with high data completeness to improve validity of comparative effectiveness research in electronic health records data
- Using Super Learner Prediction Modeling to Improve High-dimensional Propensity Score Estimation
- Why Test for Proportional Hazards?
- A multi-stakeholder platform to prospectively link longitudinal real-world clinico-genomic, imaging, and outcomes data for patients with metastatic lung cancer.
- Approaches to Addressing Missing Values, Measurement Error and Confounding in Epidemiologic Studies.
- Penalized regression for left‐truncated and right‐censored survival data
- Augmenting control arms with real-world data for cancer trials: Hybrid control arm methods and considerations
- A meta‐analytic framework to adjust for bias in external control studies
- An Approach to Solving the Complex Clinicogenomic Data Landscape in Precision Oncology: Learnings From the Design of WAYFIND-R, a Global Precision Oncology Registry
- HARmonized Protocol Template to Enhance Reproducibility of Hypothesis Evaluating Real-World Evidence Studies on Treatment Effects: A Good Practices Report of a Joint ISPE/ISPOR Task Force.
- Target Trial Emulation: A Framework for Causal Inference From Observational Data.
- Effectively Leveraging RWD for External Controls: A Systematic Literature Review of Regulatory and HTA Decisions
- Emulation of Randomized Clinical Trials With Nonrandomized Database Analyses: Results of 32 Clinical Trials.
- Estimands in Real-World Evidence Studies
- Considerations for the design and conduct of externally controlled trials for drug and biological products
- Quantitative bias analysis for external control arms using real-world data in clinical trials: a primer for clinical researchers
- Design differences and variation in results between randomised trials and non-randomised emulations: meta-analysis of RCT-DUPLICATE data
- The Next Horizon of Drug Development: External Control Arms and Innovative Tools to Enrich Clinical Trial Data
- A systematic review of real-world evidence (RWE) supportive of new drug and biologic license application approvals in rare diseases
Cited by
- Challenges in estimating the counterfactual placebo HIV incidence rate from a registration cohort: The PrEPVacc trial
- PMDA Perspective on Use of Real‐World Data and Real‐World Evidence as an External Control: Recent Examples and Considerations
- Unlocking the Mysteries of Rare Disease Drug Development: A Beginner's Guide for Clinical Pharmacologists
- Re-envisioning Clinical Trial Design in Systemic Sclerosis
- Digital twins for accelerating drug discovery and development: opportunities and challenges.
- Therapeutic Orphan No More: Role for Clinical Pharmacology and Translational Science in Developing Therapeutics for Rare and Neglected Diseases
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