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Whole-of-Life Inclusion in Bayesian Adaptive Platform Clinical Trials
Anita J Campbell1,2,3, Keerthi Anpalagan2,3, Emma J Best4,5,6
1Department of Infectious Diseases, Perth Children's Hospital, Perth, Western Australia, Australia.
Insights
Clinical trial designs are evolving to include children, addressing evidence inequity. Bayesian adaptive designs, like the SNAP trial, enable simultaneous adult and pediatric enrollment, improving data and generalizability.
Area of Science:
- Clinical Trial Design
- Pediatric Research
- Bayesian Statistics
Background:
- A significant gap exists in evidence-based care for pediatric populations due to limited clinical trial inclusion.
- Traditional trial designs often exclude infants, children, and adolescents, leading to evidence inequity.
- The "therapeutic orphan" designation highlights the need for dedicated pediatric research.
Purpose of the Study:
- To evaluate three distinct trial design strategies for incorporating children into clinical trials: sequential, parallel, and unified adult-pediatric Bayesian adaptive designs.
- To demonstrate how these designs can address evidence inequity in pediatric populations.
- To showcase the Staphylococcus aureus Network Adaptive Platform (SNAP) trial as an example of a unified whole-of-life design.
Main Methods:
- Comparison of sequential, parallel, and unified adult-pediatric Bayesian adaptive trial designs.
- Description of Bayesian methods for data synthesis and analysis in clinical trials.
- The SNAP trial utilizes a Bayesian hierarchical model for information sharing between adult and pediatric groups.
Main Results:
- Bayesian hierarchical models offer enhanced precision for treatment efficacy and safety estimates in heterogeneous populations.
- Unified designs facilitate simultaneous enrollment of adults and children, improving trial inclusivity and diversity.
- The SNAP trial demonstrates a successful whole-of-life Bayesian adaptive design for comprehensive age group inclusion.
Conclusions:
- Bayesian adaptive designs, particularly unified whole-of-life approaches, are effective in including children in clinical trials.
- These methods promote a "no child left behind" vision, ensuring evidence generation across the life course.
- The SNAP trial exemplifies how innovative designs enhance generalizability and translation of findings into clinical practice.
Importance:
There is a recognized unmet need for clinical trials to provide evidence-informed care for infants, children and adolescents. This Special Communication outlines the capacity of 3 distinct trial design strategies, sequential, parallel, and a unified adult-pediatric bayesian adaptive design, to incorporate children into clinical trials and transform this current state of evidence inequity. A unified adult-pediatric whole-of-life clinical trial is demonstrated through the Staphylococcus aureus Network Adaptive Platform (SNAP) trial.
Observations:
Bayesian methods provide a framework for synthesizing data in the form of a probability model that can be used in the design and analysis of a clinical trial. Three trial design strategies are compared: (1) a sequential adult-pediatric bayesian approach that involves a separate, deferred pediatric trial that incorporates existing adult trial data into the analysis model to potentially reduce the pediatric trial sample size; (2) a parallel adult-pediatric bayesian trial whereby separate pediatric enrollment occurs in a parallel trial, running alongside an adult randomized clinical trial; and (3) a unified adult-pediatric bayesian adaptive design that supports the enrollment of both children and adults simultaneously in a whole-of-life bayesian adaptive randomized clinical trial. The SNAP trial whole-of-life design uses a bayesian hierarchical model that allows information sharing (also known as borrowing) between trial age groups by linking intervention effects of children and adults, thereby improving inference in both groups.
Conclusion And Relevance:
Bayesian hierarchical models may provide more precision for estimates of safety and efficacy of treatments in trials with heterogenous populations compared to traditional methods of analysis. They facilitate the inclusion of children in clinical trials and a shift from children deemed therapeutic orphans to the vision of no child left behind in clinical trials to ensure evidence for clinical practice exists across the life course. The SNAP trial provides an example of a bayesian adaptive whole-of-life inclusion design that enhances trial population inclusivity and diversity overall, as well as generalizability and translation of findings into clinical practice.
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