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Trial Design: Precision medicine (Milestone 10.D)

Create new research programs to implement innovative trial designs with a precision medicine research paradigm. These efforts should include support for the development of novel statistical approaches (e.g., Bayesian methods, modeling, and simulations) for more efficient trial design and resources to evaluate the utility of intermediate, prognostic endpoints and platform trials such as umbrella, basket, etc.

Success Criteria

  • Launch at least 6 clinical trials using innovative trial designs such as adaptive trial design, functional challenge studies, pragmatic clinical trials, population-based cohort designs, and clinical trial/population-based cohort hybrid designs.

  • At least 3 clinical trials should stratify participant risk groups using dense "omics" and gene-environment interaction profiling.

  • Support the development of harmonized outcomes to be used across trials to enable comparative effectiveness analyses.

  • Support administrative data linkages with EHR and private payer claims data.

Summary of Key Accomplishments

NIA has issued several calls to build resources for dementia care research using pragmatic clinical trial designs for dementia care, and several projects have been funded. For example, the IMPACT Collaboratory facilitates embedded pragmatic dementia care intervention research with various health care systems across the U.S. Other pragmatic trial awards include those in assisted living and nursing home settings as well as community- or primary care-based programs. Novel methods and trial designs, such as adaptive designs, are also being used in cognitive training projects

NIA also supports Phase 2/3 of the Dominantly Inherited Alzheimer Network Trial to test the efficacy of two anti-amyloid immunotherapies (gantenerumab and solanezumab) using an adaptive design to enable the simultaneous testing of two therapeutic agents. While the drug treatments did not result in cognitive benefit, positive biomarker changes were observed in a subset of trial participants for one of the tested drugs.

The key accomplishments summary is current as of March 2022. 

Research Implementation Area
Trial Design
In Progress

Accomplishments/Implementation Activities

Funding Initiatives

Research Programs and Resources

Research Highlights

Relevant Recommendations

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