Design Statistics Core (D)

The design and analysis of embedded pragmatic clinical trials (ePCTs), particularly those evaluating the effectiveness of interventions to improve dementia care, pose unique challenges requiring specialized expertise. As such, the Design and Statistics Core (DSC) is critical to realizing the National Institute on Aging Imbedded Pragmatic Alzheimer's disease and AD-Related Dementias Clinical Trials (IMPACT) Collaboratory’s mission: to build the nation’s capacity to conduct ePCTs of interventions embedded in health care systems for people living with dementia (PLWD) and their care partners (CPs). In the first funding cycle, the DSC supported the development of ePCTs by providing hundreds of hours of consultation to investigators. Key areas of support included overall ePCT design, sample size estimation, analytic approach, and consideration of population differences. Additionally, it produced more than two dozen peer-reviewed publications, as well as technical documents supporting trial design and analysis; online media and guidance on issues in trial design; simulation and sample size software; and numerous national and international presentations & symposia. DSC activities during the proposed renewal period will extend these efforts while being responsive to the emergent needs of IMPACT initiatives. Methodological developments will emphasize two areas of pressing need: first, designs, sample size machinery, and analytic software appropriate to evaluating heterogenous treatment effects; and second, generalizations of dyadic designs & analyses relevant to trials among PLWD and their CPs across various populations. The Specific Aims of the DSC are: 1. To support the design and conduct of ePCTs among PLWD and their CPs by providing guidance and consultation on trial design, statistical analysis, and sample size considerations to IMPACT applicants and awardees, IMPACT Launchpad teams, and applicants to other NIA grant mechanisms. 2. To enhance investigator capacity to design and conduct ePCTs among PLWD and their CPs by providing training in the design, statistical analysis, and sample size considerations for this research. These efforts will include: participating in IMPACT training opportunities; serving as a national resource to all investigator trainees supported by IMPACT and other NIA mechanisms with a focus on ePCTs in dementia; and mentoring and engaging Faculty Scholars and post-doctoral Fellows in DSC activities. 3. To develop and disseminate knowledge to advance best practices for trial design, statistical analysis, and sample size considerations of ePCTs conducted among PLWD and their CPs. Products and modes of dissemination will include: peer-reviewed publications, guidance documents, technical reports, presentations at scholarly venues, and various other media (e.g., online tutorials, podcasts). IMPACT: The Collaboratory’s bold vision is to transform the delivery, quality, and outcomes of care provided to Americans living with dementia and their CPs. The DSC will support this agenda through rigorous collaborative engagement with investigators, training of quantitative and allied scientists, and through the development of novel biostatistical methodology.