15. AlzPED: Optimizing the Predictive Power of Drug Efficacy Studies in Alzheimer’s Disease Animal Models

Poor translation of preclinical efficacy from animal models to the clinic is a major challenge to successful therapy development for Alzheimer’s disease (AD). Assessments of preclinical animal studies have highlighted the need for an emphasis on rigor in study design, methodology and data analysis, transparent reporting methods, mitigation of publication bias due to under-reporting of negative results, and the development of a set of best practices to optimize the predictive value of preclinical research testing candidate AD therapies. AlzPED is a publicly available data repository created by the National Institute on Aging and the National Institutes of Health Library to address the key factors contributing to the preclinical to clinical gap in AD therapy development. AlzPED is designed as a web-based knowledge portal for housing, sharing, and mining of preclinical efficacy data. The data are submitted to AlzPED through a curator and gleaned from multiple sources. Each study is carefully curated by two experts for data on authors, AD animal models, therapeutic targets and agents, outcomes and most importantly the rigor of the study, prior to publication in the database. AlzPED currently houses curated summaries from 1150 preclinical efficacy studies including 190 animal model descriptors, information on 220 therapeutic targets and 1000 therapeutic agents, and, more than 1500 AD-related outcome measures, principal findings, and information related to funding sources and financial conflict of interest, and reports on the rigor of each study by summarizing 24 critical elements of experimental design. Analysis of studies curated in AlzPED demonstrates a serious deficiency in reporting critical elements of design and methodology like power/sample size calculation, blinding for treatment and outcomes, randomization, balancing for sex, inclusion/exclusion criteria, resulting in increased susceptibility to misinterpretation and decreased scientific rigor, reproducibility and translational value. To mitigate the publication bias that favors the reporting of positive findings, AlzPED provides a platform for reporting unpublished negative findings. Accepted studies will be published in the AD Knowledge Portal and assigned a citable DOI. Finally, researchers can use this resource to survey existing preclinical therapy developments, understand the requirements for rigorous study design and transparent reporting and plan preclinical intervention studies. 

  • Shreaya Chakroborty
  • Ali Sharma
  • Zane Martin
  • Jean Yuan
  • Suzana Petanceska
  • Lorenzo Refolo
  • National Institute on Aging

Authors Participating In This Event

Shreaya Chakroborty

Scientific Program Man..., NIA/NIH

Scientific Program Manager

NIA/NIH

I am a Scientific Program Manager in the Translational Research Branch, Division of Neuroscience, National Institute on Aging. I earned my Ph.D. in Neuroscience from the Rosalind Franklin...