Table 1 Sources of efficacy data that may become available to support the choice of a candidate agent for repurposing

From: Drug repurposing for Alzheimer’s disease and other neurodegenerative disorders

Epidemiologic studies and data repositories

Pharmacoepidemiologic studies

Electronic medical record studies

Genetic studies including genome wide association studies

Omic data (genomics, proteomics, transcriptomics, metabolomics, lipidomics, connectomics)

Drug databases

Extrapolation from other neurodegenerative disorders

Patient and clinical observations

Technologies

Artificial intelligence and machine/deep learning algorithms applied to big data resources

In silico network-based drug-disease proximity assessment for repurposing

Trial emulation of repurposed agents

Mendelian randomization

Cell, induced pluripotent stem cell/organoid, and animal model observations

  1. Many types of data may suggest that an agent is appropriate for a clinical trial. There is more confidence in the success of an agent when multiple data sources converge in support of its relevance to the biological processes of the neurodegenerative disorder.
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