Shifting the trajectory of therapeutic development for neurological and psychiatric disorders.
Dimitri KraincWilliam J MartinKimberley J BillingsleyFrances E JensenSarah A TishkoffWilliam Z PotterSteven E HymanPublished in: Science translational medicine (2023)
Clinical trials for central nervous system disorders often enroll patients with unrecognized heterogeneous diseases, leading to costly trials that have high failure rates. Here, we discuss the potential of emerging technologies and datasets to elucidate disease mechanisms and identify biomarkers to improve patient stratification and monitoring of disease progression in clinical trials for neuropsychiatric disorders. Greater efforts must be centered on rigorously standardizing data collection and sharing of methods, datasets, and analytical tools across sectors. To address health care disparities in clinical trials, diversity of genetic ancestries and environmental exposures of research participants and associated biological samples must be prioritized.
Keyphrases
- clinical trial
- healthcare
- phase ii
- rna seq
- open label
- human health
- double blind
- air pollution
- electronic health record
- study protocol
- genome wide
- health information
- big data
- copy number
- quality improvement
- gene expression
- cerebrospinal fluid
- machine learning
- single cell
- affordable care act
- data analysis
- brain injury
- cerebral ischemia