Precision clinical trials: a framework for getting to precision medicine for neurobehavioural disorders

The goal of precision medicine (individually tailored treatments) is not being achieved for neurobehavioural conditions such as psychiatric disorders. Traditional randomized clinical trial methods are insufficient for advancing precision medicine because of the dynamic complexity of these conditions...

Full description

Saved in:
Bibliographic Details
Published in:Journal of psychiatry & neuroscience 2021-01, Vol.46 (1), p.E97-E110
Main Authors: Lenze, Eric J, Nicol, Ginger E, Barbour, Dennis L, Kannampallil, Thomas, Wong, Alex W K, Piccirillo, Jay, Drysdale, Andrew T, Sylvester, Chad M, Haddad, Rita, Miller, J Philip, Low, Carissa A, Lenze, Shannon N, Freedland, Kenneth E, Rodebaugh, Thomas L
Format: Article
Language:English
Subjects:
Anxiety
Biomarkers
Clinical trials
EEG
Intervention
Learning algorithms
Magnetic fields
Mathematical models
Mental disorders
Neuroimaging
Patients
Phenotyping
Precision medicine
Review Paper
Statistical analysis
Transcranial magnetic stimulation
Citations: Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:The goal of precision medicine (individually tailored treatments) is not being achieved for neurobehavioural conditions such as psychiatric disorders. Traditional randomized clinical trial methods are insufficient for advancing precision medicine because of the dynamic complexity of these conditions. We present a pragmatic solution: the precision clinical trial framework, encompassing methods for individually tailored treatments. This framework includes the following: (1) treatment-targeted enrichment, which involves measuring patients' response after a brief bout of an intervention, and then randomizing patients to a full course of treatment, using the acute response to predict long-term outcomes; (2) adaptive treatments, which involve adjusting treatment parameters during the trial to individually optimize the treatment; and (3) precise measurement, which involves measuring predictor and outcome variables with high accuracy and reliability using techniques such as ecological momentary assessment. This review summarizes precision clinical trials and provides a research agenda, including new biomarkers such as precision neuroimaging, transcranial magnetic stimulation-electroencephalogram digital phenotyping and advances in statistical and machine-learning models. Validation of these approaches - and then widespread incorporation of the precision clinical trial framework - could help achieve the vision of precision medicine for neurobehavioural conditions.
ISSN:1180-4882
1488-2434
DOI:10.1503/jpn.200042