Pragmatic Precision Psychiatry-A New Direction for Optimizing Treatment Selection

Area of Science:

• Psychiatry
• Medical Informatics
• Clinical Research

Background:

• Personalized treatment for mental disorders relies on prescriptive predictors, but individual predictors are insufficient for treatment planning.
• Developing precision treatment rules (PTRs) by combining multiple predictors has shown limited success in psychiatry compared to other medical fields.

Purpose of the Study:

• To review study designs and analysis schemes for PTR development in other medical areas.
• To highlight challenges in implementing PTR research for mental disorders.
• To describe recent methodological advances for developing and validating PTRs in psychiatry.

Main Methods:

• Review of existing PTR development strategies in medicine (large trials, data pooling, observational-to-trial approaches).
• Focus on the feasibility of using large observational electronic health record databases for preliminary PTR development in mental health.
• Integration of observational data, data augmentation, appropriate statistical methods, and pragmatic trials for validation and refinement.

Main Results:

• The most feasible approach for mental disorder PTRs involves developing preliminary rules in large observational samples, followed by testing in randomized clinical trials.
• New statistical methods are available to overcome methodological challenges in this integrated approach.
• An iterative process of discovery (observational studies) and confirmation (pragmatic trials) is essential.

Conclusions:

• Advances in pragmatic precision psychiatry necessitate a shift from solely relying on randomized clinical trials.
• An iterative discovery-confirmation framework integrating observational and experimental studies is crucial.
• This integrated approach will refine and expand PTRs for real-world clinical populations.