# Biotyping placebo and treatment-specific responses for precision medicine

> **NIH NIH R01** · NEW YORK UNIVERSITY SCHOOL OF MEDICINE · 2021 · $527,651

## Abstract

Summary
This application proposes advances to precision medicine along three complementary arcs, building on our initial
work in RO1-MH099003 (Characterizing Placebo Response to Active Treatment Using Very High-Dimension-
al Data). A primary component of precision medicine involves determining optimal treatment decisions using
baseline variables. We have previously developed statistical models that can accommodate both scalar and
functional predictors in determining treatment decision functions. This renewal application addresses the fact
that available data modalities continue to evolve and grow ever more complex. Determining treatment deci-
sions is closely related to the problem of quantifying the degree to which individual outcomes are due to spe-
cific drug effects versus placebo responses. We propose to develop completely nonparametric distance-based
approaches with the goal of determining optimal treatment decisions and generalizing these to accommodate
multiple data modalities. Because no single biomarker for response to treatment has been identified, methods
to form composite biomarkers by combining predictors will be developed to incorporate data arising from com-
plex modalities. Many brain-related measures are expensive and time consuming to collect, and thus a vital
component of optimizing treatment decisions is to develop a sequential treatment decision strategy whereby
easy-to-make measurements are obtained first, and the more expensive and time consuming measurements
are only obtained if they are needed to improve confidence in a treatment decision.
 A complementary arc of the proposed research involves stratified psychiatry and developing directed
partitioning/clustering methods to improve precision medicine. This is not only important to the work on deter-
mining optimal treatment decisions, but also towards a data-driven discovery of psychiatric biotypes. Optimizing
treatment decisions is naturally related to the thorny problem of making a clear diagnosis, especially in mental
health where there is a wide degree of heterogeneity and overlap in current disease phenotypes. This research
aims to expand the notion of endophenotypes from genetics to data modalities based on brain structure, func-
tion, and integrity. Specific to this application, the notion of endophenotypes will be expanded to discover more
stable phenotypes using biomedical technologies, that those obtained based on symptoms.
 The Establishing Moderators & Biosignatures for Antidepressant Response in Clinical Care (EMBARC;
U01MH092221, U01MH092250 ) study, which is the most ambitious systematic effort to discover biomarkers
to guide treatment of major depressive disorder, has collected and made publicly available an unprecedented
collection of clinical and biological patient phenotypes. This is a proposal to develop analytic methodology that
would be able to take full advantage of rich and complex patient data as those in the EMBARC study for the
purposes of preci...

## Key facts

- **NIH application ID:** 10107865
- **Project number:** 5R01MH099003-09
- **Recipient organization:** NEW YORK UNIVERSITY SCHOOL OF MEDICINE
- **Principal Investigator:** Eva Petkova
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $527,651
- **Award type:** 5
- **Project period:** 2013-02-01 → 2024-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10107865

## Citation

> US National Institutes of Health, RePORTER application 10107865, Biotyping placebo and treatment-specific responses for precision medicine (5R01MH099003-09). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10107865. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*
