ACNP Position Paper Lays Out Roadmap for Biomarker-Guided Psychiatric Drug Trials

A new position paper from the American College of Neuropsychopharmacology (ACNP) and co-led by UCLA Health outlines a plan for developing biomarkers that could help identify which patients with psychiatric disorders are most likely to respond to a given treatment.¹

The consensus paper, published in the journal NPP – Digital Psychiatry and Neuroscience, was developed by the ACNP Precompetitive Stakeholder Task Force. Its authors include university researchers including UCLA Health, representatives of the pharmaceutical industry, scientists from the National Institute of Mental Health, and members of the FDA's Office of Neuroscience.

Precision medicine offers an opportunity to move psychiatric drug development beyond treating everyone who shares a diagnosis as though they share the same underlying biology. Biomarkers could help identify biologically meaningful subgroups, determine whether a drug is engaging its intended target, and ultimately identify which patients are most likely to benefit from a particular treatment. This could produce more informative clinical trials and reduce some of the trial-and-error that currently characterizes treatment development in psychiatry.

“The promise of precision psychiatry is not simply to find more biomarkers—it is to identify biological measures that actually change how we develop treatments and, ultimately, how we select the right treatment for the right patient,” Sahib Khalsa, a psychiatrist and director of Anxiety Disorders Research at the UCLA Semel Institute for Neuroscience and Human Behavior, told Psychiatric Times.

The Heterogeneity Problem

The authors propose using biomarkers—measurable signals from blood, brain activity or wearable devices—to more precisely identify subgroups and design better trials.² Eventually, these tools could help guide treatment choices for individual patients.

Precision biomarkers have already informed care in oncology, cardiology, and neurologic diseases such as Alzheimer disease and Parkinson disease, and the authors argue psychiatry can follow a similar path.

“Right now, patients rely on repeated trials to learn whether a medication is working for them, which is costly and time-consuming,” said Khalsa, who is also the paper’s first author. “The goal of this effort is to give clinicians, researchers, drug developers, and drug regulators better ways to collaborate and see the biology behind a person's symptoms so treatments can be matched more accurately from the start.”

Key Recommendations

One of the central conclusions of the position paper is that the bottleneck is no longer simply biomarker discovery. Psychiatry has generated many promising biological signals, but very few have progressed into validated, regulatory-interpretable tools. Getting there will require shared standards, rigorous independent validation, better data sharing, early engagement with regulators, and precompetitive collaboration among academia, industry, government, and other stakeholders.

"Precision psychiatry is unlikely to emerge from one spectacular biomarker discovery. It will require an ecosystem that can reliably move promising discoveries through validation and into tools that are actually useful for psychiatric drug development and clinical decision-making,” Khalsa told Psychiatric Times.

With that in mind, here are the paper’s top recommendations:

• Agree on common definitions and clear intended uses for psychiatric biomarkers.
• Run small, focused studies of blood tests, genetics, electroencephalography (EEG), and wearable device data that can show early signs of treatment response.
• Test promising biomarkers in existing large datasets before launching costly new trials.
• Create a precompetitive framework for data sharing, including negative results, available to both scientists and companies.
• Standardize how biomarker data are collected and analyzed across research sites.
• Consult the FDA and European regulators early, including on newer approaches that combine multiple types of data.
• Show industry and payers how biomarker-guided trials can lower costs and improve results.

Next Steps

The authors emphasize the roadmap is focused on precompetitive collaboration for drug development. Bringing validated tests into routine clinical care is a separate, later step. They also address what they describe as a common misunderstanding that may be slowing progress: biomarkers can already be used in drug trials without a lengthy FDA approval process.

"For clinicians, the key question is not whether a biomarker is scientifically interesting. It is whether knowing the biomarker result would cause you to make a different—and better—decision for the patient in front of you,” concluded Khalsa.

References

1. Khalsa SS, Barch D, Brady LS, et al. Toward actionable biomarkers in psychiatry: a collaborative roadmap for precision clinical trials. An ACNP position paper. NPP Digit Psychiatry Neurosci. 2026;18(4).

2. UCLA Health. Cancer care uses biological tests to guide treatment. Why doesn't psychiatry? News release. September 18, 2026. Accessed September 29, 2026. https://www.newswise.com/articles/cancer-care-uses-biological-tests-to-guide-treatment-why-doesn-t-psychiatry/