Sept 19: For many people with depression, schizophrenia and other psychiatric conditions, finding an effective medication can take time. A patient may try one drug, wait weeks to see whether it works, deal with side effects or limited improvement, and then start the process again with another treatment.
Researchers now want to make that process more precise by looking beyond symptoms and diagnosis.
A new consensus paper from the American College of Neuropsychopharmacology (ACNP), co-led by UCLA Health, has outlined a roadmap for using biological markers to improve psychiatric drug development and, eventually, help match treatments more closely to individual patients.
Published in NPP – Digital Psychiatry and Neuroscience, the paper was developed by the ACNP Precompetitive Stakeholder Task Force. Its contributors include researchers from UCLA Health and other universities, pharmaceutical industry representatives, scientists from the National Institute of Mental Health and officials from the U.S. Food and Drug Administration’s Office of Neuroscience.
Why the Same Diagnosis Can Mean Different Biology
One of the biggest challenges in psychiatric medicine is that people with the same diagnosis may have very different underlying biology.
Two people with depression, for example, can experience similar symptoms while having different biological factors contributing to those symptoms. Yet psychiatric drug trials often group participants primarily according to their symptoms.
That can make it difficult to identify whether a treatment works particularly well for a smaller subgroup.
A medicine that produces a meaningful response in one biological group could appear less effective when its results are averaged across a much larger population.
The ACNP task force believes biomarkers could help researchers identify these differences.
What Are Biomarkers?
Biomarkers are measurable biological signals that can provide information about a person’s health or response to treatment.
In psychiatric research, scientists are exploring potential biomarkers from several sources, including blood, genetics, brain activity and wearable devices.
EEG, for instance, can measure electrical activity in the brain. Blood and genetic tests can provide information about biological characteristics, while wearable devices can capture certain physiological and behavioral patterns.
The future of precision psychiatry may involve combining several of these signals rather than depending on a single test.
The objective is to identify biological patterns that can help researchers understand why one treatment may work for one group of patients but not another.
What Psychiatry Can Learn From Cancer Care
Precision medicine has already changed parts of cancer treatment.
Doctors can use biological characteristics of certain tumors to help determine which therapies may be appropriate. Similar biomarker-based approaches have also been explored in cardiovascular medicine and neurological conditions such as Alzheimer’s and Parkinson’s disease.
The ACNP researchers argue that psychiatry could develop along a similar path.
Rather than viewing a psychiatric diagnosis as a single biological condition, researchers could identify measurable differences among patients and investigate whether those differences are associated with treatment response.
Biomarkers Could Change Drug Trials
One of the most immediate applications could be in clinical research.
If researchers can identify biological characteristics associated with treatment response, they could design studies around more clearly defined patient groups.
This could help solve a problem that has long complicated psychiatric drug development.
Consider a hypothetical medication that works particularly well for a relatively small biological subgroup but has little effect on other participants. When all patients are analyzed together, the overall result may make the treatment appear less effective.
A biomarker could potentially help researchers identify that subgroup and investigate the treatment response more precisely.
That could also help researchers better understand why some drug candidates fail to demonstrate a strong effect in conventional trials.
The ACNP Roadmap Focuses on Collaboration
The task force has proposed several steps to move biomarker research forward.
Researchers recommend developing common definitions for psychiatric biomarkers and establishing clear intended uses for them.
They also call for small, focused studies examining potential biomarkers involving blood tests, genetics, EEG recordings and wearable-device data. Promising biomarkers could then be tested against existing large datasets before researchers commit resources to costly new trials.
Data sharing is another major part of the proposal.
The researchers recommend a precompetitive framework in which companies and scientists can share data, including results from studies that failed to produce the expected outcome. Such information could help prevent researchers from repeatedly pursuing approaches that have already been tested.
The paper also calls for greater standardization in how biomarker data are collected and analyzed across research sites.
Regulators Could Be Involved Earlier
The roadmap also highlights the importance of working with regulators early in the research process.
The authors recommend consulting the FDA and European regulators as researchers develop new approaches, particularly methods that combine multiple types of biological information.
The paper also addresses what the authors describe as a common misunderstanding about regulatory requirements.
Biomarkers can already be used in drug trials without necessarily going through a lengthy FDA approval process, depending on their intended use. Greater understanding of this regulatory pathway could potentially remove one barrier to wider biomarker research.
Could This Eventually Reduce Medication Trial and Error?
The long-term possibility is perhaps the most significant for patients.
Today, treatment decisions for psychiatric conditions are generally based on a combination of diagnosis, symptoms, medical history, clinical judgment and the patient’s response to treatment.
If reliable biomarkers can eventually be validated, they could provide another source of information.
A clinician might someday be able to use biological information alongside symptoms and other clinical factors to determine which treatment is more likely to benefit a particular patient.
However, that is not yet standard psychiatric care.
The ACNP roadmap is primarily focused on improving drug development. Turning promising research biomarkers into validated tools for everyday clinical practice would require additional evidence and a separate process of validation.
A New Direction for Psychiatric Drug Development
The significance of the ACNP paper lies less in a single new test and more in its proposed framework for collaboration.
Academic researchers, pharmaceutical companies and regulators would need to work with shared standards, comparable data and a willingness to learn from both successful and unsuccessful experiments.
For patients, the potential benefit is straightforward: a future in which psychiatric treatment is informed by more than symptoms alone.
Precision psychiatry is still developing, and not every promising biomarker will ultimately prove useful. But the new roadmap identifies a path toward understanding the biological differences that may influence treatment response.
If that research succeeds, psychiatric medicine could gradually move from a predominantly trial-and-error approach toward one that is more targeted, measurable and biologically informed.
