Precision Medicine

Key facts

Federal initiative

Precision Medicine Initiative, announced in 2015

NIH research cohort

All of Us Research Program

Key FDA device category

Companion diagnostics tied to specific drugs

Common data inputs

Genomic variants, biomarkers, clinical records

What is precision medicine?

Precision medicine groups patients by measurable characteristics, most often molecular ones, and uses those groupings to choose a test or treatment. A drug that works well for patients whose tumors carry a specific genetic variant, and poorly for everyone else, is the classic example.

The term overlaps with personalized medicine. Precision medicine is generally preferred because it describes sorting patients into subgroups based on evidence, not designing a unique treatment for each person.

In the United States, the Precision Medicine Initiative announced in 2015 led to the All of Us Research Program at the National Institutes of Health (NIH), which is building a large cohort that combines genomic, health record and lifestyle data.

How precision medicine works

Most precision medicine programs follow the same chain of steps:

• Discover a biomarker, such as a genetic variant or protein, that separates patients who respond from those who do not.
• Develop and validate a test that measures the biomarker reliably, often a companion diagnostic reviewed by the Food and Drug Administration (FDA).
• Run clinical trials that enroll or stratify patients by biomarker status.
• Write the biomarker into drug labeling, coverage policies and clinical guidelines.

Why precision medicine matters

Precision medicine changes how drug markets are sized. A therapy indicated only for patients with a particular variant has an addressable population defined by testing rates and variant prevalence, not by total disease incidence, so forecasts depend on how many patients are actually tested.

It also shapes commercial and research operations. Trial sponsors need sites that can identify biomarker-positive patients, diagnostic makers and drug makers must coordinate launches, and payers weigh test coverage alongside drug coverage. Resources such as ClinVar and FDA drug labeling are central to tracking which variants and biomarkers carry clinical weight.