Twenty-six Novel Therapies have been FDA-approved so far this year.

Medical journals, including “The Lancet”, see this as a difficult task. The Agency must maintain strict safety rules while simultaneously gaining access to novel medication.

The term “Novel medicine” in healthcare is defined as medicine that has not yet received prior FDA approval.  Using a surrogate endpoint shortens the time required to receive that approval.

Studies are still required to confirm a drug’s clinical benefit. If the drug provides a benefit, the FDA will grant its approval.

If, on the other hand, the drug being investigated does not provide a benefit, the drug may be removed from the market.

Clinical endpoints

Surrogate Endpoint Resources for Drug and Biologic Development

The U.S. Food and Drug Administration (FDA)  carries the burden of ensuring that biologics show that the drugs they use are safe and effective. Clinical trials are conducted to demonstrate that new medical products deliver a positive balance of benefit and risk.

What are clinical trial endpoints?

Clinical trial endpoints measure results of the trial. When a trial evaluates the efficacy of a new medical product or a new use for an approved product, investigators may choose endpoints that directly measure the clinical outcome they want to study. Conversely, a surrogate may be chosen.

• Clinical outcomes directly measure whether people in a trial feel or function better, or live longer. The benefit or likely benefit of a therapy, as measured by clinical outcomes (e.g., improvement in symptoms), is assessed to determine whether it outweighs any adverse effects.
• Surrogate endpoints may be used instead of clinical outcomes in some clinical trials. For example, surrogate endpoints are used when clinical outcomes, like strokes, might take a very long time to study, or in cases where the clinical benefit of improving the surrogate endpoint, such as controlling blood pressure, is well understood.
• Before a surrogate endpoint can be accepted in place of a clinical outcome, extensive evidence must accumulate, including evidence from epidemiological studies and clinical trials.
• Usually, clinical trials are needed to show that the surrogate endpoint can be relied upon to predict clinical benefit in a context of use. Surrogate endpoints that have undergone this extensive testing are called validated surrogate endpoints and these are accepted by the FDA as evidence of benefit.
• Between 2010 and 2012, the FDA approved 45 percent of new drugs based on a surrogate endpoint. Sometimes surrogate endpoints can support an accelerated approval with lesser evidentiary support, when they are “reasonably likely to predict a clinical benefit”, as described below.

When a surrogate endpoint predicts a beneficial effect, its use usually results in drug development programs.

For example, many clinical trials, using medications that lower blood pressure, have shown that reducing systolic blood pressure reduced the risk of stroke. Therefore, measurement of reduction in the surrogate endpoint of systolic blood pressure can stand in for the clinical outcome of stroke, and clinical trials targeting the reduction of risk of stroke can be conducted more rapidly in smaller populations using this validated surrogate endoint.

What is a biomarker?

Some surrogate endpoints are a small subclass of biomarkers.  A biomarker is a defined characteristic that is measured as an indicator of normal biological processes, pathologic processes, or responses to an exposure or intervention. Identifying patients for clinical trial enrollment, monitoring the safety of a therapy, or finding out if a treatment is having the desired effect on the body.

What is biomarker qualification?

Through this program biomarker developers may request regulatory qualification of a biomarker for a particular context of use in drug development.

What is the difference between biomarkers and clinical outcome assessments?

Biomarkers should not be confused with clinical outcome assessments (COAs), that describes how an individual feels or functions, or how long the person lives.

Although COAs are often used to determine whether or not a drug used in a clinical trial provides a treatment benefit, unlike biomarkers, COAs are measured using a report generated by a clinician, patient, non-clinician observer, or a performance-based assessment.

For further information on COAs, FDA has created a COA Compendium that summarizes COA information for many different diseases and conditions.

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Rose Duesterwald        September 24, 2026