Author Affiliation: Center for Drug Evaluation and Research, Food and Drug Administration, Rockville, Md.
Controversies Section Editor: Phil B. Fontanarosa,
MD, Interim Coeditor.
The use of surrogate end points as a basis for reaching conclusions
about the benefit of therapy has been met with both rising enthusiasm, reflected
in recent changes in the Food, Drug and Cosmetic Act1
and some recent US Food and Drug Administration (FDA) actions, and rising
concern, reflected in several strongly stated warnings.2- 4
The mixed response is not surprising: reliance on surrogates, when the surrogate
proves to predict clinical benefit, can bring treatment benefits to patients
years before information on clinical outcomes could be available and at relatively
low cost. But reliance on surrogates, when the effect on the surrogate does
not lead to clinical benefit, can lead to the adoption of useless or even
harmful therapies. The obvious community goal is to make decisions most likely
to yield the former of these outcomes.
Temple R. Are Surrogate Markers Adequate to Assess Cardiovascular Disease Drugs?. JAMA. 1999;282(8):790-795. doi:10.1001/jama.282.8.790