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Proteinuria as a surrogate end point—more data are needed

Nature Reviews Nephrology volume 8, pages 306–309 (2012)Cite this article

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Abstract

Surrogate end points have the potential to facilitate drug development because effects on surrogate end points can sometimes be demonstrated more rapidly and in smaller studies than can effects on clinical outcomes of interest. Proteinuria has been repeatedly proposed as a surrogate end point for renal outcomes in drug development; however, the FDA has generally not accepted effects on proteinuria as evidence of a drug's effectiveness. Proteinuria is an early marker of some kidney diseases and increases in proteinuria can predict risk of disease progression. Whether or not treatment effects on proteinuria can reliably predict treatment effects on renal outcomes is not known. Nevertheless, it may be reasonable to use effects on proteinuria as a basis for accelerated approval of a drug if certain conditions are met. Approval under this pathway carries with it a requirement to complete a study after drug approval that verifies the anticipated treatment benefit.

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Acknowledgements

The author thanks Drs Norman Stockbridge and Robert Temple from the FDA's Center for Drug Evaluation and Research for their valuable comments on this manuscript.

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  1. Division of Cardiovascular and Renal Products, Office of New Drugs, Center for Drug Evaluation and Research, FDA, White Oak Campus, Building 22, Room 4232, 10903 New Hampshire Avenue, Silver Spring, 20993, MD, USA

    Aliza Thompson

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Thompson, A. Proteinuria as a surrogate end point—more data are needed. Nat Rev Nephrol 8, 306–309 (2012). https://doi.org/10.1038/nrneph.2012.43

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