Surrogate endpoints drug development

Sikora, K. (2002). Surrogate endpoints in cancer drug development. Drug Disc. Today 7 951-956. 

Lesko, L.J. and Atkinson, A.J., Jr., Biomarkers and surrogate endpoints — Use in drug development and regulatory decision making criteria, validation, strategies, Ann. Rev. Pharmacol. Toxicol, 41, 347-366, 2001. 

Lesko LJ, Atkinson AJ Jr. Use of biomarkers and surrogate endpoints in drug development and regulatory decision making. Ann Rev Pharmacol 2001 41 347-66. 

Surrogate endpoints are of particular value in early drug development to select candidate drugs from a range of agents. Over-zealous fixation on the use of surrogate endpoints can, however, lead to serious errors in decision-making. 

In Phase 2 the proof of concept study provides scientifically sound evidence supporting the postulated effect of the new drug, where the effect may be the relevant pharmacological action or a change in disease biomarkers, established surrogate endpoints, or clinical outcomes that may be beneficial and/or toxic in nature. The proof of concept is often used for go/no-go decisions and is therefore one of the most critical steps in the drug development process. 

Wagner, J. A. (2002) Overview of biomarkers and surrogate endpoints in drug develop ment. Disease Markers, 18, 41 46. 

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