Surrogate markers in AIDS and cancer trials.
There is significant need for rapid yet reliable evaluation of promising new interventions for the treatment of patients with cancer or HIV infection. Considerable attention has been given to identifying replacement or 'surrogate' endpoints for the true clinical efficacy endpoints, in order to reduce the cost, size and duration of clinical trials. We discuss issues which affect the validity of surrogate markers. The reliability of the CD4 lymphocyte count marker is carefully considered in clinical trials of anti-retroviral agents in HIV infected individuals. The nature of surrogate markers and their reliability is discussed in cancer prevention, screening and treatment trials. Some suggested uses of marker information are also considered.
Fleming
T R
TR
Department of Biostatistics, University of Washington, Seattle 98195.
D000163:Acquired Immunodeficiency Syndrome / Q000188:drug therapy* / Q000276:immunology / Q000401:mortality; D000998:Antiviral Agents / Q000627:therapeutic use; D014408:Biomarkers, Tumor / Q000032:analysis; D015496:CD4-Positive T-Lymphocytes / Q000187:drug effects* / Q000276:immunology; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data*; D017326:Clinical Trials, Phase III as Topic / Q000706:statistics & numerical data; D005500:Follow-Up Studies; D006801:Humans; D007958:Leukocyte Count / Q000187:drug effects*; D009369:Neoplasms / Q000188:drug therapy* / Q000401:mortality; D015203:Reproducibility of Results; D016019:Survival Analysis; D016896:Treatment Outcome
D000998:Antiviral Agents; D014408:Biomarkers, Tumor