title: Therapeutic studies.
abstract: This article discusses the problems in basic design, conduct, and interpretation associated with phases I, II, and III of the cancer clinical trials and explains the various statistical solutions to these problems. The fundamental problem common to all three trials is achieving a correct and precise answer to the question posed to inform future testing and treatment while protecting trial patients from receiving treatment that has demonstrated excessive toxicity or lack of clinical efficacy. This shared problem gives rise to statistical designs with basic similarities across the three trial types.
authors.affiliation: Biometric Research Branch, National Cancer Institute, Bethesda, Maryland, USA. rubinsteinl@ctep.nci.nih.gov
authors.forename: L V
authors.initials: LV
authors.lastname: Rubinstein
mesh_terms: D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D019540:Area Under Curve; D001499:Bayes Theorem; D015415:Biomarkers; D002986:Clinical Trials as Topic / Q000379:methods / Q000592:standards / Q000706:statistics & numerical data; D017321:Clinical Trials, Phase I as Topic / Q000379:methods; D017322:Clinical Trials, Phase II as Topic / Q000379:methods; D018849:Controlled Clinical Trials as Topic / Q000379:methods; D004305:Dose-Response Relationship, Drug; D023381:Endpoint Determination; D005544:Forecasting; D017408:Guidelines as Topic; D006801:Humans; D007255:Information Services; D016015:Logistic Models; D015337:Multicenter Studies as Topic / Q000706:statistics & numerical data; D009369:Neoplasms / Q000188:drug therapy / Q000628:therapy*; D011446:Prospective Studies; D011897:Random Allocation; D016032:Randomized Controlled Trials as Topic / Q000379:methods; D012107:Research Design; D013269:Stochastic Processes; D016896:Treatment Outcome; D014481:United States
chemical_list: D000970:Antineoplastic Agents; D015415:Biomarkers