Randomized Phase II Trials: A Long-term Investment With Promising Returns

• Published in: JNCI : Journal of the National Cancer Institute Vol. 103; no. 14; pp. 1093 - 1100
• Main Authors: Sharma, Manish R., Stadler, Walter M., Ratain, Mark J.
• Format: Journal Article
• Language: English
• Published: Cary, NC Oxford University Press 20.07.2011; Oxford Publishing Limited (England)
• Subjects: Antineoplastic Agents - therapeutic use; Biological and medical sciences; Chemotherapy, Adjuvant; Clinical trials; Clinical Trials, Phase II as Topic - methods; Clinical Trials, Phase II as Topic - standards; Clinical Trials, Phase III as Topic; Confounding Factors (Epidemiology); Drugs; Endpoint Determination; Evidence-Based Medicine; Health Resources - utilization; Humans; Life Expectancy; Medical sciences; Molecular Targeted Therapy - methods; Molecular Targeted Therapy - standards; Molecular Targeted Therapy - trends; Multiple tumors. Solid tumors. Tumors in childhood (general aspects); Oncology; Patient Selection; Patients; R&D; Randomized Controlled Trials as Topic - methods; Randomized Controlled Trials as Topic - statistics & numerical data; Reproducibility of Results; Research & development; Research Design - standards; Research Design - trends; Review; Sample Size; Selection Bias; Time Factors; Treatment Failure; Tumors; Antineoplastic agent; Human; Randomization; Cancerology; Treatment; Phase II trial; Malignant tumor; Long term; Cancer

Given the multitude of novel anticancer drugs and the limited resources available to study them, phase II trials should identify drugs with the highest probability of succeeding in subsequent phase III trials. Currently, single-arm phase II trial results are interpreted relative to historical contro...