Phase I trials and therapeutic intent in the age of precision oncology: What is a patient's chance of response?

• Published in: European journal of cancer (1990) Vol. 139; pp. 20 - 26
• Main Authors: Tao, Derrick L., Kartika, Thomas, Tran, Audrey, Prasad, Vinay
• Format: Journal Article
• Language: English
• Published: England Elsevier Ltd 01.11.2020; Elsevier Science Ltd
• Subjects: Antineoplastic Agents - therapeutic use; Clinical trials; Clinical Trials, Phase I as Topic; Humans; Medical Oncology - methods; Neoplasms - drug therapy; Oncology; Phase I; Precision medicine; Precision Medicine - methods; Precision oncology; Probability; Response rate; Response rate; Precision oncology; Phase I
• ISSN: 0959-8049; 1879-0852
• DOI: 10.1016/j.ejca.2020.04.037

The advancement of therapeutic strategies in oncology such as precision oncology has generated significant interest in better estimating the response of modern phase I cancer clinical trials. These estimates have varied widely. In this commentary, we provide an umbrella review of phase I response rates and discuss methodological reasons for variation in prior estimates which include limited use of unpublished data, the inclusion of expansion cohorts that artificially raise response rates of cumulative response rates, varying enrolment of haematologic malignancies, and increased next in class drugs. •Phase I trial response rate estimates have had significant variation.•We identify 4 potential methodological reasons.•The median response rate is more relevant to patients than the overall response rate.•Future response rate estimates should disambiguate these methods.