Abstract
Clinical research is essential to introduce new treatments for cancer patients. Traditionally, a clinical research program starts with phase I trials, with the aim of describing the safety and identifying the dose and schedule to be tested in the subsequent phases of development. Phase II trials describe the antitumor activity of the experimental treatment, while the demonstration of efficacy and clinical benefit for the patient is obtained with the conduction of randomized phase III trials, which represent the highest level of evidence. Methodology of clinical trials in oncology has been profoundly challenged by the development of target-based agents and more recently by immune checkpoint inhibitors that represent the new generation of immunotherapy. Sample size, study design, selection criteria, study endpoints, and even the aim and the distinction of the phases are currently very different from the traditional program.
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References
Eisenhauer EA. Phase I and II trials of novel anti-cancer agents: endpoints, efficacy and existentialism. The Michel Clavel Lecture, held at the 10th NCI-EORTC Conference on New Drugs in Cancer Therapy, Amsterdam, 16-19 June 1998. Ann Oncol. 1998;9:1047–52.
Seymour L. The design of clinical trials for new molecularly targeted compounds: progress and new initiatives. Curr Pharm Des. 2002;8:2279–84.
Fox E, Curt GA, Balis FM. Clinical trial design for target-based therapy. Oncologist. 2002;7:401–9.
Le Tourneau C, Lee JJ, Siu LL. Dose escalation methods in phase I cancer clinical trials. J Natl Cancer Inst. 2009;101:708–20.
Dahlberg SE, Shapiro GI, Clark JW, Johnson BE. Evaluation of statistical designs in phase I expansion cohorts: the Dana-Farber/Harvard Cancer Center experience. J Natl Cancer Inst. 2014;106(7):dju163.
Therasse P, Arbuck SG, Eisenhauer EA, et al. New guidelines to evaluate the response to treatment in solid tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada. J Natl Cancer Inst. 2000;92:205–16.
Eisenhauer EA, Therasse P, Bogaerts J, et al. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur J Cancer. 2009;45:228–47.
Choi H. Critical issues in response evaluation on computed tomography: lessons from the gastrointestinal stromal tumor model. Curr Oncol Rep. 2005;7:307–11.
Seymour L, Bogaerts J, Perrone A, et al. iRECIST: guidelines for response criteria for use in trials testing immunotherapeutics. Lancet Oncol. 2017;18:e143–52.
Korn EL, Arbuck SG, Pluda JM, et al. Clinical trial designs for cytostatic agents: are new approaches needed? J Clin Oncol. 2001;19:265–72.
Rubinstein LV, Korn EL, Freidlin B, et al. Design issues of randomized phase II trials and a proposal for phase II screening trials. J Clin Oncol. 2005;23:7199–206.
Monzon JG, Hay AE, McDonald GT, et al. Correlation of single arm versus randomised phase 2 oncology trial characteristics with phase 3 outcome. Eur J Cancer. 2015;51:2501–7.
Smith GC, Pell JP. Parachute use to prevent death and major trauma related to gravitational challenge: systematic review of randomised controlled trials. BMJ. 2003;327:1459–61.
Hayes MJ, Kaestner V, Mailankody S, Prasad V. Most medical practices are not parachutes: a citation analysis of practices felt by biomedical authors to be analogous to parachutes. CMAJ Open. 2018;6:E31–8.
Di Maio M, Gallo C, De Maio E, et al. Methodological aspects of lung cancer clinical trials in the era of targeted agents. Lung Cancer. 2010;67:127–35.
Schnipper LE, Davidson NE, Wollins DS, et al. Updating the American Society of Clinical Oncology Value Framework: revisions and reflections in response to comments received. J Clin Oncol. 2016;34:2925–34.
Cherny NI, Dafni U, Bogaerts J, et al. ESMO-magnitude of clinical benefit scale version 1.1. Ann Oncol. 2017;28:2340–66.
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Di Maio, M., Perrone, F. (2021). Clinical Trials and Methodology of Cancer Research. In: Russo, A., Peeters, M., Incorvaia, L., Rolfo, C. (eds) Practical Medical Oncology Textbook. UNIPA Springer Series. Springer, Cham. https://doi.org/10.1007/978-3-030-56051-5_16
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DOI: https://doi.org/10.1007/978-3-030-56051-5_16
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