This Article Full Text Full Text (PDF) Alert me when this article is cited Alert me if a correction is posted Services Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Citing Articles Citing Articles via HighWire Citing Articles via Google Scholar Google Scholar Articles by Michaelis, L. C. Articles by Ratain, M. J. Search for Related Content PubMed PubMed Citation Articles by Michaelis, L. C. Articles by Ratain, M. J. Related Collections Related Article

Phase II Trials Published in 2002: A Cross-Specialty Comparison Showing Significant Design Differences between Oncology Trials and Other Medical Specialties

Authors' Affiliation: University of Chicago, Department of Medicine, Section of Hematology/Oncology, Chicago, Illinois

Requests for reprints: Mark J. Ratain, Section of Hematology/Oncology, 5841 S. Maryland Ave., MC2115 Chicago, IL 60637-1470. Phone: 773-702-4400; Fax: 773-702-3969; E-mail: MRatain{at}medicine.bsd.uchicago.edu.

Purpose: Phase II trials play an essential role in drug development pathway, and their conclusions often impact the decision to embark on large, pivotal trials. However, the determination of agent activity is highly dependent on trial design. Formal comparisons of phase II trial designs across medical specialties are uncommon. We hypothesized that there are significant differences in the design of trials conducted by oncologists and those conducted by other medical and surgical specialties.

Experimental Design: We screened MEDLINE for the abstracts of phase II trials published in 2002. All abstracts were analyzed and classified by a priori defined variables, including study type, intervention, subspecialty, journal impact factor, method of control, and study conclusions.

Results: Our search yielded 703 abstracts of phase II trials published in 2002. A total of 586/703 (83%) were trials on antineoplastic agents. Twenty percent (143/703) of the trials included explicit control subjects. Oncology trials, as compared with all trials done by other specialties, were significantly less likely to use control subjects (13% versus 56%, P < 0.001) and were less likely to conclude that the investigational intervention was safe and efficacious and/or worthy of additional investigation (76% versus 89%, P < 0.01).

Conclusions: There are significant differences in the phase II trials published in oncology compared with those conducted by other medical and surgical specialties. The impact that such differences have on the efficiency of drug development should be investigated.

Related Article

Phase II Cancer Trials: Out of Control?

Bruce Chabner
Clin. Cancer Res. 2007 13: 2307-2308. [Full Text] [PDF]

This article has been cited by other articles:

				J. H. Doroshow Commentary: Publishing Cancer Clinical Trial Results: A Scientific and Ethical Imperative Oncologist, September 1, 2008; 13(9): 930 - 932. [Full Text] [PDF]

				S. Kopetz, M. Overman, D. Z. Chang, K. Y. Glover, I. Shureiqi, R. A. Wolff, J. L. Abbruzzese, and C. Eng Systematic Survey of Therapeutic Trials for Metastatic Colorectal Cancer: Room for Improvement in the Critical Pathway J. Clin. Oncol., April 20, 2008; 26(12): 2000 - 2005. [Abstract] [Full Text] [PDF]

				W. M. Stadler Tumor Burden Endpoints and Phase II Clinical Trial Design ASCO Educational Book, January 1, 2008; 2008(1): 89 - 93. [Abstract] [Full Text] [PDF]

				M. J. Ratain and R. H. Glassman Biomarkers in Phase I Oncology Trials: Signal, Noise, or Expensive Distraction? Clin. Cancer Res., November 15, 2007; 13(22): 6545 - 6548. [Full Text] [PDF]

				B. A. Chabner and M. J. Ratain Clarification Regarding "Phase II Trials Published in 2002: A Cross-Specialty Comparison Showing Significant Design Differences between Oncology Trials and Other Medical Specialties" and the Accompanying Commentary, "Phase II Cancer Trials: Out of Control?" Clin. Cancer Res., November 1, 2007; 13(21): 6540 - 6540. [Full Text] [PDF]

				Design of Phase II Oncology Trials Journal Watch Oncology and Hematology, May 14, 2007; 2007(514): 6 - 6. [Full Text]

				B. Chabner Phase II Cancer Trials: Out of Control? Clin. Cancer Res., April 15, 2007; 13(8): 2307 - 2308. [Full Text] [PDF]

				M. R. Tomblyn and J. D. Rizzo Are There Circumstances in Which Phase 2 Study Results Should Be Practice-Changing? Hematology, January 1, 2007; 2007(1): 489 - 492. [Abstract] [Full Text] [PDF]