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Phase II trials in cancer: present status and analysis methods
Drugs Under Experimental and Clinical Research
|January 1, 1986
Summary
Phase II cancer studies often lack clear design, statistical analysis, and patient evaluation, leading to unreliable conclusions. Improvements in methodology are proposed to enhance the validity of clinical trial results.
Area of Science:
- Oncology
- Clinical Trial Methodology
- Biostatistics
Background:
- Phase II clinical trials are crucial for evaluating new cancer treatments.
- A significant number of published phase II studies exhibit methodological weaknesses.
Purpose of the Study:
- To critically review the design, conduct, and analysis of recent phase II cancer studies.
- To identify common deficiencies and propose improvements for future phase II trial methodology.
Main Methods:
- Systematic review of 67 recent phase II studies.
- Specific review of phase II breast cancer (17 studies) and colorectal cancer (19 studies) trials published between 1981-1983.
- Analysis of study design, eligibility criteria, statistical interpretation, patient evaluability, and randomization.
Main Results:
- Majority of studies lacked clear eligibility criteria, statistical methods for results interpretation, and consistent patient evaluability.
- Two-thirds of studies reported negative conclusions, with few providing statistical rationale for their design.
- Small study sizes and issues with patient subgroup comparability (e.g., prior chemotherapy) were noted in breast and colorectal cancer trials.
Conclusions:
- Significant shortcomings in the design, conduct, and analysis of phase II cancer studies were identified.
- Recommendations are made for enhancing the rigor and reliability of phase II trial methodology.
- Improved statistical methods and study designs, such as two-stage designs, are suggested for future cancer trials.