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Clinical trials design types PPT

The optimal design of phase II studies continues to be the subject of vigorous debate, especially studies of newer molecularly targeted agents. The observations that many new therapeutics “fail” in definitive phase III studies, coupled with the numbers of new agents to be tested as well as the increasing costs and complexity of clinical trials, further emphasize the critical importance of robust and efficient phase II design. The Clinical Trial Design Task Force (CTD-TF) of the National Cancer Institute (NCI) Investigational Drug Steering Committee (IDSC) has published a series of discussion papers on phase II trial design in Clinical Cancer Research. The IDSC has developed formal recommendations about aspects of phase II trial design that are the subject of frequent debate, such as endpoints (response versus progression-free survival), randomization (single-arm designs versus randomization), inclusion of biomarkers, biomarker-based patient enrichment strategies, and statistical design (e.g., two-stage designs versus multiple-group adaptive designs). Although these recommendations in general encourage the use of progression-free survival as the primary endpoint, randomization, inclusion of biomarkers, and incorporation of newer designs, we acknowledge that objective response as an endpoint and single-arm designs remain relevant in certain situations. The design of any clinical trial should always be carefully evaluated and justified based on characteristic specific to the situation. Clin Cancer Res; 16(6); 1764–9

Background

Many new drugs targeting molecular pathways are ready for clinical development, necessitating the use of efficient trial designs to quickly and accurately identify promising agents, while also identifying those for which all further development should be stopped. Although the development of some drugs is discontinued after phase I, the major drug development decision is generally made on the basis of phase II results. Although traditional oncology trial designs using the endpoint of response and a single arm design seem to have done this task reasonably well for cytotoxic agents, the same does not seem to be true for newer agents in which high rates of tumor shrinkage may not be expected, nor for combinations of agents (such as a new drug combined with standard treatments). Certainly, success rates for phase III trials seem to be decreasing. This decrease has led to considerable scientific discussion, debating the advantages and disadvantages of using response versus progression or other imaging endpoints, single arm versus randomized designs, patient enrichment and biomarker endpoints, and optimal statistical designs, such as adaptive design or phase I–II designs.

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When Is IRB Review of Clinical Trial Websites Required?

When information posted on a clinical trial website goes beyond directory listings with basic descriptive information, such information is considered part of the informed consent process and therefore requires IRB review and approval. Basic descriptive information includes:

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