Are there circumstances in which phase 2 study results should be practice-changing? Hematology Am Soc Hematol Educ Program 2007:489-92
Date
11/21/2007Pubmed ID
18024669DOI
10.1182/asheducation-2007.1.489Scopus ID
2-s2.0-67650267370 (requires institutional sign-in at Scopus site) 14 CitationsAbstract
New pharmaceuticals, innovative combinations of approved agents, and novel treatment modalities have resulted in a marked increase in the need for clinical trials. Evidence for treatment efficacy is best derived from large phase 3 randomized, controlled clinical trials. However, phase 3 investigations are lengthy and expensive, and consume patient resources. Furthermore, some diseases and treatment indications are rare, and adequate numbers of patients for a definitive phase 3 trial do not exist. Consequently, it is imperative for clinicians to understand phase 2 trial design, since their interpretation is required to apply the findings in clinical practice appropriately. The complexity of phase 2 studies is explored, including unique designs, possible use of randomization, and other key elements necessary for interpretation of phase 2 trials. Specific examples and application of these concepts are discussed in this review.
Author List
Tomblyn MR, Rizzo JDAuthor
J. Douglas Rizzo MD, MS Director, Center Associate Director, Professor in the Medicine department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
Clinical Trials, Phase II as TopicResearch Design