Integrated phase II/III clinical trials in oncology: a case study.
| Autor: | Wang M; Department of Statistics, Radiation Therapy Oncology Group, American College of Radiology, Philadelphia, PA, USA. sumswang@yahoo.com, Dignam JJ, Zhang QE, DeGroot JF, Mehta MP, Hunsberger S |
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| Jazyk: | angličtina |
| Zdroj: | Clinical trials (London, England) [Clin Trials] 2012 Dec; Vol. 9 (6), pp. 741-7. Date of Electronic Publication: 2012 Nov 22. |
| DOI: | 10.1177/1740774512464724 |
| Abstrakt: | Background: Integrated phase II/III trial designs implement the phase II and phase III aspects of oncology studies into a single trial. Despite a body of literature discussing the merits of integrated phase II/III clinical trial designs within the past two decades, implementation of this design has been limited in oncology studies. Purpose: We provide a brief discussion of the potential advantages and disadvantages of integrated phase II/III clinical trial designs in oncology and provide an example of the operating characteristics of a Radiation Therapy Oncology Group (RTOG) trial. Methods: We review the differences among proposed integrated phase II/III designs. Then, we illustrate the use of the design in a brain tumor trial to be conducted by the RTOG and examine the impact of association between endpoints on design performance in terms of type I error, power, study duration, and expected sample size. Results: Although integrated phase II/III designs should not be used in all situations, under appropriate conditions, significant gains can be achieved when using integrated phase II/III designs, including smaller sample size, time and resources savings, and shorter study duration. Limitations: Data submission without delay and sufficient evaluation of intermediate endpoints are assumed. Conclusions: Although there are potential benefits in using phase II/III designs, there also may be disadvantages. We recommend running design simulations incorporating theoretical and practical issues before implementing an integrated phase II/III design. |
| Databáze: | MEDLINE |
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