Abstract
The landscape of nonsmall cell lung cancer (NSCLC) treatment was significantly affected by the novel paradigm of successfully pairing biomarker-defined cohorts of patients with targeted therapeutics such as EGFR mutations as biomarkers of benefit from epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (TKI) and EML-ALK translocations and ALK TKIs. This has led to substantial enthusiasm about following this paradigm for rapid development of novel therapeutics. Despite increased investment in research and development, the path to marketing authorization of new drugs remains challenging, and occasionally inefficient. Novel approaches to clinical development and trial design could be crucial in overcoming some of these challenges. Adaptive clinical trials have the advantage of testing several biomarker hypotheses within a single clinical trial, a model that increases flexibility and maximizes the use of accumulated knowledge. Two notable examples of this approach are the BATTLE study in lung cancer and ISPY-2 in breast cancer patients. Advantages and challenges of this trial methodology along with other aspects of clinical trials are discussed in this chapter.
Original language | English (US) |
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Title of host publication | Lung Cancer |
Subtitle of host publication | Fourth Edition |
Publisher | Wiley-Blackwell |
Pages | 479-487 |
Number of pages | 9 |
ISBN (Electronic) | 9781118468791 |
ISBN (Print) | 9781118468746 |
DOIs | |
State | Published - May 27 2014 |
Keywords
- Adaptive clinical trial designs
- Biomarkers
- Novel therapeutics
ASJC Scopus subject areas
- General Medicine