Reinventing clinical trials: a review of innovative biomarker trial designs in cancer therapies.
BACKGROUND
Recently, new clinical trial designs involving biomarkers have been studied and proposed in cancer clinical research, in the hope of incorporating the rapid growing basic research into clinical practices.


METHODS
Journal articles related to various biomarkers and their role in cancer clinical trial, articles and books about statistical issues in trial design, and regulatory website, documents, and guidance for submission of targeted cancer therapies.


RESULTS
The drug development process involves four phases. The confirmatory Phase III is essential in regulatory approval of a special treatment.


RESULTS
Regulatory agency has restrictions on confirmatory trials 'using adaptive designs'. No rule of thumb to pick the most appropriate design for biomarker-related trials.


CONCLUSIONS
Statistical issues to solve in new designs. Regulatory acceptance of the 'newly proposed trial designs'.


CONCLUSIONS
Biomarker-related trial designs that can resolve the statistical issues and satisfy the regulatory requirement.
Department of Biostatistics, The University of North Carolina at Chapel Hill, 170 Rosenau Hall, Chapel Hill, NC, USA.
Ja-An
JA
Lin
Department of Statistics, Stanford University, Stanford, CA, USA he.pei@stanford.edu.
Pei
P
He
D014408:Biomarkers, Tumor / Q000378:metabolism*; D002986:Clinical Trials as Topic / Q000379:methods*; D003627:Data Interpretation, Statistical; D006801:Humans; D009369:Neoplasms / Q000175:diagnosis / Q000628:therapy*; D017410:Practice Guidelines as Topic; D057285:Precision Medicine / Q000379:methods; D012107:Research Design
D014408:Biomarkers, Tumor
adaptive design; biomarker; clinical trial; personalized medicine