Clinical trials, which are required to bring a new drug to regulatory approval for clinical use, are lengthy and very expensive and thus represent a major hurdle in the development of novel cancer therapeutics. Today there are hundreds of new cancer therapeutics in the pipeline, but prediction of efficacy and toxicity in humans from findings in animal models has often proved unreliable, and only a few drug candidates become useful therapies.  For these and other reasons, scientists seek a new paradigm that could improve the efficiency and cost-effectiveness of clinical trials, while maintaining the highest standards of quality.

For example, in traditional Phase I cancer trials a new drug or agent is administered in gradually increasing doses to a mixed group of patients who have failed all effective forms of treatment.  The targeted endpoints are documentation of dose-limiting toxicities and collection of pharmacologic data, which together determine the dose and schedule that will be utilized in further studies.  Efficacy data also are collected, and may be used to identify targeted populations for Phase II trials.  Novel trial designs could be used to extract more information from early phase trials and improve the efficiency of subsequent trial phases.

In addition, many new drug candidates target specific molecules, genes, or pathways.  In preclinical studies, most of these drug candidates are more effective when combined with other targeted agents, reflecting the complexity of multi-step carcinogenesis.  But traditionally, drugs have been developed and tested individually, so combining new therapies in clinical trials presents numerous challenges to be overcome.

The goal of this workshop was to stimulate discussion on potentially fruitful ways to reduce the cost and shorten the long period of time needed for cancer clinical trials by improving the efficiency and quality of clinical trials.  Improvement of clinical trials require the collaboration of a number of disciplines and stakeholders and the introduction of new trial designs, methods, and diagnostic tools.  It also requires adequate funding for all elements of more sophisticated and informative clinical trial designs.

The National Cancer Policy Forum hosted this public workshop addressing issues related to improving the efficiency and cost effectiveness of cancer clinical trials while maintaining the highest standards of quality.  The workshop included presentations and discussions on new clinical trial designs such as phase 0 trials and exploratory INDs, adaptive trial designs, and targeting multiple pathways with multiple drugs. The workshop also explored the role of imaging technologies, screening for predictive markers, regulatory issues, and collaborations (including funding) among academia, PhRMA, Biotech, and Government.  Workshop attendess had the opportunity to participate in small group discussions to review case studies of clinical trial designs.  The workshop, held at the open meeting of the Forum on October 4th and 5th, 2007, featured invited presentations and discussion and served to inform the Forum.  An independently authored summary of the workshop was subsequently prepared.