A Manual for Participants in Clinical Trials of Investigational Agents ...

2 Sponsors, Clinical Trial Sites, and the Investigator
When investigators perform clinical trials, two elements are crucial – the trial’s sponsor
and the clinical trial site. The next two sections (Section 2 and Section 3) discuss the
purposes and features of each.
• We describe DCTD’s role as a sponsor of investigational agent trials.
• Specifically, we review CTEP’s responsibility for overall direction of the process
of investigational agent development and its practical implementation.
• We outline the basis of the relationship between DCTD, pharmaceutical
collaborators, and investigators during the conduct of clinical trials.
• We also discuss the clinical trial site’s vital role in support of the investigator. As
is the case with all types of research, clinical trials require a substantial
institutional commitment.
2.1 The Sponsor
Development of new anticancer agents (vs new agents vs investigational agents) is a
long and complex process, but successes have been significant. The fact that some
patients with aggressive neoplasms now have long term survival is the best possible
evidence that agents with selectivity against cancer can be identified and used
effectively. On the other hand, the oncology community is well aware that for many
tumor types, systemic treatment is unsatisfactory. The motivation to develop better
therapy is therefore as powerful as ever. With the increased understanding of the
malignant process due to recent and anticipated advances in molecular biology and
biochemical pharmacology, we have every reason to expect that the development of
new agents will proceed along increasingly rational lines.
The process of new agent development is often divided into preclinical and clinical
components. Although this division is operationally useful, continual interplay exists
between these arenas. Evidence of synergy or the effectiveness of combined modality
approaches in experimental models, for example, has provided the major motivation for
a large number of clinical trials. The converse is also true; clinical observations have
also given rise to new lines of basic investigation.
Historically, the NCI has been one of the most important effectors in the discovery and
development of new anticancer agents. NCI's prominent role in new cancer agent
development has no parallel elsewhere in developmental pharmacology. The justification
for such intensive involvement of a Government agency in research and development is
clear: significant improvement in cancer treatment is in the public interest. NCI is the
largest clinical trials sponsor focused on cancer treatment and diagnosis, and currently
has a significant number of new agents in various stages of clinical testing or preclinical
development.
As part of this massive effort, NCI funds a clinical trials network that includes
Cooperative Groups and Consortia, new agent development contractors, and
investigators at Cancer Centers and University hospitals and Specialized Programs of
Research Excellence (SPOREs). More than 11,000 investigators from approximately
3,000 institutions participate in this effort.
In the United States, clinical research with investigational agents is carefully regulated.
The regulatory authority for assuring public safety in matters relating to investigational
Section 2 - Investigator’s Manual 2009 2