Clinical trials are used to evaluate the safety and e;cacy
of a potential medical product before it can be approved
by the FDA and used more broadly as part of standard
care. All clinical trials are reviewed and approved by the
FDA and institutional review boards before they can begin
and are monitored throughout their duration. ;ere are
several types of cancer clinical trials, including treatment
trials, prevention trials, screening trials, and supportive
or palliative care trials, each designed to answer di;erent
research questions. In general, they add an investigational
intervention to the standard of patient care. ;e following
discussion focuses on treatment trials, which are used to
evaluate potential new anticancer therapeutics.
Until recently, treatment clinical trials have been typically
done in three successive phases, each with an increasing
number of patients (see sidebar on Clinical Trial Phases).
One of the many advances in clinical research has been
the advent of new ways of conducting and regulating
clinical trials, which can eliminate the need for large, long
multiphase trials (see below).
Conventionally, many adult clinical trials are conducted
a;er patients have received prior treatments, like surgery,
radiation, or other therapeutics, which have already been
tested in prior clinical trials. In some cases, a clinical trial
may be designed to test a presurgery treatment, which is
referred to as a neoadjuvant treatment. Recently, such a
trial was the basis upon which the FDA approved a new use
for a previously approved breast cancer therapeutic called
pertuzumab (Perjeta) (see New Path to Approving Breast
Cancer Therapeutics, p. 64).