THE CLINICAL CANCER
The hard work of individuals throughout the biomedical
research cycle constantly powers the translation of
discoveries to new medical products for cancer prevention,
detection, diagnosis, treatment, and care (see Figure 9,
In the 12 months spanning Aug. 1, 2015 to July 31, 2016,
the FDA approved 18 new medical products— 13 new
anticancer therapeutics, one new blood-based companion
diagnostic test, one new cancer screening test, two new
diagnostic imaging agents, and a new medical device
(see Table 1, p. 10). During this period, the FDA also
approved ne w uses for 11 previously approved anticancer
therapeutics, including obinutuzumab (Gazyva).
In February 2016, the FDA approved obinutuzumab for
use in combination with the cytotoxic chemotherapeutic
bendamustine to treat certain patients with follicular
lymphoma, which is the second-most common form of non-Hodgkin lymphoma diagnosed in the United States. This
approval followed its November 2013 approval for treating
chronic lymphocytic leukemia (CLL), which was highlighted
in the AACR Cancer Progress Report 2014 ( 1). The approval
Accelerated approval. Accelerated approval is based on assessing the effect
of a therapeutic at an earlier stage by using a surrogate endpoint. Any therapeutic
approved in this way must undergo additional testing following approval to verify
that it provides clinical benefit. Atezolizumab (Tecentriq) for the treatment of
advanced urothelial carcinoma (the most common form of bladder cancer)
was approved under this pathway in May 2016 (see p. 87).
Fast track. This designation is given to therapeutics that fill an unmet medical need
and can be granted solely on the basis of preclinical data or data from nonhuman
studies. Fast track applications may be evaluated through a “rolling” or continual review
procedure, rather than waiting until study completion. Nivolumab (Opdivo) for the
treatment of advanced renal cell carcinoma (the most common form of kidney cancer)
was approved through fast track in November 2015 (see p. 83).
Breakthrough therapy. A therapeutic that shows substantial improvement
over available treatment in early clinical studies can receive breakthrough therapy
designation, making it eligible for all features of fast track designation (see above) and
additional guidance from the FDA throughout the drug development process. One
example of a therapeutic that was FDA approved, in April 2016, after receiving
a breakthrough therapy designation is venetoclax (Venclexta) for the treatment
of chronic lymphocytic leukemia (see p. 75).
Priority review. Therapeutics that have the potential to significantly improve safety
or effectiveness may be granted priority review after all clinical trials are completed.
This allows the therapeutic to be assessed within 6 months as opposed to the standard
10 months. Alectinib (Alecensa) was granted priority review and approved in December
2015 for the treatment of certain patients with lung cancer (see p. 70).
FDA’S EXPEDITED REVIEW STRATEGIES
The U.S. Food and Drug Administration (FDA) has developed four evidence-based strategies
to expedite assessment of therapeutics for life-threatening diseases like cancer.
Adapted from ( 1)