Neoadjuvant treatment for cancer is the chemotherapy
or radiotherapy that a patient receives before definitive treatment like
surgery. The addition of a pretreatment to surgery often results in better
outcomes and lowers the chances of recurrence of the cancer following surgery.
A new drug to treat breast cancer is first tried out
in patients with metastatic disease i.e. cancer that has spread to other
organs. It is available for patients with early-stage disease only after years
of clinical trials. The drawback of
this approach is that many patients miss the benefits of these new treatments.
On the other hand, starting these drugs that have not undergone adequate
clinical testing for safety may pose some risks in the form of side effects to
Taking the benefits and the possible disadvantages
of using these chemotherapy drugs early, it has been suggested that these drugs
could probably be tested first in those women with breast cancer who are more
likely to have recurrence of the cancer following usual treatment. This
includes women with high-grade tumors that are negative for estrogen receptor
(ER), progesterone receptor (PR), and human epidermal growth factor receptor 2
(HER2) (these cancers are referred to as triple negative).
For such newer drugs, the manufacturer should be
able to demonstrate that treatment with the new drug has eradicated cancer to
the point where there is no indication of cancer in the breast or lymph nodes;
this is referred to as a 'pathologic complete response'. Researchers suggested
that chemotherapy drugs likely to produce a pathologic complete response should
be granted accelerated approval.
new drug could be added to an existing regimen and its benefits when used with
the current regimen can be first demonstrated. Continued follow up would
provide efficacy and safety data over a prolonged duration. If the patient is
disease free over this period, then full approval may be granted. If the
manufacturer is unable to establish the benefit, the additional indication may
process of accelerated approval would benefit the numerous women who are at a
risk of recurrence of breast cancer following breast cancer surgery.
Pathological Complete Response and Accelerated Drug Approval in Early
Breast Cancer; Tatiana et al; N Engl J Med 2012; 366:2438-2441