FDA Issues New Guidance For Neoadjuvant Breast Cancer Therapies

Regulatory Affairs Professionals Society (RAPS) reports that the FDA has issued a guidance allowing the agency to consider pathological complete response (pCr) as a surrogate endpoint in phase 3 clinical trials for high-risk breast cancer neoadjuvant therapies. The FDA hopes that this new guidance may help accelerate approval for new and promising breast cancer treatments.

Current breast cancer treatments will consider using neoadjuvant therapies, usually a pre-operative round of drugs and chemotherapy, for multiple reasons. The primary purpose is to make an inoperable tumor operable or to improve surgical outcomes, but sometimes it is used to circumvent the need for a complete mastectomy.

Though oncologists are still debating a precise definition, neoadjuvant therapies are generally thought to achieve pCr when all visible vestiges of cancer or at least invasive cancer have been removed.

New studies suggest a strong correlation between pCr and long-term survival prognosis, though researchers are still collecting data to prove the two are intrinsically related.

In 2013, the FDA issued a guidance which would accelerate treatments for serious conditions by introducing four new programs: fast track designation, breakthrough therapy designation, accelerated approval, and priority review.

The accelerated approval designation outlines certain scenarios in which the FDA would consider a surrogate endpoint in place of complete clinical analysis.

The guidance stipulates that if a drug “treats a serious condition and generally provides a meaningful advantage over available therapies and demonstrates an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit,” the drug may be considered for accelerated approval.

The FDA’s newest guidance authorizes the use of pCr as a surrogate endpoint that can qualify neoadjuvant breast cancer treatments for clinical use.

RAPS explains that this policy differs from fast-track or priority review programs, which still require data on a primary endpoint before approval is issued. With most diseases, however, once the disease is eliminated from the body, the treatment is deemed effective and the patient is cured.

Because cancer can and does reoccur, clinical trials may require years or decades to determine the efficacy of a new drug. Many patients, especially those with particularly aggressive forms of cancer, do not have the time to wait, thus necessitating a more tailored approach to FDA approval.