FDA to shake up guidance for early breast cancer treatment

Ellie Watmuff
WVoN co-editor 

The Food and Drug Administration (FDA) in the US, issued a directive this week to allow breakthrough treatments to be trialed on women suffering from early-stage, aggressive breast cancer.

Under the new guidance women suffering with early-stage but hard-hitting form of the disease could trial drugs for several months, even prior to undergoing surgery.

This is significant as it could grant more immediate access to potential life-saving drugs. Currently women diagnosed with early stage cancer have a lengthy wait for innovative treatments, despite having the best chance of recovery.

“We’re looking at introducing drugs into a very early stage of breast cancer, where a patient has a primary tumor and the chemotherapy is given before surgery, Dr Richard Pazdur, director of the FDA’s cancer drug office, told an American Society of Clinical Oncology meeting in Chicago.

“The aim of this therapy would be to cure the woman of breast cancer”.

The current process requires that drugs be trialed first on patients whose cases are advanced, where the potential for treatment to extend life outweighs any detrimental side-effects.

There are concerns that this new guidance could expose women to potentially harmful drugs without sufficient proof to support their efficacy.

Pazdur explained that the changes “should be for patients that are most likely to benefit and also those that are at highest risk of having a recurrence of the disease”.

In order to minimise the risk from these drugs, the FDA´s guidance suggests only women suffering from a particularly aggressive form of breast cancer called triple-negative breast cancer, particularly prevalent in young women, have access to piloted drugs.

Trials would be structured by an adaptive design approach, where researchers derive their results from patient response.

Women would be treated with either chemotherapy or chemotherapy alongside an experimental drug for several months before surgery. Researchers would then compare response rates across both groups.

If the drug cures significantly more patients, it would be subject to a provisional type of approval, which with further, long-term monitoring could lead to full approval.

Dr George Sledge, co-director of the breast cancer program at the Indiana University Simon Cancer Center said that although the new approach is not a cure all answer, it’s a step in the right direction:

“It recognizes that we want to try to move drugs up front into an early disease setting as soon as possible – not wait until the patient is beat up and out of chances and out of hope before we throw a drug at [them] and hope it does something.”