NCHR Statement at FDA Advisory Committee Meeting on Tecentriq for Triple Negative Breast Cancer

April 27, 2021

I’m Dr. Diana Zuckerman, president of the National Center for Health Research. Our center is a nonprofit think tank that scrutinizes the safety and effectiveness of medical products, and we don’t accept funding from companies that make those products. I have post-doc training in epidemiology, and was a faculty member and researcher at Vassar, Yale, and Harvard. I’ve also worked at HHS, the U.S. Congress, and the White House.

We urgently need effective treatments for triple negative breast cancer patients. We agree with FDA that a randomized clinical trial that evaluates overall survival is the best way to determine whether the benefits outweigh the risks. That’s important because this drug has substantial risks.

We agree with Genentech that conducting a randomized controlled trial is difficult when a drug is already approved in other countries. But FDA grants accelerated approval with requirements for post-market RCTs to evaluate overall survival to ensure clinically meaningful benefit. How could FDA continue to offer accelerated approval if post-market RCTs can’t be done?

And given the co-pay requirements, aren’t there cancer patients who would welcome free treatments in a clinical trial so that these studies could be conducted appropriately?

What are the study alternatives?  Does it make sense to study patients who relapse within 12 months instead of after 12 months: Don’t they differ?  Isn’t that why they were studied separately?

Can a different study be used instead? NSABP B59 and IMpassion030 both include patients that aren’t PD-L1 status.  Both use taxol, a type of chemo that when used in combination with Tecentriq resulted in lower overall survival for patients in IMpassion131.

Overall, our analysis agrees with FDA that neither an interventional, nonrandomized single-arm trial nor real world study can confirm benefit. Triple Negative Breast Cancer patients deserve proven treatments. These patients were rarely studied decades ago, since “triple negative” was relatively rare among white women.  As we all know, triple negative breast cancer is more common and apparently more lethal in Black women compared to white women. And yet, Black women comprised only 6% of the patients in IMpassion 130 and 5% in Impassion131, the confirmatory study. So the studies done so far are inadequate in addition to not confirming the indication. That is another reason why a new randomized controlled trial that includes a larger percentage of Black women is needed. All patients deserve to know if there are meaningful benefits that outweigh meaningful risks.

We all want to give patients hope. Real hope, not false hope. The FDA wants to help patients get timely access to treatments. But, as a public health agency, FDA’s first priority needs to be evidence. Evidence should be based on the patients that most need the treatment.

In conclusion, we do patients no favors to continue to approve a treatment that is not proven to benefit them and is proven to harm many patients.