Research Shows that Oncologists Respond Promptly to New FDA Safety Guidelines

Penn researchers have recently found that when new safety data emerges regarding cancer therapy, oncologists are very quick to incorporate new guidelines into their practice. This new study from the Abramson Cancer Center at the University of Pennsylvania found that in the six months after the U.S. Food and Drug Administration (FDA) restricted two immunotherapies, usage dropped by roughly 50% in oncologists. The results of this study were published on September 24 in JAMA.

With the FDA’s accelerated approval (AA) program now in place, new treatments and guidelines are emerging rapidly. This research from Penn provides evidence that oncologists are both receptive of this new information and prompt in their implementation of such knowledge.

There are mixed opinions regarding the FDA’s AA program, with some feeling that it brings beneficial treatments to the patient faster and others criticizing it for prematurely bringing drugs to the market. These critics argue that new therapies can skip the phase III clinical trial phase, which is a key step in drug development research. With these conflicting views of the FDA’s new fast-track program, these Penn researchers aimed to evaluate how oncologists are adapting to this accelerated pace in their specialty.

“We felt it was important to evaluate what the data says about this program, and our findings show oncologists are quick to respond to emerging safety data for drugs approved through this process,” said senior author Ronac Mamtani, MD, MSCE, an assistant professor of Hematology-Oncology in the Perelman School of Medicine at the University of Pennsylvania.

Background of the Penn Study

This work analyzed the usage rates of two different immunotherapies that are approved for use in bladder cancer patients who do not qualify for the standard cisplatin-based treatment. These drugs, the PD-1 inhibitor pembrolizumab and the PD-L1 inhibitor atezolizumab, have been approved by the FDA since 2017 based on phase II clinical trials. With data from current phase III trials showing decreased patient survival on these drugs when compared to those on the standard cisplatin therapy, the FDA restricted the label indications of these drugs in June of 2018.

It was unique that the FDA had restricted these drugs’ indications after approving them through the AA program, therefore Mamtani and colleagues performed this data review to see how quickly this label-restriction would be implemented in the clinical setting.

Using records from over 280 U.S. oncology clinics in the Flatiron Health Database, the researchers examined the treatments of 1,965 advanced bladder cancer patients. Between May 2018 and January 2019, the rate of immunotherapy usage in these patients decreased from 51.9% to 30.3% per 100 patients, and the rate of chemotherapy usage increased from 37% to 60.6% per 100 patients. PD-L1 testing rates also increased from 9.3% to 21.2% per 100 patients.

“Given the rapid expansion of approvals for immunotherapies, understanding how oncologists react to post-approval safety concerns is crucial, and our study suggests uptake of these changing recommendations can occur very quickly,” said lead author Ravi B. Parikh, MD, MPP, an instructor in Medical Ethics and Health Policy at the University of Pennsylvania.

Although this work advocates for the oncologists’ ability to adapt in a rapidly changing specialty, the Penn researchers warn that other factors could be underlying these observed trends. They conclude that further research is warranted to analyze how these label changes affect patient care.

Author Affiliations

Further contributions to this publication came from co-lead author Blythe Adamson, PhD, a senior qualitative scientist at Flatiron Health, and co-senior author Aaron Cohen, MD, MSCE, associate medical director of Flatiron Health.