Pfizer Uses EHR Data to Support Expanded Indication for Breast Cancer Drug

Regulatory NewsRegulatory News | 05 April 2019 |  By 

In a move that may become more common as the US Food and Drug Administration’s (FDA) comfort with real-world data (RWD) increases, FDA on Thursday approved a new indication for Pfizer’s Ibrance (palbociclib) in combination with an aromatase inhibitor or fulvestrant to include men with certain types of metastatic breast cancer.

The approval is based on RWD from electronic health records and postmarketing reports of Ibrance in male patients sourced from three databases: IQVIA Insurance database, Flatiron Health Breast Cancer database and the Pfizer global safety database, according to Pfizer.

The new label reads: “Based on limited data from postmarketing reports and electronic health records, the safety profile for men treated with IBRANCE is consistent with the safety profile in women treated with IBRANCE.”

Vinay Prasad, Associate Professor of Medicine at Oregon Health and Science University, told Focus: “I am glad the FDA has concluded that Ibrance has the same side effects in men and women.  But, I would be much more curious [to know] what they think about the fact that the drug has never shown a survival benefit in women.

“I find it interesting that long term follow-up of palbociclib has failed to show a survival benefit in randomized studies, but the next regulatory action was expanding approval to men,” he added.

Aaron Kesselheim, director of Harvard Medical School’s Program on Regulation, Therapeutics and Law, explained to Focus that based on the press release it seems oncologists “would have probably been using this drug in male patients anyway, and it is likely that payors would have covered it, whether or not the FDA took this additional step.

“Still, it is reassuring to hear that, according to the press release, the FDA’s expert independent review of the observational data submitted by the manufacturer show that male breast cancer patients treated with this drug appear to experience similar observed safety outcomes and tumor responses to female patients. However, as with all approvals based on surrogate measures, and all approvals of rare disease drugs which are inherently based on small numbers of safety events, it will be important to continue to follow the outcomes from this class of patients for longer term outcomes and in larger datasets to confirm these findings,” he added.

It remains to be seen whether this expanded indication for Ibrance signals the beginning of a shift to using more real-world evidence (RWE) to alter how existing drugs are used, such as with a change in dose, dose regimen, or route of administration, new population or adding comparative effectiveness or safety information.

Back in 2016, CDER Director Janet Woodcock explained how rare it is to use RWD to expand an indication. This has previously happened, Woodcock said, but right now it's the exception rather than the rule. "Have we ever done this for an indication? Yes, but it's very rare, even for rare diseases."

In 2018, following the enactment of the 21st Century Cures Act, FDA released its RWE framework, explaining how FDA “must evaluate the potential use of RWD to generate RWE of product effectiveness to help support approval of new indications for drugs approved under FD&C Act Section 505(c) or to help to support or satisfy postapproval study requirements. FDA’s RWE Program will also apply to biological products licensed under section 351 of the Public Health Service Act.”

In the premarket space in oncology, outgoing FDA Commissioner Scott Gottlieb last year noted that FDA currently has new drug applications under review where RWD and RWE are “helping to inform our ongoing evaluation as one component of the total complement of information that we’re evaluating.”


© 2023 Regulatory Affairs Professionals Society.

Discover more of what matters to you

No taxonomy