- Pfizer on Thursday secured an expanded U.S. approval for its breast cancer drug Ibrance to men, relying not on clinical study but rather on data from electronic health records and postmarketing reports detailing real-world use of the therapy.
- Breast cancer in men is rare, accounting for less than 1% of all cases of the tumor type. As a result, clinical studies testing breast cancer drugs often exclude men, as was the case with PALOMA-2 and PALOMA-3, the two Phase 3 trials supporting Ibrance's approval.
- Under the now-departed Scott Gottlieb, the Food and Drug Administration sought to accelerate use of real-world data in its regulatory review of medicines. In December, the agency released fresh plans which, among other priorities, highlighted the potential of real-world data in supporting label expansions.
The new approval for Ibrance (palbociclib) won't move the needle much for Pfizer commercially, but the OK adds to a string of regulatory successes the pharma has had in front of the FDA.
Last fall, the FDA cleared four Pfizer oncology drugs, including two for certain types of lung cancer and one for breast cancer.
That spate of approvals caps something of a turnaround for Pfizer in a therapeutic field that has fast become one of the most active in all of pharma. As a recent report from The Wall Street Journal noted, Pfizer expects its cancer drugs — led by Ibrance — to outsell its heart and primary care medicines for the first time in 2019.
Last year, sales of Ibrance jumped 32% to total $4.1 billion, trailing only Pfizer's pneumococcal vaccine Prevnar 13 and nerve pain drug Lyrica (pregabalin).
Ibrance's commercial success is rooted in its approvals for hormone-positive, HER2 negative metastatic breast cancer. Until this week, first-line use of the drug was limited only to postmenopausal women.
Now, Ibrance is approved for the estimated 2,670 men who will be diagnosed with breast cancer this year.
To secure the FDA's nod, Pfizer submitted an analysis of real-world data from EHRs as well as reports from male patients treated with Ibrance sourced from databases including Flatiron Health's, Iqvia's and Pfizer.
The FDA has used real-world data in reviewing drugs before, but frequently such evidence has been used to construct a "historical control" to compare results from single-arm studies of a drug. That was the case with Amgen's Blincyto (blinatumomab), first approved in 2014 for a type of acute lymphoblastic leukemia.
The promise of using real-world data to create virtual control arms was part of Roche's rationale for buying Flatiron in February 2018 for $1.9 billion.
Despite that acquisition, Flatiron has continued to operate as a seaprate legal entity and to work with external partners like Pfizer.