FDA’s cancer center is working with patient and research groups on guidance to broaden the number of patients eligible to join clinical trials, officials said Wednesday.
The Oncology Center for Excellence is working with Friends of Cancer Research and the American Society of Clinical Oncology on the guidance, which might be released this year — the latest in a series of steps the center has taken to loosen traditional FDA regulation practices.
Traditional restrictions on enrollment are aimed at assuring that a drug’s effects or failures are measured in a statistically reliable way. The disadvantage is that such trials don’t reflect real-world populations and exclude patients who might benefit, said Wendy Rubinstein, director of personalized medicine at FDA.
“You’re looking for that perfect patient and there aren’t that many around,” she said. “Then you approve for a narrow group and when the drug is approved you don’t see the extent of the benefit.”
“We’re trying to push a new paradigm where the patient is eligible by default and would only be excluded for safety reasons,” said Caroline Schenkel of ASCO.
They spoke at a session marking the third anniversary of FDA’s Oncology Center of Excellence that was a kind of love-fest for Director Richard Pazdur, who used to be targeted with death threats for his conservative drug review policies but is now considered a cutting-edge reformer. FDA views his oncology center a kind of pilot project for overhauling other agency sections.
“My chief premise in my work has been, not to do the right regulatory thing but to do the right patient thing,” said Pazdur, who has been at FDA’s cancer drugs division since 1999.
Cancer drugs accounted for more than a quarter of all new drug approvals in the United States from 2010 to 2018, an increase from about 4 percent in the 1980s, according to a recent study from Tufts University.
Panelists also discussed center projects aimed at streamlining reviews and integrating the work of the drug, biologics and device centers; coordinating reviews with other countries; helping patients get more useful information on side effects; reviewing labels for older drugs; and helping community oncologists get access to clinical trials for their patients.
Patient advocates on the panels heaped praise on Pazdur, who also acts as acting director of the Office of Oncologic Diseases. Andrea Ferris, leader of a lung cancer patient advocacy group, said Pazdur had overseen the transformation of treatment of the disease. “You truly are our hero,” she said.
Ellen Sigal, chairperson and founder of Friends of Cancer Research, asked Pazdur how he’d been able to attract such “wonderful” staff — a point that principal FDA Deputy Amy Abernethy raised in an interview with POLITICO last month.
“My magnetic personality, obviously!” he responded. His also pointed to a recruitment poster for the center that highlights how a single pill can transform many lives.
“Scientists want a career, not a job,” he said, but FDA “does not do a good job” at conveying this.
In the early 2000s, The Wall Street Journal once described Pazdur as “Dr. Death” because of his refusal to approve drugs he considered unsafe or ineffective, despite anger from patient groups that included an occasional threat.
Some have speculated that Pazdur changed his approach after his wife, Mary, got a diagnosis of ovarian cancer in 2012 (she died in 2015). But he has denied this. Congress has loosened FDA rules since the late 1990s, and Pazdur says drugs coming before the agency now are simply better.
Pazdur’s creation of the center of excellence has been widely praised by other figures in the cancer field, although some worry that too much reform could allow dangerous or weak drugs on the market.
“Part of the reason for his success is that he’s never satisfied with the status quo,” Clifford Hudis, CEO of ASCO, told POLITICO. “He’s the catalyst for many stakeholder collaborations with the FDA.”