A pair of former FDA commissioners debated whether FDA should take the lead on using real-world data and real-world evidence to determine the actual value of drug therapies and in turn ensure the value translates to affordable prices. While one said he believes FDA is uniquely positioned to push widespread use of RWD to help people become healthier, the other contended that multiple components of the health system need to step up if such data is to be used in value-based payment contracts.

The conversation comes almost a week after National Cancer Institute (NCI) officials penned an article for the Journal of the American Medical Association (JAMA), in which they wrote that the institute is focused on “right-sizing” clinical trials to answer questions about the efficacy of new treatments with fewer patients. The institute also is exploring the use of novel endpoints in pragmatic trials and through augmented annotation and aggregation of new and existing trials data to answer clinical questions about a drug without the need for additional trial enrollment. These efforts, the officials wrote, could help reduce the financial pressures associated with clinical trials and in turn reduce drug costs for patients.

During a Monday (Jan. 28) discussion on RWE and value-based payments hosted by the Bipartisan Policy Center, FDA Commissioner Scott Gottlieb announced a number of new initiatives the agency is undertaking to advance RWE, including the potential for RWE to make the U.S. health care system more competitive and efficient as validated outcomes measures based on RWD are incorporated into value-based payment contracts.

During a panel after Gottlieb’s speech, former agency commissioner Robert Califf raised the issue of what he called “financial toxicity,” saying that while outcomes-based studies are needed to determine the true value of therapies, the therapies cannot in turn be priced so high that patients cannot afford them. He added, the health system currently does not have the data needed to make those decisions.

Califf, who is now professor of medicine at the Duke University School of Medicine and an advisor for Google’s Verily Life Sciences division, lobbed a criticism at NCI’s Clinical Trials Program and recently announced efforts.

In a Jan. 24 tweet, Califf wrote he likes the plan but there is a “gaping hole in trials with appropriate methodology to understand how to rationally calculate value (benefits/risks and cost) at a time when financial toxicity is a leading cause of death, esp in rural America.”

The former FDA head reiterated that sentiment on Monday, saying that while he thought the new clinical trials program is “fantastic,” it stops short of addressing financial toxicity.

“Financial toxicity in cancer is killing more people than almost any kind of cancer — people not being able to afford the treatment and therefore not getting it,” he said. “So, unless you start doing outcomes-based studies to find out the real value and set a price at a price that people can afford, where it does great benefit, it should command a great price. Where it doesn’t, it shouldn’t. But we can’t discriminate now because we don’t have the data.”

Califf and another former FDA commissioner, Andrew von Eschenbach, who is now president of Samaritan Health Initiatives, agreed that RWD leading to RWE is a solution to such problems. They disagreed, though, on whether FDA should be the primary lead on advancing RWD and RWE to assess value.

Eschenbach argued that FDA is uniquely positioned to take the lead as it has always been an honest broker and a repository of data. He added the agency simply needs to be more proactive.

“[FDA] may need to be authorized to make more widespread use of that data in directing how things get done. I think it’s been more reactive and maybe needs to be more proactive. Why not FDA?” Eschenbach asked.

While Califf commended Gottlieb for following through on the agency’s RWE framework and other initiatives, he said he finds it odd that FDA is leading the way on the issue and believes other components of the health system need to play a role.

“Let’s be a little rambunctious here. We’re former FDA commissioners, not currently under any administration. So, I agree with you in one sense. We all came to love the FDA because of the scientific rigor that exists there and the honest broker function, which is tremendous. But the FDA deals with medical products, it doesn’t deal with health care, and we need both,” Califf said. “And we need to integrate multiple medical products across a person’s illness. So, we need the other components of the system to step up and play. I love that the FDA is taking the lead, I’m just saying we need other components to step up.”

In an interview with Inside Health Policy, Jeff Allen, president and CEO of Friends of Cancer Research (Friends) noted that FDA’s Sentinel system could be a terrific source of data for RWE, but he added that FDA “is not, nor probably should not be in the role of being the sole generator or aggregator of this data.”

“There’s many other sources of information around clinical experiences, health outcomes,” Allen said. “That may be different hospital settings, different payer organizations …Where FDA can play an important role, and where they have started to move forward in trying to define, is different data standards and a framework for understanding what types of data are included in any given data set and therefore better to define its potential use. Not that FDA should be the sole source of any data. They may just be one contributor or one organization that has access in the postmarket setting.”

Allen added the FDA-approved product label may be one area in which it can be communicated that data in a clinical study or from an RWE set shows that the drug has met the approval standard.

“By having FDA as the independent medical experts defining different data standards … that will help instill confidence in [RWE’s] use,” he said, adding that it not only would play a role in clinical research but also could allow FDA to adjudicate claims made in the postmarket setting.

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