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Clinical Trials Headed for Revamp With Califf as FDA Head Pick (1)

Nov. 18, 2021, 6:10 PMUpdated: Nov. 18, 2021, 9:37 PM

President Joe Biden’s pick to lead the FDA could jolt the clinical trials enterprise toward long-sought changes aimed at easing research participation and improving the quality of evidence used to offer better patient care.

Robert M. Califf, if confirmed by the Senate, would return to head the agency during a public health crisis that has exacerbated the nation’s shortcomings, including those within a fragmented clinical trials system where just a handful of studies have yielded actionable results. Califf, senior adviser for Verily Life Sciences and Google Health, has been a vocal advocate for modernizing that system.

“There’s been a lot of self reflection by the clinical trials enterprise, especially over the last two years with Covid-19,” Esther Krofah, executive director of FasterCures and the Center for Public Health at the Milken Institute, said in an interview. “Rob has been central in a lot of these conversations.”

The system for conducting clinical trials in the U.S. has long received criticism from leaders at the Food and Drug Administration and others for being clunky, costly, and failing to provide the answers needed that lead to better patient outcomes. Recruitment also remains a pervasive challenge in research: A paper published last year found 55% of trials in a database ended primarily due to a low accrual rate, and more than 80% of trials globally fail to enroll on time.

“The discovery engines are really humming,” but how we conduct clinical trials is still “so circa 1995,” Amy Abernethy, a former second-in-command at the FDA who’s now at Verily. She said she left the agency this year to help solve these problems.

Califf has called for moving away from smaller studies that won’t actually affect patient care, making trials more diverse and accessible, and leveraging real-world data to bolster patient outcomes.

The White House Nov. 12 nominated Califf to lead the FDA for the second time, after serving as commissioner during the final year of the Obama administration. If confirmed, he would bring an experienced and steady hand to an agency that has seen five confirmed or acting commissioners since the beginning of 2019.

Robert M. Califf
Photo by Win McNamee/Getty Images

Evidence Generation

Improving the generation of evidence used to make regulatory decisions has been a constant theme of Califf’s work before and after his time at the FDA, said Adrian Hernandez, executive director of the Duke Clinical Research Institute—a job Califf once had as the founding director of the institute.

“Clinical trials is a pretty key part of that,” Hernandez said. “And one of the themes that he’s really pushed forward is: How do we get greater participation in clinical trials?”

Califf’s priorities mirror policy goals agency veteran and acting FDA Commissioner Janet Woodcock has supported, such as to build a clinical trials network into community practices, where most patients receive their health care. Such a system would allow the quick randomization of patients to generate the answers doctors need to make treatment decisions in a fast-evolving public health crisis.

“In some ways, we had taken for granted that academic research sites are primarily where research studies are going to be conducted,” Krofah said. “Then we saw this massive public health emergency, where so many people who were getting infected—and could provide data to identify what treatments are effective and safe—could not even participate in those trials” because the closest academic center was hours away.

Making clinical trials more accessible could also make the study populations more diverse, which has been a well recognized challenge. “Just like there are food deserts, or health-care deserts, there are clinical trial deserts. By having a broader system that’s easier to navigate or participate in for patients and clinicians, then hopefully you could close those clinical trial deserts,” Hernandez said.

Califf, who declined to comment for this article, wrote in a tweet that too many small trials are asking similar questions and not enough trials are providing reliable answers to them. He also called for posting clinical trial results more quickly.

Ellen V. Sigal, founder and chairperson of Friends of Cancer Research, said Califf’s priorities around making clinical trials smarter and more diverse have been clear for a long time.

“I don’t see that is changing. I do think this year with Covid, we’ve learned a lot, and I think he will take on other issues that are going be very, very important,” she said.

Robust Data

Krofah said she expects Califf to engage with center directors at the FDA to understand where things stand on accurate data collection and ensure the agency is capturing information that is relevant to the various subpopulations that are participating in the clinical trials.

“Rob will tell you this all the time, he wants to make sure that we are collecting evidence that is robust,” said Krofah, who served as co-chair of a four-part National Academies workshop on envisioning a transformed clinical trials enterprise for 2030. Califf laid a lot of the groundwork to incorporate real-world evidence—derived from electronic health records, medical claims, and other sources—back in 2016, and much of his current work involves the incorporation of digital data in health research.

The FDA issued guidance in September on how to use real-world data to support evidence generation.

Kalah Auchincloss, who was Califf’s deputy chief of staff during his first tour at the FDA, said digital health and real-world evidence will likely be priorities for the new commissioner, who previously served between 2016 and 2017.

“He knew he was only going to be there for a year, obviously. But he had a lot of things that he would have loved to have accomplished had he been there longer,” Auchincloss, an executive vice president at consulting firm Greenleaf Health, said.

“He’s probably pretty excited about the opportunity to continue where he started four years ago.”

(Updated to include comments from Amy Abernethy in the fifth paragraph.)

To contact the reporter on this story: Jeannie Baumann in Washington at jbaumann@bloombergindustry.com

To contact the editor responsible for this story: Alexis Kramer at akramer@bloomberglaw.com

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