At White House forum, Bertagnolli, Califf, Wegrzyn call for greater innovation in clinical trials

Leaders of three health agencies presented new initiatives focused on patient-centered research, diversity in clinical trial enrollment, and innovation in clinical research. 

The initiatives discussed at the White House Clinical Trials Forum on June 26 included: 

• The NIH Communities Advancing Research Equity for Health, abbreviated as CARE for Health, 

• The FDA draft guidance for industry titled “Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies,”

• The ARPA-H Advanced Analysis for Precision Cancer Therapy, or ADAPT, a program developed as part of the Cancer Moonshot.

Announced earlier this month, Communities Advancing Research Equity for Health is an NIH pilot program that will work with local communities experiencing exceptionally poor health outcomes to figure out which clinical trials would be most meaningful to them. 

“We are seeing really bad outcomes, but the bad outcomes are in certain particular segments of our population,” NIH Director Monica Bertagnolli said at the White House meeting. “And they are exactly in the segments of our population that don’t have access to care and really don’t have access to clinical trials.”

CARE for Health will support research in primary care practices, and then compile a list of dozens of potential research projects that could be conducted in the community. This allows doctors and patients to choose what research they think would benefit them the most, Bertagnolli said.  

“We don’t parachute in,” Bertagnolli said. “We come for the long duration—we build structure that is enduring, which means we also have to build it within care that is also enduring.” 

Ultimately, the goal is to form a national primary care research network. NIH is investing about $30 million into CARE for Health over fiscal years 2024 and 2025. Initial awards that will fund organizations serving rural communities are expected to be made this fall.

FDA, too, has been pushing for greater diversity in clinical trials, said Robert Califf, the regulatory agency’s commissioner. Although NIH’s clinical trials are meeting “first-order” benchmarks in terms of representing sex, race, and ethnicity in the U.S. population, FDA’s priorities are a bit different, he said. 

“We have a slightly different set of issues because we’re regulating a global industry, which obviously has a strong base in the U.S., but most of the 8 billion people in the world don’t live in the U.S.,” Califf said at the meeting. 

To some extent, FDA must persuade industry to include appropriate representation across clinical trial participants, he said.

Califf said the agency’s updated draft guidance on “Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies,” released on June 26, will help sponsors submit Diversity Action Plans to support phase III clinical trials. 

“We want people to tell us, ‘How are they planning to get the trial done and meet the criteria for engaging and enrolling a diverse group of participants?’” Califf said. 

FDA wants to go beyond those “first-order” benchmarks for diversity, Califf said. “There are many other elements of diversity in this country—urban/rural has become a huge factor.” Socioeconomic status is another, he said. 

“Access to clinical trials is something that everyone should be entitled to. But 90% of what gets introduced into clinical trials that FDA regulates turn out not to make it to market,” Califf said. “We have to be very respectful of the people involved, but also make sure they understand what the conditions are—that we’re testing something; we don’t know how effective it is at this point.”

ARPA-H Director Renee Wegrzyn used the recently-launched ADAPT program to illustrate the agency’s approach to innovation in clinical research. 

Although ARPA-H does not focus on clinical trials, the funding agency does look at how to remove some of the risk for participants, Wegrzyn said. The ADAPT program, launched in March, examines how clinical trials can adapt with participants as their tumors adapt. 

“You would think that would be a common practice, but it isn’t,” Wegrzyn said. “So, breaking what might seem like an intractable problem down into the pieces—the projects we can invest in, make progress against—is really, I think, what we do really well.” 

ARPA-H’s investments are transactional—the agency only funds a research project for its first few years—so the agency does not build relationships through long-term projects, Wegrzyn said. Instead, “we want to build relationships with clinical trials centers, with patient communities, so that we can have almost a leave-behind infrastructure.”

“We may work with one center for an osteoarthritis trial, but the next time we work with that center might be for a cancer trial—really planting those seeds to create a rooted network that we can use as an agency well into the future,” Wegrzyn said.