A key focus for new National Institutes of Health (NIH) Director Monica Bertagnolli is spearheading innovative way to get more patients into in clinical trials to improve evidence generation, she said in recent remarks, including by getting providers more involved in trial enrollment. Her remarks are in line with calls by the non-profit Reagan-Udall Foundation for a health care-wide approach to generating data that involves FDA, NIH, CMS, private payers and clinicians.
Bertagnolli spoke Tuesday (Nov. 14) at the Friends of Cancer Research annual meeting along with FDA Commissioner Robert Califf.
“We have to finally take a leap to design our evidence generation in a way that really does do much more to help that doctor or that patient make those decisions together,” Bertagnolli said. Clinicians constantly work with incomplete data during trials and there is a need for more robust evidence generation, she added.
Government-funded clinical trials have faced a significant challenge in enrollment rates, Bertagnolli noted, with a decade-long trend of lower patient participation compared to pharmaceutical industry-sponsored trials. She identified this as a critical area where NIH is lagging behind private companies. To address this issue, Bertagnolli said government-funded trials should focus on research questions “that are not of central interest to pharma.”
Califf said the medical system needs to increase clinical trial diversity by creating a “global evidence generation system” that extends beyond the United States.
Califf pointed out that the United States might not be the only place to enroll patients in clinical trials. “It would be wrong to enroll 100% of people in the U.S., (which is) 4% of the world’s population,” he said.
“We need to come to grips with the fact that although pharma is doing a great job of getting their trials done, U.S. may not be the preferred place to enroll when you really talk to the people,” Califf said.
FDA officials also have pointed to a lack of diversity in clinical trials as a safety concern for some products. The agency recently raised safety issues with a new sickle cell gene therapy, questioning whether clinical studies adequately reflect the genetic diversity of patients with sickle cell disease. Despite the condition affecting around 100,000 Americans, with a majority being Black, FDA noted deficiencies in genetic studies, databases, and reference genomes, highlighting a lack of representation for Black Americans. The agency also highlighted limitations in the 1,000 Genome Project Database, which, despite comprising 2,504 individuals, included only 61 from the southwestern United States.
Studies have shown that that cancer clinical trials often underrepresent patient populations with historically lower financial resources, including uninsured and minority patients, while those with higher socioeconomic status appear to enroll more frequently, according to a 2016 study published in JAMA Oncology.
“We could do so much better with better evidence, and I want to get the kind of evidence that helps those doctors’ patients to make the decisions together,” said Bertagnolli. — Maaisha Osman (firstname.lastname@example.org)