One of the major challenges with clinical trials is that they don’t necessarily reflect what happens in the real world and instead rely on a subset of the population with inherent biases and limitations. As technology improves, the life sciences industry can begin to lift the veil that keeps those restrictions in place, giving companies the tools to quickly identify a more diverse group of potential participants.
A lack of diversity in trials can also have implications down the road for the regulatory and commercial success of a drug, with millions of dollars at stake, a phenomenon seen clearly in the case of Biogen and Eisai’s Alzheimer’s disease drug Aduhelm.
A few years ago, the idea that clinical trial recruitment could check this box sounded like an insurmountable obstacle. But over the last couple of years, pharma companies have started hiring talent to specifically oversee diversity and inclusion in clinical trials, and investing money and energy into the effort. The FDA has also applied some pressure in this area, most recently with a draft guidance designed to encourage participation among historically underrepresented groups.
The needle is beginning to move in the right direction, says Ariel Katz, co-founder and CEO of the healthcare data and analytics company H1.
“How achievable is (diversity) today? A lot more achievable,” he says. “And in five years, I think it'll be so core to how they think about enrollment of patients and enrollment of (principal investigators) and physicians that it's going to be possible.”
But while progress is being made, finding participants remains a central challenge. That’s where technology comes into play, Katz says.
Lack of diversity, substandard results
Historically, trial participants have been pulled from the same buckets linked to the academic medical centers where trials take place, leaving out members of the broader community.
When drugs or treatments aren’t tested in a group that represents the true patient population, that can skew the results and lead to unexpected or undesirable surprises when the drug hits the market, Katz says.
One size doesn’t fit all when it comes to drug safety and efficacy. Genetics plays a role in how different populations respond to a specific drug — a lack of diversity in the development process might mean the drug doesn’t perform as well as expected or has unforeseen side effects when it rolls out to the broader population, Katz says.
Take Biogen’s ill-fated Alzheimer’s drug, Aduhelm. While many issues plagued the controversial drug, which had significant coverage sidelined by Medicare’s decision not to pay for the treatment outside of approved clinical trials — a lack of diversity in its clinical trials was likely also a factor, Katz says.
According to the Alzheimer’s Impact Movement, Black Americans have nearly double the risk of developing Alzheimer’s when compared with white Americans. Hispanics have about one and a half times the risk. But these groups were sorely underrepresented in Aduhelm’s trials. Less than 1% of participants were Black, and only 3% were Hispanic.
The FDA is now requiring Biogen to complete a more diverse new post-market confirmatory study to provide additional safety and efficacy data, which Katz says will likely add millions to drug development costs. The average post-market central nervous system trial costs $14 million, according to H1.
Aduhelm isn’t an outlier.
“This example is very representative of what you see across the entire industry,” Katz says.
Building a better trial
Every trial population should reflect the patients with that condition, Katz says. Technologies like H1’s global platform can help recruiters find them.
“Today we have about 10 million healthcare professionals on our platform, and we’re used across the whole healthcare ecosystem — from patients finding the right doctor, to a life sciences company finding the right doctor to run a clinical trial, to a payer finding the right doctor to insure and put in their health plan and network,” Katz says.
Locating the data is crucial to zeroing in on the right participants.
“Today, H1 is able to give a pharma company the race and ethnicity of every single patient that a doctor sees,” Katz says. The information is compiled using demographic data, self-reported patient data and information from partnerships with other sources. Recruiters can search by location to identify doctors that may treat the target group of patients.
For example, a search of Boston providers could identify a doctor who treats bladder cancer and has a higher percentage of Black or Hispanic patients. It also allows recruiters to see information about social determinants of health, such as patient income, education, or other factors.
“It lets me look at patient population before I actually reach out,” Katz says.
Having this information can have a tangible effect on recruitment. In some instances, users have seen as much as a 60% increase in the selection of diverse patients and physicians by using the platform, he says.
Keeping participants requires work
But Katz says it’s not enough for researchers to simply identify the right mix of people — organizations also need to ensure that the proper supports are in place to encourage participation and retention. One of the biggest challenges trials today face is building trust with historically underserved groups.
“If you have people that look like you and talk like you and that you can relate to … or you have a doctor that looks like you, or talks like you, it helps. You trust them more,” he says.
Language is another important consideration. H1 also helps to match people with providers that speak their language. Trials haven’t always done a good job accommodating non-English speakers.
Organizations should also look to meet participants where they live by adopting a decentralized trial model, Katz says.
“Walgreens and CVS just started running clinical trials. There’s a Walgreens and CVS in almost all communities,” Katz says. H1 is helping them find investigators to oversee these trials. Bringing trials into a familiar setting rather than making people travel can make research more convenient and appealing to a broader group.
Ultimately, building diversity into a trial requires commitment and investment.
“I would invest more into patient advocacy groups that have diverse patient populations. I would invest more into internal processes and communication and making sure that you’re actually reaching diverse communities, making sure you are finding physicians that see low- and middle-income patients,” Katz says.
Eventually, H1 would like to broaden its tools to include even more detailed information, such as an expertise in LGBTQ-related care, or whether buildings are wheelchair accessible.
“We don’t have that today, but we're working to get that, which we believe is the next generation of what patients need and what sponsors need and what life science companies need when they're thinking about enrolling patients,” Katz says.