The pharmaceutical industry learned during the height of the pandemic last year that taking clinical trials virtual could keep R&D going through an unprecedented disruption. Now, many companies plan to tap that newly acquired tech to take on an old problem: diversity in clinical trials.
Just 8% of patients who participated in clinical trials to support the medicines approved last year by the FDA were Black or African American. Only 6% were Asian. Eleven percent were Hispanic, and just 30% were 65 or older. This comes from the FDA’s Drug Trials Snapshot data for 2020, which recorded trial demographics from all 53 medicines approved through year-end.
Forced into a virtual world
Clinical trials are the lifeblood of any pharmaceutical company or biotech. What happens when a pandemic sends the world into lockdown? Fierce Biotech investigates
|How pharma R&D bounced back from a once-in-a-lifetime pandemic|
|Can the tech that kept pharma R&D going through the pandemic tackle trial diversity, too?|
|Novartis brings trials to the patient|
|UCB gets out of the driver’s seat|
|J&J's Janssen offers à la carte flexibility|
|At Medidata, things got real quiet|
|Takeda hunts for COVID's silver linings|
Pharmaceutical companies have long known that drugs can behave differently in people of different ethnicities. It’s also well documented that some communities are disproportionately affected by certain diseases—COVID-19 being one of them.
But the industry is not owed participation by underserved communities. Decentralized clinical trials, telemedicine and home health visits—all employed by pharmas big and small during the pandemic to keep research going—will help, but the industry has much more work to do.
“It's not just as simple as saying, OK, we're going to have a way to make it easier for minority patients to participate in the study by allowing them to get their protocol evaluations done locally,” said Janssen’s Craig Tendler, M.D., vice president of clinical development and global medical affairs in oncology at the Johnson & Johnson unit. “I mean, that's part of it. But, to be frank, it's a small part, and you have to do a lot more.”
Greg Rotz, U.S. pharmaceuticals and life sciences advisory leader for consulting firm PwC, boils the diversity in clinical trials issue down to three pillars: awareness, access and trust.
“Decentralized trials will help, but it will not solve the diversity, equity, inclusion challenge in clinical trials,” he said.
Hesitance from the Black community is completely justified because of the dark history of research on people of color, said Patrice Matchaba, president of the Novartis US Foundation and head of U.S. corporate responsibility. He cites the Tuskeegee syphilis studies between 1932 and 1972, the use of Henrietta Lacks’ stem cells for research without permission and the so-called “Father of Gynecology” J. Marion Sims’ use of African women as test subjects without anesthesia.
COVID-19 underscored that, to this day, the healthcare system is failing underserved communities and people of color. According to the Centers for Disease Control and Prevention, people from racial and ethnic minority groups are dying from the respiratory disease at younger ages, likely because they tend to develop chronic medical conditions earlier thanks to longstanding systemic health and social inequities.
“We saw them blaming the community for not participating in clinical trials, ignoring the background to that,” Matchaba said of the research that helped get a COVID-19 vaccine into arms in record time in 2020.
To overcome hesitancy, patients in underserved populations and regions have to know that their participation “is going to be handled with care," Rotz said.
"The royal we"
Another key catalyst in 2020 was the death of George Floyd and the Black Lives Matter protests that followed, renewing calls for social justice and policing reforms, Rotz said. Many companies pledged that they would do better in the wake of the movement. Pharma was no different, and diversity in clinical trials is an issue the industry is uniquely equipped to address.
And COVID-19, for better or worse, put a spotlight on the issue.
"What I've seen over the last year at the pharma level, but also the internal level, is a consistent increased focus on diversity," said Takeda's Jeremy Chadwick, head of the company's Global Development Office.
UCB’s head of site engagement, Kim Doggett, said the pandemic helped get more of the right tech into the right hands. She is grateful to see even more companies stepping up now that their eyes have been opened by COVID.
“My company and other companies have been working at this for a very long time,” she said. “This is not new to us. It's just more visible, which we're super happy about, OK? We will take press however we can get it.”
All the technology in the world can’t make a person who doesn’t trust the medical system participate in a clinical trial, Tendler said. And the needle has not significantly moved on that part of the problem since the pandemic began. People of color who have been hesitant to get COVID-19 shots frequently cite distrust as a reason.
“It's a multifactorial problem, but certainly, doing things like providing travel assistance and covering out-of-pocket costs, all of these are things that we're implementing and certainly will help begin to bridge this injustice and this gap in terms of minority representation in oncology clinical trials,” Tendler said.
So many factors weigh into how a patient’s treatment can differ from the rigors of a study environment, but it all comes back to the same problem: Patients participating in clinical trials are overwhelmingly white. So R&D is designed with that in mind from start to finish.
“Shame on the royal we of the life sciences industry to then discover that things aren't the same safety and efficacy when they're used in different practice contexts, or on different people who might have different levels of access to care than people who live in a city, who can walk to an academic medical center down the street and afford to go,” said Glen de Vries, co-CEO of clinical trials software firm Medidata.
"What's the big deal?"
Pharmaceutical companies like Janssen have to work on building trust at the community level, Tendler said. They have to bring on and provide support to research sites in more diverse areas that have historically been unable to host trials because they didn’t have the resources.
And the people doing that work need to look like the patients they serve, according to Badhri Srinivasan, head of global development operations for Novartis.
Inspired by the Black Lives Matter protests and the issues coming to a head because of COVID, the company recently launched an ambitious project to fund historically Black colleges and universities and build up the next generation of clinical investigators of color.
“It became clear that the impact of COVID on different races is not based on genes. It's based on their front-line workers,” Matchaba said when the program launched in July.
Pharma companies also have to understand the nuances in every disease to ensure trial populations are representative of the groups who will ultimately receive a medicine, Srinivasan said. Just as COVID disproportionately affects Black communities, other diseases can have different impacts on certain ethnicities, genders or other groups.
“The intent of any trial that we run is to be representative of the population that it is intended to serve. However, that also means we need to strike a balance–not including diversity for diversity’s sake,” Srinivasan said. “Our goal is to ensure that we understand how those patients who are most likely to be treated for a disease or condition will respond to a medicine, enabling patients to achieve their best possible outcomes.”
At UCB, the goal is the same. Doggett and her team are working through a community engagement partner pilot program right now that seeks to define what diversity means for each individual trial to ensure they find the right patients. So far, Doggett said they have been “amazingly surprised” at how open the healthcare workers are to helping. Often, they hear back, “’No one has ever asked me this question. Of course we can help you.’”
Takeda always had a focus on diversity, Chadwick said, but the company recently hired a person who is 100% focused on examining the issue in clinical research.
“And you might say, ‘What's the big deal, one person?’ Well, the big deal is that in the past, it was somebody who had a full-time job and was spending four hours doing the best they could,” Chadwick said.
The new hire has allowed Takeda to dig deeper into some of the key issues that prevent some communities from being eligible to participate in research. This includes ensuring informed consent documentation is available in the correct language, understanding demographics of the indications in testing and tailoring trials for the populations that will be most affected by a given condition, Chadwick said.
"We're thinking of ways to make sure that patients from different backgrounds feel comfortable coming into the study," he said.
Janssen is now requiring every new study team to submit diversity strategy plans that are reviewed before the trial can go forward, Tendler said. New study starts in the U.S. must be representative of the population that will ultimately use the drug for the future label indication.
Companies are increasingly making more targeted therapies, too, specifically in oncology where obscure mutations in small populations have become a pillar of new drug development. Clinical trials often fail, Medidata's de Vries pointed out. And while failure can be valuable to inform future research, companies also lose valuable time and money. But if diversity were considered from the get go, researchers may be able to pick out different populations that might see a greater effect from a therapy.
"The more inclusive we make clinical trials, I actually think the better science we're doing," de Vries said. "The better science we're doing, the more likely you are to come up with the right drugs."