Lifesaving cancer medications. High-tech medical devices. Depression therapies that have the potential to help when others haven’t.
Without clinical trials to test their effectiveness and safety, none of these innovations would have seen the light of day.
Johnson & Johnson sponsors and supports thousands of these trials each year, in more than 40 countries—and is also at the forefront when it comes to improving how those clinical trials are conducted.
Take, for example, the company’s current efforts to get more children’s medications into the research pipeline and cutting-edge “direct-to-patient” (DTP) trials it’s conducting that take studies out of the clinical setting altogether and into people’s homes.
“Patients and consumers are, and always have been, at the center of everything we do,” says Joanne Waldstreicher, M.D., Chief Medical Officer, Johnson & Johnson. “Today, we are operating in an environment where they are increasingly involved, educated and engaged in their own healthcare. To address this evolution, we have systematically changed the way we work to meet their changing needs.”
On the eve of Johnson & Johnson’s second DTP trial kicking off this month, we take a closer look at the many ways the company is putting new ideas to use in clinical trials, all of which revolve around a tenet it has adhered to for over a century—putting people first.
1.
Designing clinical trials with people in mind
Traditionally, people who sign up to participate in a clinical trial don’t have much say over how the study is being conducted—but companies like Johnson & Johnson are working to change that.
“We believe that involving patients in clinical trials before they even start can improve the participant experience and help make it more likely that they’ll stay in a trial for the duration,” explains Katherine Capperella, Global Patient Engagement Leader, Janssen Pharmaceutical Companies of Johnson & Johnson. “This, in turn, may lead to better data collection and accelerate development of therapies for the patients who need them.”
In 2017, for instance, Janssen researchers asked patients to act out going on visits to a clinical trial site for a study on one of its compounds. Through their observations, the researchers realized that providing transportation and shortening visits went a long way toward improving people’s experiences as trial participants.
A year after implementing those changes in the actual study, zero patients had dropped out—a noteworthy improvement, considering the attrition rate for clinical trial participants is generally 18%. The study also won an award for best patient-centric clinical trial at the 2017 CARE Awards, which recognize excellence in the biopharmaceutical industry.
Since then, Janssen has collected feedback from an additional 1,700 trial patients in 19 countries. It also just launched HealthCaring Conversations for Clinical Research, a program that uses a science-based framework for patient-centered conversations to improve the clinical trial experience for both patients and investigators. It’s already active in multiple trials, and will be offered to all Janssen trials globally by the end of the year.
2.
Pioneering the “patient-centric” clinical trial
Travel can often be a deterrent for clinical trial participants, but it’s not the only one. The burden of filling out necessary paper-based questionnaires and assessments can also affect their overall experience.
To make the process of taking part in clinical research easier, Janssen set out to pilot patient-centric solutions that would leverage digital platforms and tools.
“For some trials, data can be collected remotely, so our goal is to build the trial around those patients, enabling them to participate from the comfort of their own home,” explains Dmitri Talantov, Senior Medical Director, Clinical Innovation, Janssen Research & Development.
As part of this, Janssen launched its first DTP trial last year to study how autoimmune diseases develop in high-risk individuals. Patients were identified and recruited online, using databases of insurance claims and lab results. Blood samples for biomarker tests were collected from them at their homes. And all study-related activities were managed via a telemedicine platform, rather than in person. Researchers are now gearing up for a second DTP trial that kicks off this month to explore how remote biosensors can help them study autoimmune diseases.
Another patient-centric solution from Janssen is a platform called iSTEP (Integrated Smart Trial & Engagement Platform), which the company is incorporating into clinical studies to make participating in trials easier for both patients and trial sites alike.
How? Think scanners that automatically track when medication kits are delivered and returned, and electronic drug labels that can inform patients in real time of changes they need to make in their regimens—innovations designed to improve operational efficiency while also generating richer and more accurate data.
We want to create a culture where inclusive and diverse clinical trials is the immediate expectation.
3.
Advancing clinical trials for kids
Clinical trials can often go on for years. And clinical trials that investigate medications for children can run even longer. On average, it takes about 15 years to complete a pediatric drug development program.
In order to try and improve the process for pediatric clinical studies, Janssen helped create the nonprofit Institute for Advanced Clinical Trials for Children (I-ACT for Children)—a global network of pediatric centers conducting studies on pediatric medications.
Then, with the sponsorship of the Innovative Medicines Initiative (IMI) from the European Commission and the European Federation of Pharmaceutical Industries and Associations, conect4children (c4c) was launched in 2018. It’s a six-year project to bring together 19 pediatric clinical trial networks across Europe designed to unite drug companies, patient advocacy groups, children’s hospitals and other public research organizations across the continent.
“Our plan is to foster the development of an infrastructure to improve clinical trials,” explains Katharine Cheng, Senior Director, Pediatric Drug Development, Janssen. “Currently, when sponsors want to do a study in Europe, they have to go to individual countries and sites to implement it, which slows them down. This way, all the different networks come together. Our hope is that this will allow us to do research more quickly, and answer important questions on safety and efficacy in children.”
That hope will be put to the test this year, with the launch of four landmark pediatric studies, led by academic institutions, that will examine the use of steroids to help treat the rare inflammatory disorder Kawasaki disease, and a treatment for brittle bone disease, among others.
4.
Diversifying clinical trials
Not everyone who takes a medication responds in the same way—what’s effective for one patient may not be for another, or worse, may have unwanted side effects.
One way to help ensure every drug works similarly for as many people as possible is to test it on a diverse pool of people whose ethnic and racial makeup are all different, and reflects the actual population who will ultimately use it it. And yet, according to a report from the U.S. Food and Drug Administration, just 2.1% of U.S. clinical trial participants in 2015 and 2016 were Asian American, and fewer than 3% of participants in trials for cardiovascular disease were African American or black—a group that’s at higher risk of the disease than the average population.
It’s stats like these that help explain why Janssen has developed a new strategy designed to increase enrollment of underserved and underrepresented populations in its clinical trials.
“We realized the importance of having clinical trials include data from all patients that carry the burden of disease,” says Staci Hargraves, Executive Sponsor of Diversity and Inclusion in Clinical Trials at Janssen Research & Development. “We want to create a culture where inclusive and diverse clinical trials is the immediate expectation.”
That includes working to modify Janssen’s approach to clinical trial design and operational processes, increase the number of principal investigators who have diverse patient populations and proactively identify and address barriers to enrollment by patients in underrepresented communities.
Another important step is raising awareness of clinical research at the community level.
“We are now making an active effort to go out and simply talk to people,” says Cassandra Smith, Diversity and Inclusion in Clinical Trials Lead, Janssen Research & Development. “There’s a knowledge gap in many underrepresented communities, as well as a mistrust of clinical research. We want to be on the front lines when it comes to getting out there and breaking down some of the barriers that prevent participation in some communities.”
Janssen is already seeing positive results from this work. The company’s GRACE (Gender, Race and Clinical Experience) trial, for instance, was the largest trial to focus on women of color taking HIV drugs to date. It’s been a key source of insight into how to recruit and retain minority trial participants, including in studies that are in development for diseases such as prostate cancer and lupus.
Lupus, in fact, is two to three times more prevalent in women of color than in Caucasian women—and yet the first drug approved to treat lupus in 50 years does not have enough data to support whether it’s effective in African Americans with the condition.
So, when Janssen researchers launched the groundbreaking LOTUS trial last year, they actively recruited a diverse population at the outset, in part by doing local and social media outreach using imagery and educational materials that would appeal to real patients, providing transportation assistance and choosing trial sites with inclusion in mind.
So far, the study is on track to meet its participant goals.
“It’s a bit of a myth that seeking out a population of diverse clinical trial participants incurs heavy costs and project delays,” Smith says. “That’s just not true if you plan an inclusive trial from the beginning. With concentrated effort, we can move the needle and make sure our trials are well-represented by all patients.”
5.
Using real-world data—or reusing trial data—in a unique way
Controlled clinical trials have long been the gold standard for assessing the safety and effectiveness of treatments. Today, though, we have access to hundreds of millions of real-world data (RWD) points about how people are using them, including electronic health records, health insurance claims and even mobile phones. And, in many cases, these data points can be more useful to researchers than trial data. There are also situations in which reusing clinical trial data can reduce the need to conduct further trials.
“They allow us to take a closer look at populations that may not have been well represented in the original clinical trial and then study the impact of a particular treatment over a longer period of time,” explains Jesse Berlin, Sc.D., Vice President and Global Head, Epidemiology, Janssen.
Cases in point: Existing data from four clinical trials on a Janssen treatment revealed that it can help prevent symptomatic blood clots and even mortality following knee or hip replacement surgery. And 29 studies worth of data on Listerine® showed that it was effective in reducing gingivitis and promoting overall oral health, leading to the approval of a new health claim to be used on Canadian product labels.
More recently, Janssen developed a new model for translating RWD into real-world evidence (RWE) of health benefits by using de-identified patient information from four U.S. administrative claims databases as part of the largest and most comprehensive evaluation of the risk of hospitalization and other complications for certain type 2 diabetes treatments.
“Going forward,” Berlin says, “we will be continuing to engage with agencies and others to explore how the innovative work we’ve been doing can inform regulatory and clinical decisions.”
