Patient Engagement: The True Benchmark in Clinical Trials
By Catherine Gregor, MBA, CCRC, CCRP
This article originally appeared in STAT+. You can find it here.
Patient engagement and community outreach may be hot topics in today’s clinical trial landscape, but they aren’t new ideas. Researchers and patient advocacy groups have focused on including patients and volunteers in study design and data reporting for many years. What’s new is the vital role technology plays in patient engagement as decentralized clinical trials become more popular.
Patient engagement simply means patients working with their health care providers to make decisions about their health. In the context of clinical trials, this could mean participants giving advice on trial designs or tracking their own data using wearable devices and mobile applications.
Technology can increase engagement by allowing participants to join trials and submit data from their doctors’ offices, pharmacies, and even their homes. But as companies and organizations embrace technology to increase patient engagement, it’s important not to forget about the power of human connection between patients and clinical trial staff.
Increasing engagement requires experimenting with a mix of decentralized technology and human ingenuity to recruit participants and keep them invested in the trial.
Why focus on patient engagement?
Evidence suggests that involving patients as early as the trial-design phase leads to faster enrollment, higher retention, and better compliance with study procedures. In turn, high enrollment, retention, and compliance create quality data to support drug or device approval.
Engagement strategies like community outreach can bring currently underrepresented populations into clinical trials. Engaging a diversity of participants is essential for understanding the safety profile of different therapeutics and for complying with FDA guidelines around diversity. The day may be coming when drugs and devices are not approved because they weren’t tested on a diverse array of participants.
The biopharma industry has a long way to go to achieve parity: White people made up nearly 75% of the 32,000 participants in trials of the 53 novel drugs the FDA approved in 2020 even though white non-Hispanic people make up 60% of the U.S. population. The good news? Recent data suggests that community outreach and patient engagement efforts improve the representation of Black patients. The percentage of Black participants in clinical trials increased by 11% over the last 10 years.
The same community outreach and patient engagement efforts may help reach other underrepresented groups, like Asian, Hispanic or Latino, and Indigenous people. Improving enrollment and engagement in all currently underrepresented groups would increase the chances of knowing how a treatment works across the board — and of that treatment being approved.
But many trials remain socially and financially biased, with inclusion/exclusion criteria and time commitments that make it more difficult for some populations to participate than others. Challenges like those must be overcome to improve engagement.
Reducing barriers to patient engagement
Barriers to engagement in clinical trials include things like how far participants must travel to the study site, the overall time commitment to the trial, and the need for complex study visits. These make it difficult for trials to recruit and retain participants.
Technology can play a role in reducing social and financial barriers to clinical trials. Potential participants who have strict working hours may not be able to travel long distances for appointments during the workday. But if they could check in at a neighborhood pharmacy or, better yet, from home at times that are convenient for them, trial participation would become more feasible.
That said, planning decentralized and patient-centric protocols takes time and requires upfront investment by both trial sites and sponsors. Some sponsors may get frustrated with the prolonged timelines needed to vet study protocols through community advisory boards. But given that 50% of sites don’t meet enrollment expectations and 85% of trials don’t retain enough patients to meet their study endpoints, it’s clear that sites and sponsors need to change their traditional approaches.
A review of all Phase 3 trials published in PubMed since 2008 found that those developed using patient engagement practices were 20% more likely to reach approval status than trials that didn’t use those practices, regardless of therapeutic area.
In other words, the time teams invest in planning patient-centric protocols can increase the chances of approval for their devices or drugs.
Using technology to keep participants engaged
Technology can improve the patient experience by facilitating real-time communication with study staff, streamlining the screening and consenting processes and increasing the accuracy of patient-reported outcomes.
Technology makes it possible for participants to ask clinical research staff questions immediately via text, video call, or chat instead of waiting for an appointment. They can also sign informed consent documents on a phone or tablet and use electronic patient-reported outcome software to automatically log data from home.
Trial participants also need easy access to information about their health in languages they understand. Helping them comprehend the treatment they’re undergoing is an essential element of engagement.
The FDA has endorsed a digital, patient-centric approach in the hope of facilitating faster approvals and more cost-effective drugs or devices. As former FDA commissioner Scott Gottlieb said, “Digital technologies are one of the most promising tools we have for making health care more efficient and more patient-focused.”
New technologies won’t just improve participation in clinical trials. They will also transform how participants’ data are used to determine treatments’ long-term safety and efficacy.
New streams of real-world data gathered from electronic health records, lab tests, wearable devices, insurance claims, and even social media can provide important evidence on product safety and effectiveness. This data can also come from more inclusive settings and populations than those found in a traditional clinical trial.
Going beyond technology to increase patient engagement
Technology alone won’t make clinical trial participants more engaged. They crave connections with clinical research staff and need to trust the researchers they’re working with. To build this trust, sponsors, clinical research organizations, and trial sites must develop active partnerships with the communities they serve. Some methods that sites and sponsors can pursue are:
Patient advisory boards. These can consist of patients affected by the disease as well as family members, caregivers, and advocacy groups. Patient advisory boards can review protocols before a trial begins and identify requirements that represent hardships for participants, like too many in-person visits or complicated technology. When potential participants know that researchers have listened to their concerns and the protocols don’t place too much hardship on them, they’re more likely to enroll in and engage with clinical trials.
Diverse site landscapes. Many academic medical centers have trouble recruiting diverse patients because they’re located in major cities. By expanding clinical trials to doctors’ offices and pharmacies in lower-income neighborhoods and rural areas, these centers can improve the diversity of the patients in their clinical trials.
Building patient engagement in an era of decentralized trials
No single method will instantly increase engagement in clinical trials. Many participants appreciate the convenience of decentralized trials, and the technology involved in these trials can greatly increase engagement for them.
But others, especially older individuals and those who aren’t familiar with technology, want in-person services. And all participants can benefit from strong, trust-building connections with clinical research staff.
To increase patient engagement with clinical trials and, in the process, increase recruitment and the chance of trials finishing successfully, sponsors, trial sites, and other stakeholders must combine remote technology with listening to participants’ concerns when building protocols.
Only then will they see diverse and fully engaged participants in their clinical trials.
Catherine Gregor, MBA, CCRP, CCRC is the chief clinical trial officer at Florence, a software company focused on remote connectivity in clinical research, and was the former director of clinical research administration at the Vanderbilt-Ingram Cancer Center.