Clinical Researcher—December 2024 (Volume 38, Issue 6)
RECRUITMENT & RETENTION
Jena Daniels
Engaging patients in clinical research has long been seen as important, yet it has been slow to evolve. For years, clinical research has largely been designed with the assumption that participants should conform to rigid protocols, often neglecting the voices of the very people who will be impacted most by the outcomes. A 2018 study, published in Research Involvement and Engagement, estimated that far less than 1% of clinical trials meaningfully and actively engage patients in any part of the research process.
While too many clinical trials are centered around the convenience of researchers and sponsors rather than patients’ needs and preferences, this paradigm is shifting—thanks, in part, to the U.S. Food and Drug Administration’s (FDA’s) June 2024 draft guidance on submitting Diversity Action Plans, which requires sponsors of certain clinical studies to submit plans that detail the steps sponsors will take to include underrepresented populations in their trials, addressing factors such as race, ethnicity, age, and sex. The FDA also highlighted the importance of early patient engagement in trial design and planning, illuminating the significant value of patient advisory boards.
Additionally, patient advocacy networks are growing and getting louder, incorporating the voices of patients into clinical trials and developing unique ways to improve patient participation and adherence.
Karen Utley, whose daughter Samantha was born with a rare neurodevelopmental condition called CDKL5 Deficiency Disorder, speaks directly to the benefits such networks offer to trial participants: “Both mine and my daughter’s experiences were carefully considered during the decentralized trial she participated in,” said Utley. “And these learnings were incorporated to enhance processes and technologies to improve the trial experience for both my daughter and me as her caregiver. I found the experience to be immensely rewarding—a genuine gift of personal experience for the benefit of others.”
Beyond just their participation, patients are a valuable resource for researchers. Now, thanks to such efforts, their voices are being heard more clearly.
“Patients love to get involved in research and have their stories heard,” said Wes Michael, founder and CEO of Rare Patient Voice, an organization that helps to amplify the voices of patients and caregivers. “Patients have valuable feedback to offer so sponsors need to get them involved early and often and not just pay lip service to them.”
Amplifying Patient Voices
Patient advocacy networks, as well as the rising prevalence of decentralized trials and remote patient monitoring technologies, are providing patients with more flexibility and convenience. These developments are reducing some of the logistical burdens that have historically made participation in clinical trials difficult. It’s a step in the right direction, but more work must be done to create a genuinely patient-centric research environment.
The integration of patient perspectives earlier in the clinical trial process—well before recruitment—is key. One way to do this is by utilizing upfront study simulations that involve patients, caregivers, and research staff in mock trials to work through protocols as if the study is already underway. This allows participants to identify issues they might face in a real trial, such as challenges with recruitment materials, consent forms, and logistical aspects including travel and compensation. By proactively involving patients early, sponsors can refine the trial design and reduce the likelihood of patient dropouts or delays due to unforeseen obstacles downstream. The extra time up front can save time and money in the long run, as well as result in better outcomes.
This approach can also provide patients with a better understanding of the tools and technologies they are being asked to use, leading to better adherence. For example, in a recent cancer trial, patients were asked to use a wearable device with a single-size armband and provide feedback. Since these patients were going through chemotherapy, they experienced extreme weight fluctuation, suggesting an adjustable armband was more appropriate. The patients also reported that the device wasn’t easy to clean, its battery life was insufficient, and it looked too much like a medical device. While these patients fully understood the value of the device, they were less likely to adhere to the protocol if these issues went unaddressed. Once this information was shared with the sponsor, it found a better solution that undoubtably led to better adherence. This kind of real-world feedback early in a trial when adjustments can still be made can only come from patients who are actively participating from the start.
Finally, the clinical research enterprise must continue to take a broader view of patient populations when designing trials. Rare diseases, chronic conditions, and diverse demographics often face unique challenges in accessing research. These challenges require nuanced approaches that acknowledge the lived experiences of people. By embracing diversity and including a wide range of individuals in the design of trials, researchers can ensure that clinical studies reflect the varied needs of those they aim to serve.
Overcoming the Remaining Hurdles
Despite the clear benefits of a patient-centric approach, significant hurdles remain. Building and sustaining a robust patient engagement initiative not only requires commitment but also resources. Collecting patient feedback through surveys, focus groups, or simulations takes time and money, and many trial sponsors are hesitant to allocate those resources upfront. Further, with the increasing complexity of trials and the diversity of therapeutic areas, the convenience of a one-size-fits-all solution for patient engagement is rare.
While wearables and remote monitoring technologies can enhance patient adherence and participation in some studies, they are not universally applicable. Certain patient populations, such as the elderly or those with limited access to technology, may struggle to engage with digital tools. Moreover, studies involving complex drug regimens or invasive procedures might not easily lend themselves to decentralized formats. These disparities between trials require a flexible, adaptable approach to patient engagement, which can be difficult to scale.
Additionally, the regulatory framework governing clinical trials presents a significant challenge. While the FDA and other agencies have made strides in embracing innovation, headwinds persist around the approval and standardization of new engagement tools and methodologies. Flexibility and collaboration between sponsors, regulators, and patient groups will be essential for overcoming these barriers.
Collaboration is Key
One of the most powerful drivers of change in clinical research is the collaboration between stakeholders: research sponsors, trial sites, technology companies, patient advocacy groups, and regulatory bodies. Each has a unique role to play in advancing patient engagement. However, these groups often operate in silos. To break down these silos and achieve meaningful change, there must be a shift toward collaborative partnerships.
The inclusion of research sites in this conversation is key—and too often, the missing piece to this puzzle. Sites are on the front lines of patient recruitment and retention and are uniquely positioned to provide practical feedback about the realities of implementing patient engagement strategies. Sites can highlight issues such as staff training, protocol complexity, and patient preferences that might otherwise be overlooked by sponsors or technology developers. Incorporating the site’s perspective into early-stage planning will lead to smoother trial operations and better patient experiences.
Similarly, patient advocacy groups play an important role in educating patients about clinical trials, addressing concerns, and providing support throughout the process. These groups are often the best advocates for ensuring that trials are designed with the patient’s needs in mind, rather than just research tools. Involving patient advocates from the earliest stages of study design will allow researchers to identify potential barriers to participation, address misconceptions, and ensure that the trial is responsive to patient concerns.
Prioritizing Patient Engagement
To truly transform patient engagement in clinical research it must be integrated into every phase of trial design and implementation. Too often the industry treats patient engagement as an afterthought—a vague notion that can be “tacked on” at the end of protocol development or implemented sporadically through post-trial surveys. However, as Michael at Rare Patient Voice pointed out, this is no longer acceptable if we want to improve both the experience and the outcomes for patients.
“Patient participation should be part of a trial’s standard procedure up front,” Michael said. “You don’t want to have to go backwards, so patients need to be involved from the beginning, before a trial goes too far. Today with modern technology and expanding regulatory frameworks for patient participation, it’s easier than ever to engage patients if you make the effort.”
Patients are experts in their own experiences, and their insights can be instrumental in shaping successful trial protocols, study design, and operational strategies. By embedding patient engagement into every stage of the trial, researchers and sponsors will ensure that patient needs are met from the very first step to the final follow-up.
Open-ended questions should replace simple yes-or-no surveys to better understand the complexities of the patient experience. These dialogues invite patients to share concerns, preferences, and ideas for improvement that may not be captured through basic quantitative measures. Asking questions like “What would make participation in this trial easier for you?” will foster meaningful feedback that can inform trial adjustments.
Upfront study simulations—where patients, caregivers, and trial staff work through study protocols together—are a crucial tool for gathering this feedback. These simulations allow researchers to anticipate challenges and mitigate them before the trial is underway, saving time and resources in the long run. With patient engagement built into the trial’s design phase, sponsors can avoid costly delays, dropout rates, and dissatisfaction that can arise from an inadequate understanding of patient needs.
The Future is Now
Patient engagement is no longer a “nice-to-have.” It is a necessity for achieving meaningful, reproducible outcomes in clinical research. By actively collaborating with patients, sites, advocacy groups, and regulators, we can create trials that not only meet scientific goals but also respect the lives of those participating in them. Much work needs to be done, but by embracing collaboration and committing to patient-centered practices, stakeholders can create a clinical research environment that prioritizes not just the success of the trial but the experience of the people who make that success possible.
“People understand that clinical trials are important, and they want to participate in them, but they also can’t turn their lives upside down to do so,” Michael concluded. “People have to find a way to work the trial into their lives, and sponsors need to meet them where they are to help make that happen.”
Jena Daniels (jena@medable.com) is a manager with 12 years of experience in enabling digital solutions within academic and industry-sponsored clinical trials. As a 2021 PharmaVoice 100 recipient, Best Decentralized Clinical Trial Businesswoman of 2022, and Top 50 Women Leaders of San Francisco for 2023, she is most known for establishing Medable’s Patient Caregiver Network, which seeks to engage and empower expert patient and caregiver advocates to assist product development, refine study workflows, and tailor the overall trial experience. She has coauthored more than 30 peer-reviewed publications focusing on how to leverage machine learning, artificial intelligence, and remote patient monitoring to triage, diagnose, and provide early intervention for children with autism spectrum disorder.
