Empowering Patients: The Impact of Lay Patient Perspectives in Clinical Trial Design

Discover how to optimize patient engagement efforts by engaging with lay patients.

While the definition of an “empowered patient” may vary somewhat within the health care industry, empowered patients experience enhanced control over their health care journey and actively participate in their health care through informed, confident decision making.^1,2 Within clinical research, patient engagement activities can lead to patient empowerment.¹ These activities aim to uncover a deeper understanding of the patient’s lived experiences and invites them to participate in research decisions.³ While researchers are increasingly engaging with patients on trial design and implementation, challenges persist in participation and diverse representation in clinical trials. Therefore, the opportunity remains to optimize the impact of these patient engagement efforts to empower a broader, more diverse group of patients to confidently participate in clinical research.

The purpose of this article is to present a new strategy to address this opportunity: engaging with “lay patients.” Lay patients have diverse perspectives and unique lived experiences that are more closely representative of the trial’s intended patient population. When researchers conduct patient engagement activities, it can lead to the empowerment of those specific individuals through the act of their involvement in the activity. Researchers can further advance their engagement efforts by engaging with lay patients and integrating their insights throughout the trial development process, which can lead to the empowerment of all patients who touch the trial at any level.

Patient engagement strategies in clinical trial development

Current patient engagement strategies offer opportunities to empower patients as key stakeholders throughout the clinical trial development process. Clinical trial professionals are incorporating strategies, such as collecting feedback to inform patient reported outcome surveys, trial materials and recruitment strategies, to ensure the patient voice is represented. Combining these efforts with initiatives related to increased data transparency and privacy protections can further empower trial participants by enhancing their control and ability to make informed decisions.^1,4 Historically, however, these patient engagement activities have been most commonly conducted through patients who have already opted in to research, such as patient advocates or patient experts, who speak on behalf of their broader patient population.⁵ As the inclusion of the patient voice in clinical research grows, it is our responsibility to engage with a variety of patients and empower all patients to confidently participate in clinical research, paving the way for more effective, inclusive and patient-centered research.

Engagement with lay patients

Clinical trial teams can optimize the impact of patient engagement efforts by engaging with “lay patients.” Lay patients are everyday individuals invited to share their personal experiences with a specific disease or condition through the lens of their awareness and understanding. Importantly, they are not medical professionals, patient advocates or experts with health care or pharmaceutical industry knowledge. Lay patients can also include caregivers who may be caring for someone with a specific disease or condition, with neither having deeper health care knowledge.

Lay patients provide a deeper, more meaningful and nuanced perspective. They offer a spectrum of diverse perspectives that complement patient expert and advocacy channels by validating and expanding on experts’ insights. Lay patients often have no previous clinical trial experience, nor do they have much understanding of what a trial entails. This absence of awareness is not from a lack of interest, but because they have yet to be informed that trial opportunities exist and haven’t been provided the basic information about clinical trials. Essentially, engaging with lay patients explores the experience of an everyday patient learning about a clinical trial opportunity for the first time. This unique perspective provides trial teams with new insights that mirror individuals who are often underrepresented in clinical research.

While patient engagement activities can lead to the empowerment of the individuals who participate in the activities, engagement with lay patients can contribute to the empowerment of all patients participating in the clinical trial at any level. Lay patients, by definition, are more likely to be unempowered patients. One of the hallmarks of an “empowered patient” is the confidence to ask questions, articulate concerns and advocate for one’s needs. An unempowered patient who is presented with a clinical trial opportunity may not feel comfortable communicating their questions and concerns, thus self-selecting out of participating based on inaccurate assumptions. Lay patient engagement can uncover unspoken needs, concerns and assumptions that trial teams can address throughout the trial, overcoming potential barriers for patients to confidently participate in the trial.

Innovative methodologies for engaging lay patients

Traditional patient engagement activities include qualitative research methodologies like in-depth interviews, focus groups or listening sessions. However, there are innovative approaches that are optimally suited to gather insights from lay patients, who may have limited clinical understanding. These approaches put the lay patient in the shoes of a potential participant, engaging them in a less hypothetical situation and a more immersive, hands-on experience.

• Clinical trial simulation: A “rehearsal” of key visits from your clinical trial to assess burden and uncover any challenges that may be barriers to participation or retention before protocol finalization. This includes interactive role-play of specific study visits with a physician investigator (PI) and study coordinator (SC) team and a patient and/or caregiver. The PI/SC team engages with the patient and/or caregiver, role-playing the study visit and explaining the assessments in a risk-free environment. The simulations are not scripted, resulting in authentic interactions. Following the simulations, all participants participate in debrief interviews with a professional qualitative research moderator to explore their perspectives and generate patient insights on ways to mitigate concerns and potential burdens. This novel approach cultivates meaningful feedback by putting patients and site staff “in the shoes” of participants and brings the protocol to life to generate impactful insights.
• Digital tool field testing: Testing digital tools, wearables, apps and/or eDiaries with lay patients in a manner that replicates the real-life usage and trial expectations in various environments such as home, school and the workplace. This approach goes beyond traditional user testing and explores the individual’s preferences, habits, lifestyle and individual needs/burdens when using digital tools and devices as they would in the trial. This approach informs technology optimization to reduce patient burden while maintaining compliant and effective usage by patients.

These are only two examples of novel methodologies to engage with lay patients. Trial teams can establish a bespoke approach that considers the specifics of the trial itself, the therapeutic area, appropriate sample size and patient population characteristics.

Engaging with lay patients can present unique challenges for trial teams, but there are solutions available to aid in recruitment efforts. Teams should build in budget and time to find and engage with these patients, as they are not as accessible or available as patient experts or advocates who are accustomed to contributing to and collaborating with the health care industry. Further, there are no exact demographics that qualify an individual as a lay patient — they come from all age groups, genders, races, ethnicities, socioeconomic statuses and geographic locations.

While these are challenges to consider, engagement with lay patients doesn’t have to be cost-prohibitive or time-consuming. Lay patient engagements can occur in tandem with trial design and development activities, allowing trial teams to incorporate feedback in real time. The recruitment process can be simplified, often with smaller sample sizes to yield sufficient saturation. Trial teams can consider working with professional recruitment vendors or engage in community-based, word-of-mouth recruitment. Additionally, since lay patient engagements are meant to inform internal processes, trial teams can soften criteria, ask potential participants to self-report and screen for previous clinical trial involvement.

Examples of lay patient insights

Engagement with lay patients provides clinical trial teams with abundant insights that can enhance a wide range of aspects of the clinical trial that serve to empower all participants who may participate. While protocol adjustments based on lay patient feedback are one outcome, these critical insights can impact various aspects of the trial, such as:

Effective patient support services: Identifying support services that are deemed the most valuable and influential (and least valuable and influential) to patients to tailor resources effectively.

• Example: An Alzheimer’s trial intended to offer ride-share reimbursement, a service traditionally viewed as positive and patient-centered. Engaging with lay patients uncovered that, because of their condition, some participants didn’t like the idea of a ride-share service because they were uncomfortable riding alone with a stranger. Offering a transportation concierge service with the same driver for each study visit instead helps participants overcome that barrier.

Important aspects in site selection: Informing site selection based on patient priorities related to travel requirements, site location, parking access and cost, site size, and site navigability, and identify patient preferences of what could be traded off for higher priority site aspects.

• Example: Engagement with lay patients revealed that some individuals prefer separate reception areas for participants, reduced walking distances during study appointments (e.g., walking from the physician meeting to the lab), and having site staff speak the participant’s language (e.g., Spanish-speaking receptionists). Selecting sites that meet some or all these needs help provide welcoming and inclusive environments and meet participants’ specific needs.

Effective investigator training: Providing investigators with insights on unique patient concerns, challenges, questions and needs to build empathy and understand the patient’s perspective, improving their effectiveness in addressing patient challenges throughout the trial.

• Example: Multiple engagements with traditionally underrepresented populations revealed that some participants thought they weren’t eligible to participate in a clinical trial because they were under- or uninsured. Some participants also admitted to being intimidated by the health care system, often not asking questions for fear of appearing uneducated or uninformed. Offering training for investigators to specifically address these unspoken misperceptions up front opens the door to potential participants who might otherwise have opted out.

Accessible, easy-to-follow patient materials: Creating patient-friendly materials that provide clear direction, address questions at the forefront of patient minds, and highlight benefits likely to influence participation and offer tips to improve data quality.

• Example: A simulation with lay patients for a protocol that requires at-home eDiary completion resulted in insightful logistical questions from participants. Revising materials to clarify how to complete eDiary tasks empowers patients to confidently and accurately participate in at-home data collection, which contributes to better data quality.

Enhanced trial team strategies: Building strategies that overcome internal trial team assumptions of feasibility by walking patients through trial requirements to better understand their perspective about potentially uncomfortable or undesirable requirements that could hinder recruitment efforts.

• Example: Engagement with lay patients found that they were comfortable with collecting, storing and shipping at-home bio samples because those patients would prioritize finding a treatment solution for a condition that is always uncomfortable to them. Contrary to expectations, the at-home sample collection requirement of the study wasn’t a problem or inconvenient in comparison to the everyday lived experience of the patients, enabling the trial team to confidently kick off recruitment activities.

These examples show how trial teams can translate insights generated from lay patient engagement into creative, meaningful solutions to empower all patients who participate in the trial. Additionally, these insights can contribute to broader patient-informed communication and enhance ongoing health care professional interactions, leading to increased patient knowledge and awareness of clinical research, which ultimately foster trust and empowers patients to actively participate in their health care journey.

The evolving landscape of patient engagement in the development of clinical trials underscores the growing importance of patient empowerment, which can be enhanced through integrating lay patient insights. Engaging with lay patients can address the unmet needs of diverse patient populations and promote empowerment and active participation in clinical research. Innovative methodologies, such as clinical trial simulations and real-life testing of digital tools, provide valuable insights that can lead to patient empowerment. As we continue to evolve and implement these strategies, the potential for more empowered patients and increasingly effective, inclusive and patient-centered clinical trials becomes more attainable, ultimately benefiting both patients and the broader health care community.

Ensuring patient-centricity throughout your product journey

Our Patient Insights Research team amplifies the voice of the patient, leverages patient insights and informs patient-centered initiatives at every stage of the pharmaceutical product life cycle. We have learned that when patients become partners, innovation is sparked. Leveraging our team members’ decades of experience and scientific rigor, we are defining a path forward to uncover deep insights that lead to more effective treatments and better outcomes for patients. Our team delivers innovative and industry-leading programs to support study design/execution, disease-based programs, market access and post-approval activities, while turning data into actionable insights for sponsors.