Patient Centricity in Clinical Trials: Turning Up the Volume on Patient Voices
Clinical trials lay the foundation for breakthrough drugs, digital therapeutics, and medical devices—and, at the center of these studies are the patients.
The decentralized clinical trial movement can improve the study experience for patients. But, as the above data illustrates, there’s more work to be done—not only addressing the issue of burdensome trial designs but also a deep-seated problem within the culture of the industry: Instead of conducting trials for patients, we need to conduct trials with patients. It’s only after patients are considered equal partners in clinical trial planning that the industry will see the true impact of DCTs.
In this article, we’ll explore how to bring patients into the process of clinical trial planning, helping sponsors and study teams choose meaningful endpoints and deploy the right digital tools to reduce participant burden, encourage engagement, and create a truly patient-centric clinical trial experience.
Key Takeaways:
- Patient centricity requires the prioritization of participants’ needs and experiences; to do this, patients must be involved in clinical trial planning.
- Patient-centric clinical trial design begins with choosing endpoints and outcomes that are meaningful to patients.
- While not every study can be fully decentralized, DCT components offer many advantages for reducing the patient burden and increasing patient centricity.
Let’s begin by exploring what patient centricity in clinical trials means.
Defining Terms: What is Patient Centricity in Clinical Trials?
“Patient centricity” is a buzzword frequently used throughout the healthcare ecosystem, encompassing everything from healthcare affordability to increased patient education. Across the continuum of medical research and delivery, a shared understanding of the concept emphasizes patients’ voices and lived experiences.
Within clinical trials, patient centricity can be more narrowly defined as prioritizing participants’ needs and experiences through every stage of the study. By integrating tools and support systems that accommodate these factors into a study from the beginning, sponsors can achieve end-to-end patient centricity.
Regulatory Guidance on Patient-Centric Drug Development
In addition to the growing expectation of patient centricity throughout healthcare, clinical trial sponsors face mounting pressure from advisory boards and regulatory bodies that have issued frameworks and guidance on the topic.
For example, FDA guidance on Patient-Focused Drug Development (PFDD) recognizes the importance of incorporating patients’ priorities and perspectives into the drug development process. The PFDD guide notes that patients are “experts in what it is like to live with their condition” and are therefore “uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation.”
The takeaway is clear: Patient centricity in clinical trials is becoming table stakes for the industry—and its impact on the perceived and actual success of any clinical will only continue to grow.
Co-Creating Clinical Trials with Patients
According to a peer-reviewed article on how to include patient perspectives in clinical trials, patient involvement is often confused with patient opinion surveys. While collecting data from patients can be important for gaining diverse viewpoints, the involvement of patients should go deeper. Researchers should be developing a “continuing and reciprocal relationship with patients and make decisions with them about the research.” The article continues by providing a number of recommendations for how to effectively build this system of reciprocity:
- The minimum number of patients to work with is two patients.
- Using terms such as “co-production” or “co-planning” can help recognize patients as equal partners and encourage them to speak up.
- It’s important to work with diverse backgrounds and personal experiences to gather a full and accurate understanding of the disease experience.
- Be careful to avoid bias. Patients who work with research teams over many years can risk losing the “eye of the public.”
Once patients are brought on as partners to co-design a clinical trial, the advantages are tangible. Patients can help researchers:
- Ensure the research is relevant to patients, thereby increasing enrollment.
- Design a trial that reduces burden, thereby improving compliance and retention.
- Ensure the study outcomes really matter to patients, thereby increasing the likelihood that the intervention is implemented into clinical care.
Choosing Meaningful Endpoints and Outcomes Measures
Co-creating a clinical trial often starts at the end—the study’s endpoints. Too often, researchers fail to consult patients on what therapeutic improvements matter most to them, instead choosing endpoints that are easy to measure. Recent publications have explored the fallibility of this approach: “If a research question is of low priority to the people affected by the condition, or important outcomes are not considered, and/or the intervention in question is considered unacceptable to patients, then further research is wasteful.”
In a letter to the editor of Scandinavian Cardiovascular Journal, researcher Johan Bundgaard argues that many current clinical trial endpoints are subject to the “streetlight effect”—“people searching for something where it is easiest to look.” This is, perhaps, unsurprising—patients’ therapeutic priorities may be more complex to measure. But, industry experts have noted that anchoring a study in what’s important to patients will not only resonate with patients themselves but “with all stakeholders—payers, providers, sponsors, regulators,” and patients alike.
Once a meaningful endpoint is chosen, the challenge then becomes finding the most patient-centric methods to measure outcomes. Many measurement tests do not translate to the real world, leaving patients to question why these tests are being performed.
For example, a recent digital biomarker article points to the impracticalities of the 6-minute walk test (6MWT), describing it as a “poor measure” that does not “represent a patient’s meaningful functional abilities in real life.” The industry needs to ensure measures reflect patients’ lived experiences, not only to acknowledge what matters to patients in their everyday lives but to support research authenticity and real-world practicality.
Fortunately, technology is facilitating new ways to measure and capture data. When included strategically in clinical trial design, these innovative tools can lead to novel and more patient-centric outcomes and endpoints—while also reducing patient burden.
Working with Patients to Reduce Burden in Clinical Trials
One pain point consistently emerges during the process of co-designing clinical trials: the heavy burden of participation. Research shows that, depending on the phase of the trial, between 15 and 40 percent of patients enrolled at the beginning of a study later drop out, citing reasons associated with burden. This data is supported by a recent study of 334 adult oncology patients participating in clinical trials, which found that 33.3 percent of those surveyed perceived the benefits of participation as being less than the burdens.
The reality—and the consequences—of high participant burden in clinical trials is well known within the industry, as is the potential for decentralized clinical trials to contribute to reduced hardship. However, to ensure a truly patient-centric experience, decentralized components should be vetted by patients to assess their tolerability, usability, and usefulness.
Below, we explore some trial components that patients and researchers may want to consider together:
- Comprehensive eConsent
- Virtual site team support
- Online training and onboarding
- Remote data capture
- More convenient points of care
Comprehensive eConsent
Informed consent is the heart and soul of patient centricity: If a patient doesn’t fully understand what they are consenting to when they join a trial, their participation is on ethically shaky ground.
One of the biggest difficulties with informed consent is that patients’ understanding of the study protocol varies according to many factors—such as their medical knowledge, educational experience, reading speed, and emotional state.
Research has shown that providing concise, yet thorough, consent materials can strike the balance needed to convey necessary information in a truly accessible way. Moreover, seeking patient input on consent documents can unearth assumptions and misconceptions, so study teams can address them before enrollment.
Enter comprehensive eConsent, which can be configured to meet both patients’ and the specific study’s needs. eConsent affords participants the time to review each paragraph or concise summaries of key information at their own pace. This “progressive disclosure” approach is designed to support patients’ reasonable understanding of their role in the study, as well as engagement and adherence in later phases.
Ideally, an eConsent study module should also include features such as interactive elements and comprehension quizzes to boost understanding. Better still, a virtual site team should be accessible to patients via chat, email, or phone to address any questions or concerns.
Virtual Site Team Support
Virtual site teams provide ongoing support to participants throughout the trial. Whether it’s during informed consent or the final week of the study, these teams can answer participant questions, respond to concerns, and assist with any technical problems.
Researchers should seek patient recommendations on how often a virtual site team should engage with patients throughout the duration of the trial. How much interaction is overbearing? How does this patient population prefer to communicate—phone call, video, text, email?
In addition to communication, virtual site teams can monitor patients’ data, so that when a patient doesn’t complete a required trial task or attend a designated online check-in, trial team members can reach out. They might find that the patient is experiencing unwanted side effects or that life stressors are drawing their attention away from the trial. The team member can then trouble-shoot with the patient, and—when warranted—encourage them to continue with their trial tasks.
Ongoing virtual support centers the patient experience in an especially crucial way: protecting the patient’s health through the early detection of adverse events. In a traditional trial, patients might assume their symptoms aren’t serious, or forget to report symptoms in person. But, with access to a virtual site team, if patients report side effects outside the expected norm, a team member can alert them to seek medical attention.
Online Training and Onboarding
After patients are enrolled, setting them up for success using virtual training and onboarding supports their long-term retention. Sponsors can deploy a bring your own device (BYOD) app to eliminate the need for site visits in the onboarding process, removing yet another demand on busy patient schedules.
To accommodate different learning styles, study teams can create and deliver robust, accessible trial training using a variety of mediums, including video, written instruction, or animated video. Study training should also remain available for patients to revisit whenever they have questions and concerns.
Doubling down on patient education is key to improving the patient experience—and converting patients into engaged participants. When patients know what to expect in a trial, they’re more likely to complete the trial as requested.
Remote Data Capture
Remote data capture enables patients to measure outcomes from the comforts of home. This reduces trips to a clinic and, because outcomes capture occurs in the real-world, makes it easier for patients to collect consistent, accurate data. Below are some remote data capture options for measuring outcomes:
Sensors and Wearables
When properly selected and integrated, sensors and wearables can easily and precisely collect vitals, signals, and symptoms. Many of these devices can capture the data passively, meaning the patient wears a sensor and data is captured by that sensor as the patient goes about their daily life. But, trial sponsors need to consult with patients to understand situational barriers with the patient population that might make digital tools more burdensome. Devices designed for precise measurement may not be intuitive or easy to use, leading to low compliance. The key is to focus on user-friendly devices best suited for the study population, condition, and treatment.
Audio and Image Capture
Smart audio, video, and photo capture are capabilities also worthy of mention, especially suited for studies requiring objective patient reporting. These functions allow participants to submit their personal medical data seamlessly, with as much fidelity and accuracy as possible. In the case of a vaccine trial, a patient can capture an image of an injection site reaction (ISR) and submit it to clinicians virtually, as opposed to traveling to a clinic for the ISR to be evaluated in person.
ePRO
The simple use of electronic patient-reported outcomes (ePRO) can improve quality of life. A randomized clinical trial recently revealed that cancer patients who used ePRO had improved health-related QOL and fewer visits to emergency rooms at 6 months versus a control group. Despite this staggering data, only 6 percent of oncology clinical trials deployed ePRO between 2010 and 2021.
While ePRO is an effective method for capturing patient symptoms—and certainly more convenient than paper PROs—patients should be consulted when choosing which validated questionnaires to use, how many to use, and at what frequency. Some trial designs bombard patients with too many questionnaires, resulting in waning compliance as patients grow tired of responding to similar questions repeatedly.
More Convenient Points of Care
For most trials, visits with a clinician are necessary. This is often the biggest burden in a trial design, so it’s necessary for trial planners to consider more convenient ways to accomplish objectives. Can the visit be conducted via telehealth? Or is it possible to send a home healthcare nurse to the patients’ homes? In many cases, it’s best to give patients options.
For appointments that can’t be completed online or in the home, geographic and scheduling barriers can be mitigated through partnerships with local medical centers, clinicians, and pharmacies. If patients require a vaccine, for instance, they could travel to a local drugstore ten minutes from their home instead of a major medical center an hour away.
By leveraging these different points of care, sponsors and study teams can not only improve the patient experience but also increase access to clinical trials for those without personal transportation or those with mobility difficulties.
Delivering on the Promise of Patient Centricity in Clinical Trials
When patients work with researchers as co-creators of clinical studies, the industry can provide a better trial experience. As researchers and patients collaborate, decentralized clinical trial methods should be considered for their abilities to reduce patient burden.
With this combination of patient voices and DCT methods, sponsors will see the results in the data—higher compliance and retention, more accurate outcomes, and, ultimately, stronger therapeutic evidence.
