Patient-Centric Co-Creation in Clinical Trials

Successful clinical trials depend on the enrollment and retention of significant numbers of eligible participants. Over the years, however, this has proven difficult to accomplish. Insufficient recruitment and high dropout rates have led the industry to seek out new ways to conduct clinical trials, ways that work better for the patients.

Regulators have upped the ante, as requirements are in place all over the globe that clearly state that study protocol designs must reflect participant feedback.1 The hope is that by gathering and analyzing input directly from patients well in advance of the study going live, we can make studies more accessible and less of a burden for participants and their families, allowing researchers to get eligible patients into studies more quickly
and keep them engaged throughout the course of a study.

To date, much of the progress around making clinical trials
more patient-centric has come from sponsors. Certainly, advances have been made as evolutions like decentralized clinical trials (DCTs) and the growth of data mining to aid in recruitment are having positive impacts on finding and conducting studies that work better for participants. Now is the time for study leaders to take the next step toward delivering the best possible research experiences for patients by co-creating their studies with patients.

Patient co-creation in clinical research means a shift from thinking about the patient to thinking with the patient. This means that patients are giving feedback about the study, sharing their concerns, and providing input on specific areas like visit schedules or dosing requirements. Co-creation doesn’t stop with patients, but can also include feedback from caregivers, advocates, and clinical sites - any and all stakeholders that can provide valuable insights on how to make studies work better for patients. For study leaders, this means collecting all of this feedback and working to create the best possible study in terms of both patient experience and the quality of data produced. Patient feedback should be analyzed and reflected in the study design, taking into account the capabilities and preferences of the patient population along with addressing any unmet needs. It can also mean the discovery of new or additional endpoints that help to prove safety and/or efficacy. Importantly, co-creation helps sponsors to develop studies that can put eligible patients in the best position to successfully take part in the study.
This improves the chances of meeting recruitment timeline goals and retaining participants over the course of a trial. True patient co-creation provides the end-user – the patient and/or their caregiver(s) - with the same agency over the process as all other stakeholders.

Co-creation helps ensure that a clinical study is designed in a way that addresses patients needs that, historically, have not been met with older study design processes. Co-creation also serves to reduce barriers to participation. There are many such barriers that keep otherwise eligible and motivated patients from participating in research. Common examples include:

• Requirements for travel to study sites, which may include overnight or long-term stays for patients and families
• Study designs that patients find overly complex or overwhelming
• Outdated tests and assessments that increase patient burden, yet are included because “that’s the way we’ve always done it,” as opposed to their ability to produce valuable data
• The use of medical devices, including wearable devices, that some patients may find uncomfortable
• The use of technologies that many patients may find complex and difficult to use
• The lack of broadband and/or cellular data infrastructure for some patients

By involving patients from the beginning of the study planning stage, we can better anticipate these kinds of barriers and design our studies to account for them. Indeed, effective co-creation sets the stage for better engagement of participants over the course of the trial. The alternative trial-and-error style approach has proven to slow down studies and lead to poor participant retention.

During the planning stages of a Parkinson’s disease study using DCT elements, a sponsor sought to understand how potential study participants, patients and caregivers alike, were likely to react to and interpret the informed consent process. They were particularly curious about whether the design was clear in how it presented study purpose and participant responsibilities, as well as how it laid out the potential risks and benefits of participation.

The sponsor worked with THREAD on a patient listening program powered by the newly-acquired voice research company, inVibe. inVibe enables Life Science companies to conduct automated interviews with key healthcare stakeholders, and then extract insights from those interviews by analyzing not only their words, but also tone, emotion, and vocal affect. Through this program, the sponsor was able to learn that patients were concerned about the lack of details around potential side-effects, and were similarly concerned about a trial requirement for lumbar punctures. Patients did not feel the purpose of the punctures was adequately explained. On the other side, the sponsor learned that patients were overwhelmingly positive about the use of DCT elements, finding that the right balance of in-home and in-office activities made the study more accessible. Taking all of this feedback into account, the sponsor was better able to deliver a study experience that worked for patients, setting the trial up to be successful.

Certainly, making studies easier for participants is an important goal of co-creation, but there are other factors that make engaging with patients to design studies vital for modern clinical research.

Co-Created Studies Are More Compliant

As noted before, regulators such as the U.S. Food & Drug Administration (FDA) and others around the world are making it clear to sponsors that participant feedback and perspectives must be taken into account when designing and conducting clinical trials.2 

Co-Created Studies Are More Inclusive

Co-creation allows sponsors to anticipate and work to remove barriers for the likely study population. Many of these barriers may be cultural or socio-economic in nature and reaching out to patients living in these kinds of communities or situations can help to design more inclusive and representative clinical trials.

Co-created Studies Produce More Complete Data

This leads to the fact that more patient-centric and inclusive clinical studies are equipped to deliver more complete and accurate therapy data.

Co-created Studies Drive Innovation

Listening to patients and implementing new approaches
based on their feedback spurs creative problem-solving and
can lead to advances in protocol design, helping to push the
industry forward.

There are a few common ways to gather patient insights. A helpful resource on how patient input can be collected was recently released by the FDA. The guide is focused on how research stakeholders can collect and submit patient experience data and other relevant information in order to improve therapy development and regulatory decision-making.

There are, of course, different methods for collecting patient insights. Per the FDA, these include:

• Qualitative methods, e.g., interviews and focus groups that seek to obtain deeper insights from patients in their own words
• Quantitative methods, e.g., surveys that seek to gather quantifiable data that can be analyzed to describe, compare, and relate different measures of patient experience
• Mixed methods, e.g., surveys that include both open-ended and fixed response questions and/or interviews complemented by fixed-response surveys

Leveraging technology is essential to making these methodologies more scalable. Doing so will allow the collection and analysis of diverse patient voices needed to provide clear direction to sponsors as they draft and amend study designs.THREAD recently acquired inVibe Labs to do just that.

Through inVibe, THREAD enables companies to gain valuable patient insights across important areas in the drug development process, including the patient experience, disease burden, trial design, recruitment, and retention. inVibe works by collecting all of the patient voice data, then analyzing it with a combination of human linguistic expertise and advanced machine learning technology. The interactive, searchable, filterable online portal enables our clients to listen to the actual voice responses, share audio clips with other team members and researchers, and/or simply review the high-level executive summary and visual reports created by inVibe analysts.

Once participant insights are collected, the next step is addressing their thoughts and concerns in the study design. Again, there is a great deal of value in the feedback pulled from patients, value that will be wasted if sponsors treat the gathering of patient perspectives as a compliance checkbox. It can be easy for study planners to rely on their own experience and their internal teams to determine the best clinical trial endpoints. Failing to address patient perspectives when determining those endpoints, however, can lead to frustration.

Solution providers like Modus Outcomes, another innovative company recently-acquired by THREAD, take patient feedback data and apply them to the creation of actual clinical trial endpoints, resulting in a more complete picture of safety and efficacy data for the therapy. Not only that, but they build studies to include approaches that can be successful for a representative cohort of patients, leading to more comprehensive study data that accurately reflects the real-world population. For example, a Parkinson’s disease study may be designed to be almost entirely decentralized due to the burden of travel on patients and their caregivers. Participant feedback can help sponsors determine which remote collection technologies will be the best fit for patients and plan for the provision of devices or even internet access to ensure that all participants have the support they need to remain engaged
in a study.

Part of implementing participant feedback in trial design is determining which technology solutions to employ for data collection and patient engagement. It is important to consider which solutions to use, but also how they will be used and what the user experience will look like. Most sponsors do not have experts on staff with expertise developing unique and effective user experiences through applications, software, and/or devices. These points of view are beneficial and can make a significant difference in terms of the success of co-created study design elements. Whether through hiring experts in the field of user interface/user experience design or collaborating with outside specialists, sponsors should consider involving these types of thinkers as early in the co-creation and study design journey
as possible.

Sponsors, to be compliant, must demonstrate their efforts to gather patient feedback and perspectives on their clinical trials. Beyond focusing on the regulatory requirement, however, digging deep into the insights given by patients is both the right thing to do and a valuable resource for optimizing clinical research. By effectively capturing participant feedback and implementing revised or new approaches based on that input, life science companies can make research more accessible to a broader range of participants while discovering more data than ever about the therapies under development.