3 Principles for Patient-Centered Research Design, Clinical Trials

December 02, 2019 - Clinical trial research is the heartbeat of pharmaceutical drugs and medical devices. This rigorous series of testing, in which researchers look at real-world evidence to determine a treatment or tool’s efficacy, is essential for safety and clinical protocol.

But the traditional system for clinical trial tests has one fundamental flaw: it neglects the role of the patient, arguably one of the biggest stakeholders in developing new treatments. Limited patient-centered research design comes with numerous pitfalls, including non-response bias, missing key study populations, or omitting key questions patients and providers have with regards to a new study protocol.

Without engaging patients during the clinical trial design process, researchers run the risk of misunderstanding the outcomes that are most important to patients or strategies for disseminating research in a way that is usable and understandable for both patients and caregivers.

Most medical researchers agree on this fact, but nonetheless very few are actually able to properly engage patients during the research development stage, according to data presented at the National Kidney Foundation 2018 Spring Clinical Meetings.

A survey of over 1,500 healthcare and kidney disease stakeholders, including nearly 700 kidney disease researchers, found that fewer than one-quarter of researchers have consulted patient needs and preferred outcomes as a part of their kidney disease research.

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Kidney disease researchers are also not publishing their study results in places that are easily accessible for patients. About forty-seven percent of studies are published exclusively in medical journals, as opposed to other media non-clinical laypeople may view.

Biotech and pharmaceutical companies working to develop drugs and technologies tailor-made for patient needs may consider further integrating patients into the clinical trial design process. By accounting for patient views about trial recruitment, study question development, and results dissemination and publication, researchers can ensure clinical trials and other medical research endeavors are more suited to patient needs and preferences.

Tapping patient expertise for clinical trial recruitment

Clinical trials rely on ample patient participation, and especially diverse patient participation. Including patients with different backgrounds, preferences, lifestyle needs, and socioeconomic factors can prevent non-response bias and ensure more accurate and actionable trial results.

A patient advisory group dedicated to helping tailor clinical trial design can help in this respect, according to Jean Slutsky, PA, MSPH, the chief engagement and dissemination officer at the Patient-Centered Outcomes Research Institute (PCORI). While institutional review boards oversee the ethical and procedural components of research recruitment, patients can offer guidance on this process.

“Patient partners can actually provide information about how useable informed consent documents are, how readable they are,” Slutsky said in an interview with PatientEngagementHIT.com. “They can’t replace institutional review boards, but they can help the investigator think about ways to reach patients in more comprehensive ways and ways in which it is easier for patients to participate.”

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Patients can also be instrumental in retaining clinical trial participants. Over the course of a trial, it is probable that some participants may become non-responsive, skewing the study’s results.

“Patients can be incredibly helpful when you talk about how to recruit patients into a study and how to retain them,” Slutsky noted. “They can give good advice by saying, ‘well if I were a patient you tried to approach for a study, if you approached me during this time I might be too overwhelmed to even consider contributing to the study.’”

Patients help shape important research questions

Clinical trials can fall flat when they do not consider factors that are important to patients, the ultimate stakeholders of new initiatives and treatments. While it will always be important for researchers to look at factors that impact clinical workflow and best practices, understanding which outcomes are also important to patients will lead to a multi-faceted and comprehensive study.

Consulting with patient advisors has had positive impacts on study design, PCORI reported in a 2019 study looking at the factors that constituted patient-centered research.

“Partners contributed to identifying the topic, defining research questions or aims, selecting comparators to examine, and establishing study outcomes (that is, constructs to study),” PCORI researchers said. “Authors wrote that because of engagement, projects selected, prioritized, added, or modified primary or secondary outcomes—including health status and well-being, knowledge and understanding, and evaluation of care.”

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These findings align with Slutsky’s experience, she said in the interview.

“It’s about the actual research question,” Slutsky explained. “It must be a question that folks would have. The outcomes that are measured in the study must be ones that are important to patients. Oftentimes, that happens when the principle investigator sits down with their patient partners and talks about the outcomes and the questions that they’re going to be researching.”

Driving patient engagement, access to study results

Just as understanding patient needs and preferences in designing clinical trials is important, so is ensuring trial results are accessible. Efforts on that front are twofold.

First, PCORI recommends researchers publish findings in medical journals that are accessible to patients.

Second, PCORI recommends ensuring patients have access to readable and understandable study results. In doing so, patients can use that information to engage with their care.

Researchers can accomplish this by identifying partner organizations that can help deliver trial results to patients in an actionable format. Partnerships with patient advocacy groups, national medical foundations, and the media can help ensure patients have access to and understand clinical trial results.

Ultimately, these efforts will help researchers create clinical trials and other medical research projects cater the patient.

“We all appreciate that oftentimes when we hear about study findings, it may not necessarily reflect information on what’s important to us as patients or caregivers,” Slutsky remarked.

“Patients are faced with making medical decisions about their care all the time. If they don’t have the information to help them make those decisions, it’s a disadvantage for them,” Slutsky concluded. “The more research is funded around questions that they would want answers to and outcomes that are important to them, the more likely that it will be that the research is useful to them when they are faced with healthcare decisions.”