A New Tool to Assess Patient Engagement Efforts Across Organizations
Jennifer Kim
Tufts University
Ellyn Getz
CSL Behring
Valerie Fraser
Patient & Research Advocate
Keri McDonough
Syneos Health
Maria Paula Bautista Acelas

atient engagement is defined as inspiring and mobilizing stakeholders beyond clinical research professionals, including patient advocacy groups, healthcare providers, and policy makers, to amplify and incorporate the patient voice in the development and delivery of new medical treatments. While efforts have been undertaken to incorporate the patient voice, the patient engagement movement has remained aspirational and tokenistic. Though there have been calls to grow the patient engagement movement, the number of trials reporting patient engagement remains extremely low. This article briefly discusses the reasons driving this gap and presents potential solutions that organizations can incorporate into their strategies to engage patients.

State of the Industry: Patient Engagement Implementation

Despite the movement to engage patients in clinical research, the number of studies reporting any form of patient engagement has remained dismally low. For instance, across 371,159 trials, only 23 reported patient engagement practices. Additionally, there is no uniform way of reporting patient engagement across trials, so patient engagement is reported in different locations within a research report. Further, the scope of information about the rationale for the type of patient engagement used and related methods provided varies widely, contributing to the irregularity and inconsistency associated with patient engagement implementation. Factors contributing to the inertia include but are not limited to internal resistance to modify legacy practices; lack of funding to staff and implement initiatives; lack of guidance; and lack of a standardized way to benchmark patient engagement activities. Consequently, drug development sponsors have remained aspirational in their support of patient engagement initiatives.

What is Being Done to Address This Patient Engagement Gap?

The lack of standardized metrics to measure and report patient engagement methods and activities is a major contributor to the current gap in patient engagement. To address this particular gap, the Drug Information Association (DIA) and Tufts Center for the Study of Drug Development (CSDD), in collaboration with a working group of stakeholders from the drug development industry and a patient steering group, collaborated in a multiphase study effort to develop an assessment tool that organizations can use to measure patient engagement capabilities. Throughout the multiphase study, the development and refining of the tool were informed by literature consultation, feedback, and input from a working group comprised of organizations that included sponsors, contract research organizations (CROs), consulting firms, vendors, and patient input.

Lessons Learned: Sponsor, CRO, and Patient Advocate

The following section presents testimonials from three working group participating stakeholders, representing the perspectives of a sponsor, a CRO, and a patient advocate. The sponsor and CRO stakeholders share their experiences with the Patient Engagement Capabilities Questionnaire (PECQ) and the insights they gathered from administering it in their organizations; the patient advocate shares how this can elevate the patient experience and voice.

Ellyn Getz: Director, R&D Patient Partnerships, CSL

“We were proud to be part of this industry collaboration to measure the impact that patient engagement activities have on the drug development process and study-level operational outcomes. From this experience, we identified the most and least commonly used patient engagement activities and spotlighted gaps in patient engagement efforts related to health equity. The survey tool was easy to implement, and it took less than 20 minutes for each person to complete. We recognized crossfunctional champions while the survey tool was in development, and these individuals provided feedback on the survey design and identified others within our R&D and commercial functions to participate in the assessment. Due to this early buy-in, we were pleased with the response rate and the diverse therapeutic areas represented among our respondents. One challenge identified across the industry collaborative was evaluating the impact of patient engagement activities on study performance outcomes, as obtaining this data is not standardized across companies. However, the research team was able to recognize that a more robust patient engagement plan (a higher PECQ score) does in fact impact study performance, most notably with respect to decreased substantial protocol deviations and adverse events.

“These benchmarked performance metrics and the patient engagement preparedness tool have strengthened our company’s patient focus by:

  • Establishing methods to further raise awareness about and value meaningful patient engagement activities;
  • Identifying new ways to co-create with patient advocates and lived experience experts; and
  • Obtaining insights to optimize our protocol development and operational design.

“We hope to routinely implement this internal assessment every two years to keep track of our progress in making our clinical trials more accessible, seamless, and integrated in the healthcare experience.”

Keri McDonough: Head of Patient Voice Consortium, Syneos Health

“Our internal Patient Voice Consortium jumped at the opportunity to team up with other leaders in the space to refine, disseminate, and encourage utilization of the PECQ. Our patient engagement, insights, and advocacy experts work closely with companies at all stages of patient engagement maturity. Across the board, they knew their customers needed an easy-to-use and replicable way to holistically assess the impact of well-intentioned yet sometimes disconnected and under-resourced patient engagement efforts. They also knew that this type of tool would be most impactful if built, tested, and endorsed by a multistakeholder initiative.

“In addition to seeing the strategic value this tool would deliver to its customers, our company used its individual PECQ report and the larger pool of aggregate data to further refine its suite of patient-focused solutions designed to ensure that the patient voice is represented at the forefront of innovation. To roll out the PECQ internally, we selected a representative mix of commercial, medical affairs, and clinical colleagues to feed into the assessment and work together to mine its output for the most salient and actionable insights. The output from the easy-to-use tool has already delivered significant value, and we plan to revisit the assessment on an annual basis and are encouraging customers to use it, too.”

Valerie Fraser: Patient and Research Advocate, SWOG Cancer Research Network, Inflammatory Breast Cancer International Consortium

“As a member of the Patient Steering Group of the Tufts CSDD and DIA on this project, I and other patient members of the group were able to bring the important patient voice to shape and facilitate discussion in the development of this study design. Patient engagement strategies in clinical research have never been more important than right now, especially in building equity and access to care, and reducing barriers for patients. Although there’s great interest in incorporating many patient engagement strategies within organizations, there is also a great need for developing a roadmap and more specific guidance. This is why this type of study is so valuable both for patients, organizations, and clinical trial developers. Through incorporating a patient engagement strategy that measures and reports patient engagement methods and activities, trials will have the opportunity to move forward in evolving to become more patient centric.

Effective communication in all forms is at the heart of patient-centered research and is so important to instill trust and assure patients that the trial design has taken their needs into consideration. All too often, patients who may be desperate for a new treatment, other tools, or who are just eager to learn, participate, and benefit from a clinical trial, can encounter challenges and disappointments with the experience. Satisfaction for patients goes a long way! It may come from materials and information that are culturally sensitive and written in ‘plain language,’ or from addressing up front what might be a patient concern. Including patients that reflect the study population in the study team is important to identifying some of these challenges. It is always comforting to know that the trial developers have considered the patient’s perspective and privacy, and that their concerns are respected and listened to in a courteous and compassionate environment. These points are also particularly important in developing the language for the Informed Consent, as this is often the first important communication the patient will receive. It should be easily readable and understood by the patient.

Patients participating in clinical research do so for the benefit from the treatment, but also for the benefit of future patients and to move science forward. Their expectation is that the therapy, drug, or intervention will be more effective, safe, and have less toxic side effects. They have an interest in knowing the end results of the trial as well as all personal treatment information that might impact their care. Patients can also provide valuable feedback to the study team, such as what they considered the pros and cons of their experience in the trial. Were they hopeful through the process? Would their experience support recommending the trial to other patients? A plan for gathering patient feedback would be important for moving the organization’s future clinical research forward as well as for patients to feel valued and included in the process of the trial. Having an assessment tool to guide organizations and trial developers in this process would most definitely drive patient-centered clinical research forward and address many of the concerns and critical communication gaps for patients.”

Call for Participants: Where Do We Go From Here?

As one of the first projects to systematically measure patient engagement efforts across sponsor organizations, this project represents a significant milestone in the field of patient engagement. The DIA-Tufts team will continue to administer the tool and collect additional data to increase the generalizability of the study findings. We encourage organizations to learn more about this research and utilize the assessment tool to diagnose and drive their own patient engagement efforts. As part of this effort, DIA and Tufts are aiming to collect outcome measures, including patient-reported outcomes, to provide empirical evidence of the positive impact of patient engagement on clinical and patient outcomes.

To learn more about or contribute to this effort, please contact Science@DIAglobal.org.