Enhancing Neurofibromatosis Clinical Trial Outcome Measures Through Patient Engagement: Lessons From REiNS

Abstract

Objective: As part of an evaluation of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration patient representative program, we surveyed REiNS members to (1) identify facilitators and barriers to involving patient representatives and (2) understand whether and how involving patient representatives affected recommendations for clinical trial outcomes.

Methods: We administered an anonymous online survey to all REiNS members. Facilitators and barriers to patient representative involvement were solicited using a modified free listing technique; responses were inductively grouped into higher-order categories and ranked based on saliency score (Smith s). Open-ended questions assessed patient representative expectations for engagement, perceived benefits/costs of patient engagement, and patient representative contributions; responses were analyzed using conventional content analysis.

Results: A total of 63/172 (37%) members responded, including 18/30 (60%) patient representatives. Providing sufficient opportunities to meaningfully engage in research tasks and cultivating a respectful, inclusive atmosphere were key facilitators to patient representatives' satisfaction and ability to make an impact. Respondents perceived that patient representatives directly (through their input on research tasks) and indirectly (through effects on other stakeholders' knowledge and communication style) improved the organization's research, leading to selection of more meaningful, relevant, and feasible clinical trial outcome measures. Ongoing challenges to patient engagement include difficulty scheduling meetings and concerns about the level of scientific knowledge patient representatives needed to effectively engage.

Conclusions: Involving patient representatives in REiNS improved perceived quality of neurofibromatosis clinical trial outcome measures. Negotiating sufficient opportunities to engage, fostering an inclusive atmosphere, and navigating time pressures are key to effective patient engagement.

Figure 1. Patient Representatives' Reasons for Engaging

Scree plot of coded free listing responses describing why patient representatives joined the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) research collaboration. The y-axis is calculated saliency score (Smith s) for each code and the x-axis is codes presented in ranked order by saliency score.

Figure 2. Factors Affecting Whether Patient Representatives' Expectations for Engagement Were Fulfilled

Graphic depicting the 3 major contributors to fulfilling patient representatives' expectations for their role (as derived from content analysis of survey responses) and the resulting effect of achieving these goals on patient representatives' satisfaction. Having sufficient opportunities to engage was the foundation for building an inclusive climate, which facilitated patient representatives' ability to have an effect on research.

Figure 3. Patient Representative Contributions to and Effect on Clinical Trial Outcomes

Graphic depicting how and why patient representatives were perceived to have improved Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) recommendations for neurofibromatosis clinical trial outcomes, as derived from content analysis of evaluation survey responses.