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

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 if 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 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: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.

Further content validation of the 18-item NCCN/FACT Ovarian Symptom Index and its Disease Related Symptom-Physical (DRS-P) subscale for use in advanced ovarian cancer clinical trials

Background This study evaluated pre-defined aspects of content validity of the 18-item NCCN FACT-Ovarian Symptom Index (NFOSI-18) and its Disease-Related Symptoms-Physical (DRS-P) subscale, as clinical trial outcome tools for patients with advanced ovarian cancer. Methods Twenty-one women (mean age 59.5 years) diagnosed with advanced ovarian cancer completed the NFOSI-18 and participated in a cognitive interview to explore: (1) whether ‘pain’ and ‘cramps’ are considered redundant; (2) whether ‘fatigue’ and ‘lack of energy’ are overlapping concepts; (3) whether patients consider severity when responding to the item “I am bothered by constipation;” and (4) factors considered when responding to the item “I am sleeping well.” Interviews were audio-recorded, transcribed, and analyzed qualitatively. Results Pain was associated with discomfort, hurt, and life interference; ‘cramps’ was associated with pain, muscle tightening, and menstrual or digestive issues. Most (81%) considered the items “I have pain” and “I have cramps in my stomach area” to be more different than similar. Participants associated ‘fatigue’ with intense tiredness and ‘lack of energy’ with motivation and capability to complete daily activities. Item comparisons revealed a majority (65%) considered the items to be more different than similar. When responding to “I am bothered by constipation,” patients indicated constipation severity was related to bother. Finally, patients considered disease, treatment, and other factors when responding to “I am sleeping well.” Conclusions Findings support content validity of the NFOSI-18 and its DRS-P as originally constructed. We propose an alternative scoring option that excludes the item “I am sleeping well” from the DRS-P when used as a symptom-focused index for clinical research in a regulatory context.