Patient and family perspectives on clinical trial participation in cystic fibrosis during the modulator era
Rajeshree Joshi, Heidi Stapp, Samantha Lazaro, Erin Carrick, Neha Purohit, Nicole Wimmer, Sharad Purohit, Varsha Taskar, Caralee Forseen, Kathleen McKieAbstract
Introduction:
This study evaluates the impact of a research education initiative aimed at enhancing patient and family engagement in cystic fibrosis (CF) clinical trials. The primary objective was to understand patient perspectives to support increased enrollment and improve trial selection by aligning studies with patients’ psychosocial considerations.
Materials and Methods:
This single-center cross-sectional survey study was conducted between May 2023 and April 2024. Adults with CF (AwCF) and parents/families of children with CF (PfCF) received 1 h of nurse-led research education during a clinic visit; knowledge and perspectives were then assessed via survey.
Results:
Of 153 participants, 74 were AwCF and 79 were PfCF. 37.3% were initially unaware of clinical research and opportunities. Despite 56.2% previously declining participation due to side effect concerns, the intervention led to 75% reporting improved understanding and 94.8% expressing future willingness to enroll. Key motivators included physician recommendations and the desire to contribute to new cures. Physician-led discussions significantly fostered patient empowerment and engagement.
Conclusion:
In advancing care for the chronic and rare disease of CF, this study supports a patient-centered clinical research model that prioritizes education, builds trust, and aligns trial design with the psychosocial needs, preferences, and lived experiences of the CF community.