Qualitative understanding of experiences of people with cystic fibrosis in a treatment discontinuation trial: The QUEST study.

Abstract

Background: As people with cystic fibrosis (pwCF) live longer due to the breakthrough drug elexacaftor-tezacaftor-ivacaftor (ETI), they have questioned whether other CF therapies could be safely discontinued. SIMPLIFY was the first prospective, randomized trial to evaluate non-inferiority of discontinuing versus continuing two therapies. The QUEST (Qualitative Understanding of Experiences in the SIMPLIFY Trial) study was conducted to understand experiences of pwCF enrolled in SIMPLIFY, including why they joined, perceptions of randomization, decision-making around study withdrawal, and considerations for future discontinuation studies.

Methods: QUEST enrolled SIMPLIFY participants 14 years or older stable on ETI and caregivers of the 14-17 year-olds. Interviews were audio-recorded, transcribed, and coded. A phenomenological approach was used to inductively develop codes with no a priori hypotheses; identified themes were then organized around current research and recruitment literature.

Results: 114 interviews were completed (68 adults, 23 teenagers, and 23 caregivers). Among pwCF, median age was 27.8 years, 49 % were female and 80 % had participated in research before SIMPLIFY. Five themes were identified: ((Gul and Ali, 2010 [1]) Experience with SIMPLIFY randomization, CFF Patient Registry Annual Data Report [Internet] (2017) [2] Trust, CFF Patient Registry Annual Data Report [Internet] (2022) [3] Altruism, Goss et al. (2008) [4] Perceived personal benefits, and (Goss et al., 2002 [5]) Perceived risks and protocol burden.

Conclusion: QUEST findings highlight how a long-standing culture of research and thoughtful protocol design contributed to SIMPLIFY's successful recruitment and retention. This included understanding the importance of remaining in the trial despite not being randomized to their preferred treatment assignment. Using patient-centered approaches to select research questions, design a protocol to minimize participant barriers, and frame recruitment materials messaging contribute to successful research participation.