Qualitative Understanding of Experiences With the SIMPLIFY Trial

Overview

This qualitative longitudinal study is designed to elicit and thematically analyze the perspectives of SIMPLIFY subjects about treatment withdrawal research and treatment burden in the context of triple combination CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator therapy (TCT). Cohorts will be determined based on sub-groups of the assigned treatment arms from the SIMPLIFY study. Participants will be asked to participate in two interviews; the first interview will be conducted at the completion of the SIMPLIFY study and the second interview will be conducted approximately 4 months after the first interview. Trained research staff will conduct the semi-structured interviews that will be audio-recorded and transcribed for thematic analysis. Each interview should last between 30-60 minutes.

The recommended daily treatment regimen for CF is complex and results in high treatment burden for many people with CF (PwCF). Newly developed triple combination CFTR modulator therapy (TCT) has shown dramatic health benefits on top of standard CF therapies in a pivotal clinical trial and will soon be a treatment option for a larger population of PwCF. Widespread implementation of TCT will provide an unprecedented opportunity to rigorously study whether PwCF can safely discontinue some previously prescribed daily therapies. The randomized, open-label SIMPLIFY study has been designed to test the hypothesis that withdrawal of dornase alpha and/or nebulized hypertonic saline (HS) is not inferior to continuation of one or both treatments with respect to short-term change in lung function. As this is the first of its kind study design in CF, it unknown how people with CF thinking about withdrawal studies or discontinuing treatments. Here, the investigators intend to conduct a qualitative interview study that complements the SIMPLIFY protocol in which the investigators aim to assess the experiences of PwCF participating in a treatment withdrawal study. In particular, the investigators will examine post-hoc the beliefs and opinions of a representative subgroup of SIMPLIFY participants about treatment withdrawal research and perceived treatment burden. This qualitative study will provide the opportunity to learn about behaviors after the SIMPLIFY trial, including restarting vs. continuing to maintain medications, withdrawing other medications or therapies, and how patients and families partner with care teams around these decisions. Through these interviews, the investigators will learn how individuals with CF perceive taking part in a drug withdrawal study and how participants discern treatment assignment.