(281) Experiences and Decision-Making with Highly Effective CFTR Modulator Therapy: Preliminary Findings from a Qualitative Study

Background: The introduction of highly effective CFTR modulator therapies (HEMT), including Ivacaftor, Tezacaftor/Ivacaftor, and Elexacaftor/Tezacaftor/Ivacaftor, have ushered the Cystic Fibrosis (CF) community into an unprecedented era of hope. While the potential of HEMT is life-altering, important physical and psychosocial needs remain for people with CF (pwCF). PwCF who are eligible for HEMT face uncertainty related to their disease course, their response to HEMT, and a future that may look very different than previously imagined. PwCF who are ineligible for HEMT also face many challenges including feeling “left behind” or uncertain about future decisions related to other aspects of their CF care. Overall, the advent of HEMT represents a transformative moment for the CF Community and, regardless of eligibility for HEMT, the experiences and medical decision making (MDM) processes of pwCF, their families, and their care teams need to be characterized and understood so that they may be effectively addressed.

Methods: Semi-structured qualitative interviews were conducted with pwCF, parents of pwCF, and care team members at four pediatric and three adult CF centers. The Ottawa Decision Support Framework was used as a theoretical model for developing the initial interview guides. Interviews were conducted remotely and transcribed prior to coding. A priori codes were generated by the study team to develop initial coding guides for the interview transcripts, and additional codes were generated in ongoing transcript review. All transcripts were coded by at least two members of the study team, and all discrepancies were resolved by full group consensus. After the completion of all interviews, categories and themes will be generated to summarize the qualitative data.

Results: All interviews completed and coded by October 2022 will be included. To date, 27 interviews have been conducted with pwCF (n = 21; non-binary = 1, male = 9, female = 17; Mage = 36 years old), parents of pwCF (n = 3), and multidisciplinary care team members (n = 4). Most interviews so far have been with pwCF or parents of pwCF who are eligible and taking HEMT (n = 16). Interviews have also been conducted with pwCF who are eligible but not taking HEMT, and pwCF or parents of pwCF ineligible for HEMT. Preliminary findings suggest that the experiences of pwCF in the era of HEMT are not homogeneous. Emerging categories from coding indicate that many factors impact MDM and there are several avenues for support. For example, some pwCF described a need for more data about HEMT, while others describe feeling that the decision to start HEMT is not experienced as a choice. Decisions are informed by care team members and the broader CF community. PwCF have discussed behaviors that are not helpful for MDM, such as feeling rushed or pressured in clinic. Initial interviews with CF team members suggest that providers have personal beliefs about HEMT that contribute to practice changes.

Conclusions: The experiences and needs of pwCF in the era of HEMT are diverse and wide ranging. In addition to describing the experiences of MDM in the era of HEMT, results from this study will be used to generate patient and provider resources to support and help guide the MDM process.

Acknowledgements: This work was supported by the Cystic Fibrosis Foundation [CANTER21A0-I]. We acknowledge collaborators and participants from Nemours Children’s Health, the Johns Hopkins Medical Institute, the University of Kansas Medical Center, and the University of Pennsylvania.

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