Patient perspectives of participation in clinical studies in late-onset Pompe disease: Structured exit interviews from cipaglucosidase alfa plus miglustat studies

Background Cipaglucosidase alfa plus miglustat (cipa+mig) is an approved treatment in many countries for adults living with late-onset Pompe disease. Objectives To capture patient insights into clinical studies. Design We conducted an exit interview study based on the 2022 US Food and Drug Administration (FDA) guidance recommending structured interviews. Methods We conducted structured exit interviews with participants (≥18 years) in the UK or USA who completed the final visit or had data available at the final data cut-off date for three long-term clinical trials (ATB200-02 [NCT02675465], ATB200-04 [NCT03911505], and ATB200-07 [NCT04138277]) and from the cipa+mig UK Early Access to Medicines Scheme (EAMS). Participants were asked to rate their experience with the study conduct, using a two-component therapy, and disease-specific and general quality-of-life (QoL) outcomes. Results Of 91 eligible participants, 23 (13 male) completed an exit interview (clinical trials n=19; EAMS n=4). For most participants (83%), looking for another treatment option and/or wanting to contribute to drug development was the motivation for joining a study. Almost all participants stated they were provided with appropriate information before joining the study (96%), and the time commitment was acceptable (91%). Most participants (74%) did not find the process of taking the two-component therapy challenging, while the remaining 26% had neutral experiences. Around half (52%) rated the fasting process before taking mig as very convenient or convenient; 39% were neutral, and 9% rated the fasting process as mildly inconvenient. Finally, 57% of participants felt that cipa+mig provided benefits and improved their QoL. Conclusion Overall, alongside perceived improvements in disease-specific and QoL outcomes, participants found taking the two-component therapy neither challenging nor inconvenient. These exit interviews provide valuable insights into patient perspectives of cipa+mig and study processes not captured using standard patient-reported outcome measures and should be considered in future studies in line with FDA guidance.