Drivers of Patient and Caregiver Preferences for Growth Hormone Deficiency Treatments in France: A Discrete Choice Experiment

INTRODUCTION: The aim of this study was to identify drivers of preference for growth hormone deficiency (GHD) treatment in French children, and their caregivers, and to quantify the relative importance of different aspects of treatment modalities using a discrete choice experiment (DCE). MATERIALS AND METHODS: Attributes characterizing GHD treatment modalities were identified through a literature review, qualitative interviews, and focus groups with children, adolescents, and caregivers. A DCE questionnaire of 12 choice tasks was administered online to four groups of participants: autonomous adolescents (12-18 years), nonautonomous adolescent/caregiver dyads, caregivers of nonautonomous children (3-11 years), and autonomous children/caregiver dyads. The survey was pilot tested. A multinomial logit model with random effects was used to estimate preference weights for all attribute levels. RESULTS: Frequency of administration, injection pain, dose setting, type of device, storage, and device reusability were selected as DCE attributes following the qualitative research phase and a pilot study. A total of 105 patients were represented in the DCE survey. Frequency of administration and injection pain were the attributes with the greatest influence on respondents' preferences and had similar importance. Weekly administration was significantly preferred over daily administration by all groups of participants. Respondents' choices were also significantly influenced by the type of device, dose setting, and device reusability. CONCLUSION: Children with GHD and their caregivers prefer a less frequent injection schedule and lower injection pain. Both aspects of treatment modalities are important to consider in treatment decisions to alleviate the daily burden for GHD patients and their families and potentially enhance treatment adherence.