Discrete Choice Experiment (DCE) Methodology Associated With a Multi-Phase Patient/Caregiver Preference Study in Growth Hormone Deficiency (GHD)

Speaker(s)

Chen JV1, Yuen KCJ2, Kelepouris N3, Shah S4, Mason B5, Wayser G6, Miller BS7
1Genesis Research Group, Fishers, IN, USA, 2Department of Neuroendocrinology, Barrow Pituitary Center, University of Arizona College of Medicine, and Creighton School of Medicine, Phoenix, AZ, USA, 3Novo Nordisk Inc, Plainsboro, NJ, USA, 4Jazz Pharmaceuticals Inc., Philadelphia, PA, USA, 5Adelphi Values Patient-Centered Outcomes, Bollington, Great Britain, 6Adelphi Research, Doylestown, PA, USA, 7Division of Pediatric Endocrinology, Department of Pediatrics, University of Minnesota Medical School, M Health Fairview Masonic Children's Hospital, Minneapolis, MN, USA

Presentation Documents

OBJECTIVES: Once daily (Q1D) short-acting growth hormone (SA-GH) and once weekly (Q1W) long-acting GH analog (LA-GHA) treatments are available to treat growth hormone deficiency (GHD) in children and adults. Three Q1W LA-GHA treatments injections received United States (US) regulatory approval as of 2023. Each regimen differs per device administration, dose preparation, storage/handling, and safety. Previous preference studies (e.g., discrete choice experiments [DCEs]) have quantified preference for SA-GH vs. LA-GHA use. However, no published studies have explored features among LA-GHA devices, independent of injection frequency. We outline DCE methodology associated with a three-phase study based in the US.

METHODS: DCEs measure and evaluate trade-offs made when deciding between treatment options. Phase 1 involved a targeted literature review to identify attributes and levels relevant to GHD patients/caregivers. Phase 2 involved conducting in-depth qualitative interviews with GHD patients/caregivers to substantiate attribute/level selection, identify any key missing attributes, and inform the design of the DCE. The Phase 3 DCE intends to recruit approximately 120 participants (caregivers of pediatric patients [0-11 years], caregiver/adolescent [12-17 years] dyads, adult patients with GHD [18 years+]). The planned total sample supports a 3-level grid design with up to 5-9 attributes. The caregiver/adolescent dyads will be moderator-assisted, to both mitigate completion burden and monitor response consistency.

Pilot testing (cognitive interviews [n=12]) will confirm usability of the survey instrument. The DCE analysis will output: 1) Estimated part-worth utilities using hierarchical Bayesian estimation, 2) Relative attribute importance, 3) Patient/caregiver trade-offs, and 4) Estimated preference shares for profiles corresponding to available LA-GHAs alongside choice share changes per adjustments to levels. Meaningful differences in preferences between patient/caregiver subgroups may be explored depending on sample size.

RESULTS: Expected in 2024.

CONCLUSIONS: Results will provide insights into patient/caregiver preferences and trade-offs related to LA-GHA device attributes for GHD devices, thereby facilitating shared patient/caregiver-physician decision-making.

Code

PCR27

Topic

Patient-Centered Research

Topic Subcategory

Instrument Development, Validation, & Translation, Patient Behavior and Incentives, Patient Engagement, Stated Preference & Patient Satisfaction

Disease

No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases