Patient and Caregiver Perspectives Regarding Once-Weekly Injection Devices in Growth Hormone Deficiency: Qualitative Interviews To Inform a Patient Preference Study

OBJECTIVES: Growth hormone deficiency (GHD) is a rare disease estimated to affect approximately 1 in 3,500 to 10,000 children. Treatments typically include once-daily injections; however, the treatment landscape may be changing with the introduction of once-weekly long-acting growth hormone analogs (LA-GHAs). This study explored the perspectives of patients with GHD and their caregivers (CGs) regarding LA-GHA injection devices to inform the development of a quantitative patient preference survey.

METHODS: A draft attributes and levels (A&L) grid was developed following a targeted literature review of available clinical literature, product labels, peer-reviewed patient-focused literature, and other publicly available information. Twenty participants based in the USA were interviewed: adults with GHD aged ≥18 years (n=6); adolescents with GHD (aged 12–17 years) with a CG (n=8); and CGs (n=6) of children with GHD (aged <12 years). Qualitative interviews used an in-depth, think-aloud, cognitive debriefing approach to assess content validity of the A&L grid. The interviews examined the perceived importance of attributes, perceived impact on decision-making, and meaningfulness of level differences.

RESULTS: More than two-thirds of participants considered “injection device type”, “how to set the dose”, “preparation time”, “storage”, and “injection-site pain” to be important attributes that would impact GHD treatment decisions, expressing a preference for devices with simple preparation steps, minimal injection-site pain, and high room-temperature stability. The attributes “injection time” and “needle visibility” were considered less important and impactful relative to other attributes.

CONCLUSIONS: Patients and CGs value a once-weekly LA-GHA device that is convenient to use with minimal injection-site pain. Findings from this qualitative study and upcoming pilot interviews will inform a larger preference study that will quantify the relative importance of attributes corresponding to LA-GHA devices and explore the trade-offs that patients and CGs are willing to make.