Patient-Centric Design of the Lonapegsomatropin Auto-Injector for Growth Hormone Deficiency: Usability Validation of Safe and Effective Use.

Abstract

Purpose: Treatment of pediatric growth hormone deficiency (pGHD) with daily somatropin (human growth hormone [hGH]) injections is associated with challenges including needle anxiety, which may lead to poor adherence and ultimately result in suboptimal growth outcomes. The lonapegsomatropin (TransCon hGH, SKYTROFA^®) Auto-Injector, designed to deliver once-weekly lonapegsomatropin for the treatment of pGHD while minimizing injection challenges, was validated in usability studies in pGHD.

Patients and methods: The Auto-Injector was evaluated in a usability validation study conducted under simulated-use conditions in accordance with FDA guidance, involving simulated injections performed by injection-naive or injection-experienced patients with pGHD or proxy medical conditions, caregivers, and health care providers (HCPs) responsible for injections or training patients/caregivers. Half of the patient and caregiver participants received training by a Nurse Trainer.

Results: All participants (60 children, 60 caregivers, and 15 HCPs) were able to complete an injection successfully. All participants reported that they could follow the instructions as written, and 98% felt they could use the device as intended on their own or, for pediatric patients, with supervision. Use deviations observed for injection tasks for all participant groups were low: patients (3.2% of tasks), caregivers (2.4%), and HCPs (2.8%).

Conclusion: The user-centric design of the lonapegsomatropin Auto-Injector enables successful and confident use to deliver an injection as intended, with the potential to overcome known barriers associated with GH injections and facilitate adherence, which may improve treatment outcomes.