Developing a Process for Preference Measures in Pediatric Growth Hormone Deficiency: Challenges and Solutions.

Area of Science:

• * Patient-reported outcomes
• * Health economics and outcomes research
• * Drug development

Background:

• * Patient experience data is increasingly vital for drug development and risk/benefit analysis.
• * Assessing patient preferences is key to evaluating new therapies.
• * Pediatric Growth Hormone Deficiency (PGHD) treatment was used as a model for developing patient-centric measures.

Purpose of the Study:

• * To develop a prototype process for assessing patient-centric treatment preferences.
• * To create validated questionnaires for measuring patient preferences in PGHD.
• * To incorporate the patient voice into the drug development continuum.

Main Methods:

• * Literature review and concept elicitation interviews with experts, caregivers, and children with PGHD.
• * Data analysis using adapted ground theory.
• * Development and cognitive debriefing of the GHD-Preference Measure (GHD-PRM) and GHD-Attribute Measure (GHD-ATM).

Main Results:

• * Key caregiver preferences included ease of preparation, convenience, and side effects.
• * Treatment impacts on children's lives involved travel, social activities, and routines.
• * Findings were consistent across patient groups and treatment types (injectable vs. oral).

Conclusions:

• * The GHD-PRM and GHD-ATM are novel, rigorously developed, disease-specific preference questionnaires.
• * These measures provide valuable patient-centric data for various stakeholders.
• * Novel preference measures enhance risk/benefit analysis and inform treatment evaluations.