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Patient Preference and Adherence
Article . 2025 . Peer-reviewed
License: CC BY NC
Data sources: Crossref
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Dove Medical Press
Article . 2025 . Peer-reviewed
Data sources: Dove Medical Press
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Developing a Process for Preference Measures in Pediatric Growth Hormone Deficiency: Challenges and Solutions

Authors: Meryl Brod; Kathryn Pfeiffer; Suzanne Alolga; Jane Beck; Morgan Murphy; Aleksandra Bruchey; Aristides Maniatis; +1 Authors

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

Abstract

Patient experience data capturing the patient voice is gaining increasing recognition across the drug development continuum for use in risk/benefit analysis to evaluate new drugs. The aim of this study was to delineate a prototype process for and then, following this process, develop questionnaires to rigorously assess patient-centric treatment preferences, using pediatric growth hormone deficiency (PGHD) treatment as a model.A literature review and concept elicitation interviews with clinical experts (n=5), caregivers of children with PGHD (n=15), and children with PGHD (n=15) were conducted. Most respondents were on injectable treatments with a small subsample on an investigational oral treatment. Data were analyzed based on adapted ground theory, and the GHD-Preference Measure (GHD-PRM), and GHD-Attribute Measure (GHD-ATM) were developed. These questionnaires were cognitively debriefed, refined, and finalized. Best practices for patient-reported outcome measure development and guidelines on assessing patient preferences were followed.Beyond efficacy, some of the most important treatment aspects determining preference for caregivers were the ease of preparation/setup, convenience, and side effects. The most frequently reported reasons for missing, postponing, or changing their child's medication (eg, dosage) included travel/being away from home and flexibility of dosing. The most frequently reported treatment impacts on children's daily lives were travel/being away from home, social activities/relationships, and evening routine/schedule. Findings were generally similar between caregivers and children, and those on injectable vs oral treatment. The GHD-PRM is intended for use when treatment comparisons are appropriate; the GHD-ATM is intended for use when treatment comparisons are not available. Each has a caregiver and child version.The GHD-PRM and GHD-ATM can be considered disease-specific prototype preference and attribute questionnaires developed according to a rigorous patient-centric process. Novel, well developed preference measures such as these can provide valuable data to researchers, clinicians, regulators and reimbursement agencies.

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Keywords

Patient Preference and Adherence, Original Research

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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
0
Average
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Average
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