Brod Meryl, Pfeiffer Kathryn M, Alolga Suzanne Lessard, Beck Jane F, Murphy Morgan, Bruchey Aleksandra K, Maniatis Aristides, Pitukcheewanont Pisit
Health Outcomes Research, The Brod Group, Mill Valley, CA, USA.
Research & Development, Lumos Pharma, Inc., Austin, TX, USA.
Patient Prefer Adherence. 2025 May 8;19:1365-1384. doi: 10.2147/PPA.S500330. eCollection 2025.
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.
在药物研发的整个过程中，收集患者声音的患者体验数据在用于风险/效益分析以评估新药方面越来越受到认可。本研究的目的是描绘一个原型流程，然后按照这个流程开发问卷，以严格评估以患者为中心的治疗偏好，以儿童生长激素缺乏症（PGHD）治疗为模型。
对临床专家（n = 5）、PGHD患儿的照料者（n = 15）和PGHD患儿（n = 15）进行了文献综述和概念激发访谈。大多数受访者接受注射治疗，一小部分接受研究性口服治疗。基于改良的扎根理论对数据进行分析，并开发了生长激素缺乏症偏好量表（GHD-PRM）和生长激素缺乏症属性量表（GHD-ATM）。这些问卷经过认知反馈、完善并最终确定。遵循了患者报告结局测量开发的最佳实践以及评估患者偏好的指南。
除疗效外，决定照料者偏好的一些最重要的治疗方面是准备/设置的简便性、便利性和副作用。最常报告的错过、推迟或更改孩子药物（如剂量）的原因包括旅行/离家外出和给药的灵活性。最常报告的治疗对儿童日常生活的影响是旅行/离家外出、社交活动/人际关系以及晚间日常安排/时间表。照料者和儿童之间以及接受注射治疗与口服治疗的人群之间的结果总体相似。GHD-PRM适用于进行治疗比较时；GHD-ATM适用于无法进行治疗比较时。每个量表都有照料者版和儿童版。
GHD-PRM和GHD-ATM可被视为根据严格的以患者为中心的流程开发的特定疾病原型偏好和属性问卷。这样新颖、完善的偏好测量可为研究人员、临床医生、监管机构和报销机构提供有价值的数据。
