Pfizer Inc, 235 East 42nd St, New York, NY, 10017, USA.
Endpoint Outcomes, Boston, MA, USA.
Ther Innov Regul Sci. 2022 May;56(3):454-463. doi: 10.1007/s43441-022-00377-1. Epub 2022 Feb 22.
The United States Food and Drug Administration is developing a series of patient-focused drug development guidance documents regarding the collection of patient experience data, including methods for understanding treatment benefit from the patient perspective. The goal of this research was to investigate the concern that the global impression of change scale is subject to recall error and thus not optimal for use as an anchor for estimating meaningful within-person change thresholds. We explored whether memory assistance for recalling baseline status would make a difference in how study participants diagnosed with Parkinson's disease (PD) responded to a patient global impression of change (PGIC) and patient global impression static (PGIS) item.
The research was completed as a secondary objective of a non-interventional 28-day (± 4 days) study among persons with Parkinson's disease and associated motor fluctuations. At baseline, participants completed the PGIS and then recorded a voice message to their future self in which they spoke about how their PD had affected their "day-to-day" activities over the preceding few days. At the final visit, the PGIC and PGIS were completed, after which participants listened to their memory assistance voice recording, and then completed both items for a second time to calculate a memory-assisted global impression static and change scores (MAGIS and MAGIC, respectively). Spearman correlations (ρ) were examined for the pre- and post- memory assistance evaluations. The degree of agreement pre- and post-memory assistance was quantified using the Shrout & Fleiss intraclass correlation coefficient (ICC [2,1]). An ICC(2,1) ≥ 0.7 served as the pre-specified criterion of acceptability for both the ρ and ICC(2,1) values.
Participants in the analytic sample were mean age 68.7 and mostly white (91.7%) and male (69.4%). The average length of time since PD diagnosis was 6.5 years. Correlations between the PGIS and MAGIS were ρ = 0.88; correlations between PGIC and MAGIC were ρ = 0.86. The estimated ICC(2,1) for both the PGIS/MAGIS and PGIC/MAGIC exceeded target success criterion of ICC(2,1) ≥ 0.70.
Our results show that the MAGIS/MAGIC methodology is feasible and that memory assistance did not substantially alter the PGIS/PGIC scores at the final visit.
美国食品和药物管理局正在制定一系列以患者为中心的药物开发指导文件,内容涉及患者体验数据的收集方法,包括从患者角度理解治疗获益的方法。这项研究的目的是调查一种担忧,即变化整体印象量表可能会受到回忆误差的影响,因此不适合用作估计有意义的个体内变化阈值的基准。我们探讨了记忆辅助是否会影响诊断为帕金森病(PD)的研究参与者对患者整体印象变化(PGIC)和患者整体印象静态(PGIS)项目的反应。
这项研究是一项为期 28 天(±4 天)的非干预性研究的次要目标,研究对象为帕金森病患者及其相关运动波动。在基线时,参与者完成 PGIS 后,录制一段语音信息,向未来的自己讲述过去几天 PD 如何影响他们的“日常生活”活动。在最后一次就诊时,完成 PGIC 和 PGIS,然后参与者收听记忆辅助语音记录,然后再次完成这两个项目以计算记忆辅助整体印象静态和变化评分(分别为 MAGIS 和 MAGIC)。检查预记忆辅助和后记忆辅助评估之间的斯皮尔曼相关系数(ρ)。使用 Shrout 和 Fleiss 组内相关系数(ICC[2,1])来量化记忆辅助前后的一致性程度。ICC[2,1]≥0.7 作为 ρ 和 ICC[2,1]值的可接受性的预定标准。
分析样本中的参与者平均年龄为 68.7 岁,大多数为白人(91.7%)和男性(69.4%)。PD 诊断后的平均时间为 6.5 年。PGIS 与 MAGIS 之间的相关性为 ρ=0.88;PGIC 与 MAGIC 之间的相关性为 ρ=0.86。PGIS/MAGIS 和 PGIC/MAGIC 的估计 ICC[2,1]均超过了 ICC[2,1]≥0.70 的目标成功标准。
我们的结果表明,MAGIS/MAGIC 方法是可行的,并且记忆辅助在最后一次就诊时并未显著改变 PGIS/PGIC 评分。