Novel therapies with the ability to delay disease progression are a gap in the care of people living with Parkinson disease (PD) today. Clinical outcomes assessments (COAs) that are sensitive to the earliest clinical changes in PD are deemed essential for a successful therapeutic development. To understand the current landscape of COAs use in clinical trials in PD and define priorities for future research in the field, a stakeholder roundtable meeting was held in November 2022. The current paper 1) proposes the collaborative development of patient-centric COAs that can adequately document the effectiveness of disease modification therapies in PD based on key priorities identified during this initial meeting, 2) summarizes the progress made in the subsequent 12 months, and 3) presents the deliverables expected in the near future. Key priorities include 1) the development of a consensus conceptual model of early PD experiences, 2) the adaptation of existing patient-reported outcomes (PROs), 3) the investigation of the role of observer-reported outcomes in addition to 4) enabling diversity in PD research and advocacy, 5) fostering data sharing, and 6) reaching consensus on a biological staging system for PD to drive the development of appropriate PROs for biologically defined populations.

中文翻译：

---

以患者为中心的早期帕金森病临床结果评估的发展：关键优先事项和进展

能够延缓疾病进展的新疗法是当今帕金森病 (PD) 患者护理中的一个空白。对 PD 最早临床变化敏感的临床结果评估 (COA) 被认为对于成功的治疗开发至关重要。为了了解 COA 在 PD 临床试验中的使用现状并确定该领域未来研究的优先事项，利益相关者圆桌会议于 2022 年 11 月举行。当前论文 1) 提出合作开发以患者为中心的 COA，可以充分根据首次会议期间确定的关键优先事项，记录帕金森病疾病缓解疗法的有效性，2) 总结随后 12 个月取得的进展，以及 3) 介绍近期预期的可交付成果。主要优先事项包括 1) 开发早期 PD 经历的共识概念模型，2) 适应现有患者报告结果 (PRO)，3) 调查观察者报告结果的作用，此外 4) 实现多样性帕金森病研究和宣传，5) 促进数据共享，6) 就帕金森病生物分期系统达成共识，以推动针对生物学定义人群开发适当的 PRO。