临床试验效率的提高在肿瘤学领域成功地得到证明，采用多臂、多阶段试验方法可以同时评估多个治疗候选。对于那些在中期有效信号测试后通过的候选者，可以无缝地招募到第三阶段。在像帕金森病这样的疾病中复制这种复杂的创新试验设计是很有吸引力的，但与评估单一潜在疾病修复干预措施的任何试验相关的挑战一样，多臂平台试验还必须特别考虑PD的异质性，以及可能测试具有不同作用机制的多种治疗方法的愿望。在多臂试验中，需要适当地分层治疗组，以确保每个治疗组与共享的安慰剂/标准护理组相当。然而，在PD中可能有一个偏好，即要富集一个子组病人，他们可能对特定治疗方法最有反应。解决这个难题的方法在于明确定义每个治疗组的纳入标准以及考虑预定义感兴趣的亚组，以及评估每种治疗对PD患者整体人群的影响的分析计划。此外，还必须制定强有力的治疗选择流程和共识衍生的测试标准，以确认目标参与和中期有效性评估，以及考虑支持招募的基础设施和平台的长期资金和可持续性。这必须包括临床医生，试验人员，监管机构和尤其是帕金森病患者的各种优先事项。 ©作者（2023发表）。由牛津大学出版社代表Brain的保证人出版

An increase in the efficiency of clinical trial conduct has been successfully demonstrated in the oncology field, by the use of multi-arm, multi-stage trials allowing the evaluation of multiple therapeutic candidates simultaneously, and seamless recruitment to Phase 3 for those candidates passing an interim signal of efficacy. Replicating this complex innovative trial design in diseases such as Parkinson's disease is appealing but in addition to the challenges associated with any trial assessing a single potentially disease modifying intervention in PD, a multi-arm platform trial must also specifically consider the heterogeneous nature of PD, alongside the desire to potentially test multiple treatments with different mechanisms of action. In a multi-arm trial, there is a need to appropriately stratify treatment arms to ensure each are comparable with a shared placebo/standard of care arm, however in PD there may be a preference to enrich an arm with a subgroup of patients that may be most likely to respond to a specific treatment approach. The solution to this conundrum lies in having clearly defined criteria for inclusion in each treatment arm as well as an analysis plan that takes account of pre-defined subgroups of interest, alongside evaluating the impact of each treatment on the broader population of PD patients. Beyond this, there must be robust processes of treatment selection, and consensus derived measures to confirm target engagement and interim assessments of efficacy, as well as consideration of the infrastructure needed to support recruitment, and the long-term funding and sustainability of the platform. This has to incorporate the diverse priorities of clinicians, triallists, regulatory authorities and above all the views of people with Parkinson's disease.© The Author(s) 2023. Published by Oxford University Press on behalf of the Guarantors of Brain.