Supplementary Material for: Regulatory qualification of a cross-disease digital measure: benefits and challenges from the perspective of IMI consortium IDEA-FAST

Background: Innovative Medicines Initiative (IMI) consortium IDEA-FAST is developing novel digital measures of fatigue, sleep quality and impact of sleep disturbances for neurodegenerative diseases and immune-mediated inflammatory diseases. In 2022, the consortium met with the European Medicines Agency (EMA) to receive advice on its plans for regulatory qualification of the measures. This viewpoint reviews the IDEA-FAST perspective on developing digital measures for multiple diseases and the advice provided by the EMA. Summary: The EMA considered a cross-disease measure an interesting and arguably feasible concept. Developers should account for the need for a strong rationale that the clinical features to be measured are similar across diseases. In addition, they may expect increased complexity of study design, challenges when managing differences within and between disease populations and the need for validation in both heterogeneous and homogeneous populations. Key messages: EMA highlighted the challenges teams may encounter when developing a cross-disease measure though benefits potentially include reduced resources for technology developer and health authority, faster access to innovation across different therapeutic fields and feasibility of cross-disease comparisons. The insights included here can be used by project teams to guide them in the development of cross-disease digital measures intended for regulatory qualification.

背景：创新药物倡议（Innovative Medicines Initiative, IMI）旗下的IDEA-FAST联盟正针对神经退行性疾病与免疫介导性炎症疾病，开发疲劳、睡眠质量及睡眠障碍影响的新型数字化评估指标。2022年，该联盟与欧洲药品管理局（European Medicines Agency, EMA）会面，就相关指标的监管资格认定计划征求建议。本述评梳理了IDEA-FAST联盟针对多疾病数字化评估指标开发的观点，以及EMA给出的指导意见。

总结：EMA认为跨疾病评估指标是一项颇具吸引力且具备可行性的构想。研发团队需确保具备充分依据，证明拟评估的临床特征在不同疾病中具有一致性。此外，研发团队或将面临研究设计复杂度提升、需应对疾病人群内部及人群间的异质性差异挑战，以及需在异质性与同质性人群中完成验证的要求。

核心要点：EMA强调了研发跨疾病评估指标可能遇到的诸多挑战，但其潜在收益包括降低技术开发者与卫生主管部门的资源投入、加快不同治疗领域的创新落地，以及实现跨疾病对比的可行性。本文梳理的相关见解，可用于指导项目团队开展用于监管资格认定的跨疾病数字化评估指标开发工作。