2024-05-26-艾昆纬-使用患者体验数据评估医疗干预措施——第7章（英）_43页_761kb

Chapter 7 Summary: Generating Patient Experience Data (PED) in Complex Populations and Programs

Pediatric Populations

• Children aged 5–8 may have limited ability to self-report experiences due to cognitive immaturity, while those aged 8–11 can engage in some qualitative research with proper support.
• Adaptive technologies (e.g., visual aids, digital tools) and interviewer training (e.g., avoiding childish language) are recommended to facilitate PED collection.
• PRO instruments must use age-appropriate language, simple response scales, and cognitive accessibility principles to avoid bias.

Central Nervous System (CNS) Disorders

• Patients with CNS disorders often face physical, cognitive, or sensory limitations that hinder self-reporting; qualitative interviews and observer-reported outcomes (ObsROs) may supplement patient insights.
• DHTs (e.g., wearables) can passively capture behavioral data, but rigorous validation is needed for clinical use.
• Mixed-methods approaches (qualitative + quantitative) are essential for contextualizing treatment-related benefits.

Cancer Programs

• High attrition, disease progression, and use of accelerated trial designs (basket/umbrella trials) complicate PED generation.
• PRO instruments must align with FDA/EMA guidelines for core outcomes (e.g., symptoms, physical function), and digital tools can support real-time data collection.
• Ethical considerations include managing open-label bias and addressing missing data in small-sample trials.

Rare Disease Programs

• Limited patient populations and heterogeneity require leveraging patient advocacy networks and mixed-methods strategies (e.g., concept elicitation + adapted PROs).
• Regulatory agencies emphasize the importance of QoL outcomes and patient preferences, but methodological challenges persist.
• DHTs offer potential for global collaboration but face barriers in implementation (e.g., data harmonization, training).

Key Takeaways

• Tailored approaches (e.g., proxy reporting, cognitive accessibility, DHTs) are needed across complex populations.
• Future PED strategies should integrate qualitative insights, innovative technologies, and ecologic validity to enhance decision-making in healthcare.