Rare Disease Real-World Data Lab

Dedicated to studying the quality of life and disease burden among rare disease patients. We deeply explore the critical stages and needs in their care journeys to facilitate the development of precise support and intervention measures.

Core Research Directions

Patient-Centered Database Construction

Establishing a real-world research database based on self-reported data from rare disease patients and their primary caregivers, comprehensively capturing patient experiences.

Quality of Life and Disease Burden Assessment

Investigating the impact of rare diseases on patients' and families' quality of life and disease burden through in-depth analysis.

Multistakeholder Social Surveys

Conducting comprehensive social surveys targeting rare disease patients, patient organizations, and medical professionals to understand diverse needs and perspectives.

Assisted Reproductive Technology Expansion Research

Employing ethnographic methods to observe and explore the development of assisted reproductive technologies across regions, including Shanghai, Lanzhou, and Thailand.

Impact and External Engagement

Beyond our research activities, our lab is committed to translating findings into real-world impact and fostering collaboration within the rare disease community. Our work, including collaboration with patients, NGOs, grassroots patient organizations, medical professionals, anthropologists, and sociologists, has contributed to policy advancements at both national and international levels.

Research Outcomes & Value

  • Evidence-Based Practice: We provide empirically-driven guidance for clinical and care practices for rare disease patients, offering scientific evidence for relevant policy development.
  • Disease-Specific Registries: Our lab has established disease-specific patient registries and quality of life assessment systems that track the long-term progression of individual rare diseases.
  • Academic Achievements: We have published 2 monographs in Chinese and English, 55 SCI & SSCI academic papers related to our laboratory's research focus, led 6 academic research projects, and secured over 1.8 million RMB in research funding.

Our Director, Dr. Dong Dong, plays key roles in organizations including Asian Pacific Alliance of Rare Disease Organisations (APARDO), China Alliance for Rare Diseases, and Rare Disease Hong Kong (RDHK).