Research Overview
Our research at the Rare Disease Real-World Data Lab focuses on generating evidence-based insights to improve the quality of life and reduce the disease burden for rare disease patients and their families. We utilize a multidisciplinary approach, integrating quantitative and qualitative methods, to build comprehensive real-world data and inform precision support and interventions.
Selected Research Projects & Activities
Details on our recent and ongoing research projects and activities, grouped by start year:
2024
- Report of Dravet Syndrome: Designed and implemented a nationwide survey with teams from Peking University International Center for Pharmaceutical Management Research, Peking University First Hospital, Beijing Children's Hospital, and Representatives of patients with Dravet Syndrome. (January 2024)
- Qualitative Validation Project for the EQ-5D-5L Scale in Patients with Duchenne Muscular Dystrophy (DMD): Initiated a qualitative validation project with Professor Xu Huan (The Hong Kong Polytechnic University). (January-February 2024)
- Mixed-Method Study on Special Dietary Needs and Natural History of Rare Disease Patients: Conducted a comprehensive study in extensive collaboration with Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai Pediatric Medical Research Institute, Shanghai Children's Rare Disease Diagnosis and Treatment Center, and The Illness Challenge Foundation. (May - December 2024)
- Establishing Collaborative Partnerships and Fieldwork in ART Research: Built research collaborations and conducted fieldwork with the Department of Reproductive Medicine, First Affiliated Hospital of Lanzhou University and the Department of Reproductive Medicine, Qinghai Provincial People's Hospital, and collaborated with Professor Zhu Jianfeng, Fudan University. (August - October 2024)
- ART Symposium: "Making a Better Baby: Social and Ethical Issues in Assisted Reproductive Technologies": Hosted an academic symposium in Shanghai bringing together experts from institutions including The Chinese University of Hong Kong, Fudan University, Chulalongkorn University (Thailand), and several leading hospitals. (December 28, 2024)
2022
- Investigating the Impact of Quality of Life in Patients with Advanced Hodgkin Lymphoma: Examined quality of life aspects, funded by Hongmian Cancers and Rare Disorders Charity Foundation of Guangzhou. (March 2022 - December 2023)
- 2022 Health Report on Myasthenia Gravis Patients in China: Conducted a survey and published a report, funded by Beijing Aili Myasthenia Gravis Care Center. (April - July 2022)
- Exploring the Patient Journey of and Translating/Validating Research Instruments for Patients with Dravet Syndrome in China: Focused on patient experience and research tools, funded by Jingdezhen Dravet Syndrome Patient Service Center. (December 2022 - December 2023)
2020
- Real-world Research of People with Rare Diseases in China: A broad national study funded by Beijing Society of Rare Disease Clinical Care and Accessibility. (July 2020 - June 2023)
Future Directions
Looking ahead, our research will focus on:
- Empirical Guidance for Practice: Utilizing empirical data to inform and guide clinical and nursing practices for rare disease patients, providing scientific evidence for policy formulation.
- Single-Disease Registry and Evaluation: Establishing systems for specific rare disease registries and quality of life assessment frameworks to enable long-term monitoring of disease progression and outcomes.
- Fostering Integration of ART and Rare Disease Research: Expanding research into the application and potential impacts of Assisted Reproductive Technologies (ART) within rare disease populations, addressing their unique sociocultural needs and challenges from a sociological perspective.