China – significant strides are being made in the diagnosis and treatment of rare diseases, largely driven by the active involvement of patient groups in developing patient registries and conducting survey research. This collaborative approach is revolutionizing the way rare diseases are understood and treated in the country.
Progress in Rare Disease Treatment and Research
According to World Economic Forum, rare disease patient groups in China have become increasingly active, with about 75% of the 130 groups participating in patient registries or conducting surveys. These efforts have substantially contributed to improving access to medications, reforming healthcare towards patient-centered and value-based models, and developing real-world data systems.
Impact of Patient Group Research
Patient groups have been instrumental in creating extensive patient registries and initiating survey research. Their contributions are enhancing the quality and quantity of data available, thereby playing a vital role in promoting rare disease diagnosis and treatment as well as in shaping medical programs.
Challenges and Recommendations
Despite these achievements, there are areas that require further attention to optimize the use of patient group data. Improving data dimensions, quality, and continuity of research are essential. To support this, policy support for patient engagement and guidance on standardizing research practices is recommended. This includes:
Encouraging patient groups to undertake data registration and survey research.
Promoting patient involvement in policy-making related to healthcare and health insurance.
Guiding professional institutions and philanthropies to support patient groups in a systematic and effective manner.
Takeda China’s “Teach a Man to Fish” Project
Takeda China has initiated the “Teach a Man to Fish” project to enhance patient groups’ capacity in patient registries and survey research. The project, divided into three phases, aims to establish a structured framework and methodology for improving patient registry and survey research, develop a digital guidance platform, and enable patient groups to formulate policy proposals based on collected data.
This comprehensive approach is expected to lead to significant advancements in patient registration and survey autonomy by 2024, potentially transforming the landscape of rare disease treatment and policy-making in China.