How China is leveraging patient groups to develop patient registries and survey research for rare diseases

Thanks, in part, to concerns raised by healthcare professionals and government, the diagnosis and treatment of rare diseases and related medical programmes have seen rapid development in China. To build upon this progress, it is critical to collect firsthand, patient-group-generated data and evidence to further improve health services for patients with a rare disease.

Patient groups can make an important contribution to the development of patient registries and the survey research process, so critical to advancing care for rare disease patients in China. Rare disease patient groups in China have grown significantly in recent years – among the 130 rare disease patient groups, approximately 75% have taken an active part in patient registries or conducting patient surveys.

Systematic patient registries and survey research work could significantly improve patient access to rare disease medications, contribute to the reform of patient-centred and value-based healthcare, enhance patient engagement in the development of a real-world data system and support government efforts to design a health and medical insurance policy framework.

Rare disease patient groups in China have made significant progress in this area. Three-quarters (75.4%) of patient groups have developed patient registries on a large scale. Thirty-seven patient groups have initiated survey research. Survey results have been improved in terms of quality and quantity and are playing a more important role in the promotion of rare disease diagnosis and treatment and the development of related medical programmes.

While these achievements in the healthcare system are notable and highly commendable, there is more work to be done. Key areas to focus on include increasing the dimensions of data collection, further improving data quality and the continuity of research projects. Taking these steps will make utilizing data and evidence from patient groups much more efficient and enable patient groups to make a greater contribution towards optimizing healthcare services and protection systems.

Drawing upon international best practices and experience, we recommend strong policy support to enhance patient engagement and to provide guidance to patient groups on how to conduct their research in a standardized and orderly manner. This will maximize the value of the patient-related information patient groups collect. Specific recommendations for the treatment of rare diseases in China include:

Recognize the value of patient-related information conducted by patient groups, encourage more patient groups to conduct patient registration and survey research, and raise patient groups' awareness of and attention to patient registration and survey research. In addition, relevant departments should encourage professional organizations to provide guidance on standards and methodologies, such as clarifying the patient-group-generated data needs of relevant departments, setting standards for patient registry and survey research, improving analysis and transparency, and promoting the systematization and professionalization of data collection and research work.

The National Health Commission (NHC), the National Healthcare Security Administration (NHSA) and other related policy-making departments must enhance patient group access to feedback channels to enable patient groups to communicate with these departments and provide patient-group-generated data support for policy formulation and optimization.

Relevant guiding departments should encourage academic institutions, think tanks, consulting and research institutions, as well as other professional institutions, to assist rare disease patient groups in the collection of patient-related information and completing survey research reports in a systematic manner. They should also guide philanthropic organizations, especially large foundations, on how to keep abreast of developments within the rare disease field and teach them how to more effectively contribute to the systematic development of the patient service ecosystem.

As the saying goes: “Give a man a fish and you feed him for a day; teach a man to fish and you feed him for a lifetime.” To accelerate patient groups’ capacity building in patient registries and survey research, Takeda China initiated the Teach a Man to Fish project. The project is divided into three phases. Phase 1 focused on developing a structured framework and methodology for improving patient registry and survey research. A detailed analysis of the current autonomous registration and survey environment was conducted, as well as a demonstration of local and international best practices. Phase 2 is ongoing to develop a digital guidance platform that will serve as a tool for patient groups' future reference, so we expect to see a significant increase in patients' registration and survey autonomy by 2024. The patient groups should be able to formulate policy proposals in phase 3, based on the data collected to improve policies and regulations about rare diseases at the national and regional levels.