Takeda ：Patient-centered and Value-based Healthcare– Promote Capacity Building of Data Registration and Research by Rare Disease Patient Organizations
Takeda (China) Holdings Co., Ltd.
Executive summary
Promoted by the concern raised by clinical experts and government departments, diagnosis, treatment, and medication security of rare diseases have made rapid development in China. Due to the characteristics of rare diseases, it is urgent to collect first-hand real-world data (RWD) and evidence to further improve medical services related to rare diseases. At present, rare disease patient organizations in China have made great strides in benefiting society and garnering support from all parties. In recent years, these organizations have taken an active part in registering patient data and conducting thematic research with patient groups.
Orderly and systematic patient registration and research work can directly contribute to making drugs more accessible for rare disease patient groups and to the reform of patient-centered and value-based healthcare. It can also promote patient engagement in related work, help in constructing an RWD system for health outcomes, and assist the government's medical insurance decisions. Overall, the related work of patient organizations in China has made significant progress in recent years. Rare disease patient organizations have generally carried out patient registration in increasingly diversified forms. Many patient organizations increased the quantity and improved the quality of actual output which could contribute to the formation of regulation and guidance within the industry, and play an important role in promoting the development of rare disease diagnosis and treatment and medication security work. Although remarkable progress has been made in the work of patient organizations, much can still be improved upon, such as increasing the dimensions of data collection, improving data quality, and improving the continuity of research projects. These limitations make it harder to apply data and evidence from patient organizations, and therefore the value they hold for optimizing healthcare services and protection systems is not maximized.
From international practices, we have learned that to maximize the value of data collected from patients, there needs to be strong policy support for patient engagement and for guiding patient organizations on how to conduct their research in a standardized and orderly manner. Specific recommendations in this report include:
1. Encourage and guide patient organizations to carry out data registration and research. Recognize the value of patient organizations' data, encourage patient organizations to conduct data registration and research, and raise patient organizations' awareness of and attention to registration and research. Meanwhile, relevant departments need to promote professional organizations to provide specifications and methodological guidance, such as clarifying the data needs of relevant departments, setting standards for data collection, analysis, and transparency, and promoting the systematization and professionalization of the work.
2. Introduce patient advice and enhance patient engagement in the formulation of health care, health insurance, and other related policies. The National Health Commission (NHC), the National Healthcare Security Administration (NHSA), and other related policy-making departments need to open communication channels for patient organizations to provide targeted communication and data to support policy formulation and optimization by central and local governments.
3. Guide participation of professional institutions and charities to form a relevant public welfare ecosystem. Relevant guiding departments should encourage academic institutions, think tanks, consulting and research institutions, and other professional institutions to assist rare disease patient organizations to collect patient data and complete research reports in a systematic manner. They should also guide social charity organizations, especially large foundations, on how to keep abreast of developments within the rare disease field and how to contribute to the systematic development of the industry’s ecosystem.
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