China has the highest proportion of rare disease patients, but only 24% of rare disease patients receive the best evidence-based treatment

The Economist Intelligence Unit released the report “Silent Suffering: An Assessment of the Recognition and Management of Rare Diseases in Asia Pacific”, sponsored by CSL Behring, The challenges faced in the diagnosis and management of rare diseases in five Asia-Pacific economies (Australia, China, Japan, South Korea, and Taiwan) are explored. The report emphasizes that improving the status of rare disease management requires prioritizing three core challenges: how to provide patients with correct and timely diagnosis; how to provide financial assistance improvement programs; and how to provide patients with more support for non-medical needs. There are currently 6000-7000 known rare diseases worldwide. Although classified as a “rare disease”, according to statistics, there are about 258 million rare disease patients in the Asia-Pacific region, of which about 50% are children. In mainland China (according to the report, it refers to the People’s Republic of China (excluding Hong Kong Special Administrative Region, Macau Special Administrative Region and Taiwan region), hereinafter referred to as China), medical experts generally rate the incidence of neonatal diseases as 1 in 10,000 and other diseases as 50%. One in 10,000 serves as the dividing line for defining rare diseases. According to estimates by the Chinese Center for Disease Control and Prevention, China has the largest number of rare disease patients in the world, with 16.8 million in 2014. Healthcare professionals interviewed said that due to a lack of relevant clinical guidelines, approved treatments, and funding for testing or treatment, on average only one-third of rare disease patients receive best-in-class care. It demonstrates the heavy disease burden and unmet treatment needs of patients with rare diseases that cannot be ignored. In addition to identifying three core challenges for rare disease management, the report recommends that policymakers prioritize goals that can be achieved in the short term, including better data collection and use, enhanced training for healthcare workers, Popularize existing rare disease knowledge, cooperate with patient organizations to integrate social resources to provide better support, etc. By establishing a more comprehensive and unified treatment plan, there is an opportunity to improve the lives of rare disease patients, ensure the quality of life of rare disease patients, ease the economic burden, and allow them to be more proactive in the diagnosis and treatment decision-making process, thereby changing their future. Rare Diseases in China:According to the estimates of the Chinese Center for Disease Control and Prevention, China has the largest number of rare disease patients in the world (16.8 million in 2014); more than 90% of them are rare The patient’s income is insufficient to cover the living and treatment costs. Clinicians have little chance of encountering rare disease patients in their careers, yet they remain confident in their knowledge of rare diseases. In the survey, Chinese respondents rated the speed and quality of the healthcare system the highest; however, they also indicated that only 24% of rare disease patients received the best evidence-based The ratio is the lowest among the five largest economies. 37% of respondents said that the availability of professional physician resources is “always a major challenge.” A small number of respondents felt that patient organizations should not be involved in the development of treatment plans (18%), and studies to determine clinical efficacy (15%), which were not seen in other economies. The results of the Asia-Pacific report show that:Professional knowledge of healthcare workers is the cornerstone for improving the diagnosis and management of rare diseases. In all markets, healthcare workers feel that they are not adequately equipped to diagnose and manage rare disease patients because they rarely encounter such patients. About 14% of respondents said they had never encountered a rare disease patient in their careers. Therefore, making a correct diagnosis within a reasonable time frame is the first challenge that healthcare workers must overcome. Patients are at the heart of driving change and action in rare disease management, and they need opportunities to be heard and embraced. The report found that most healthcare workers were unaware of the existence of patient groups in their area. The voice of the patient is an important determinant of reform, and understanding patient needs can help plan treatments and care that best meet patient needs and drive action by key decision makers in healthcare and government agencies. Rare disease patients require comprehensive, patient-centered care that is not limited to drug therapy. In addition to poor health, people with rare diseases face many different challenges, including the financial burden of disease-related drug and care costs, as well as social barriers to mobility and employment. As 94% of rare diseases lack an approved treatment option, the care of rare disease patients requires support from non-medical services so that patients’ quality of life can be guaranteed in the absence of treatment options.


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Reprinted from: The Light of Dream Love

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