The organization fighting for sufferers of rare diseases

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In China, charities fighting rare diseases are still in the early stages of development. Most organizations are established by patients and their families and have a limited focus. The organizations often face numerous challenges in terms of obtaining treatment and livelihood protection for patients suffering rare diseases.


However, these seemingly gloomy prospects did not stop Ma Tao from devoting her career to fighting rare diseases. She joined Illness Challenge Foundation (ICF) in 2019, just as the organization was going through a rough patch.


More than 70 percent of rare disease patients in China visit five hospitals before they get a diagnosis, with the average diagnosis taking five years. According to the first Rare Disease Catalog released by the National Health Commission of China in 2018, there are 199 kinds of drugs available globally for the 121 existing rare diseases. 


Because drug approval processes can take a lot of time, of the 103 drugs on the Chinese market, only 73 are covered by medical insurance, and patients still need to bear the majority of treatment costs. Besides, not many domestic pharmaceutical companies are conducting drug research and development.


To Ma Tao, the mission of the ICF is to help patients with rare diseases and respond to their needs, which cannot usually be met by one organization alone. To improve services for these patients, organizations in this field need to consider how to work with more stakeholders, such as the government, hospitals, and pharmaceutical companies.


Ma has noticed that some provinces have started to include costly drugs in their provincial health insurance. From there, she made the judgment that although it was difficult to promote rare disease medical insurance policies from the national level, promoting local medical insurance to cover part of the medical expenses would solve many problems facing patients.


She then led her team to visit provincial health insurance bureaus. Currently, Shanxi, Shandong, Jiangsu, and Zhejiang provinces have established a multi-sourced payment model for rare disease treatment through the efforts of the ICF, Shuidi Gongyi, and local medical associations.


Besides advocating for policy changes, Ma also worked to gather more resources to provide long-term support for patients, facilitating cooperation with hospitals to offer better treatment and screening, with government departments to promote localization of drugs, and with research institutions to build think tanks researching rare diseases.


On the less conventional side, Ma has also been putting efforts into bringing technological innovations to ICF operations to create better outcomes for patients. 


Once, a patient’s mother told Ma that because her child could have seizures at any time, she and her husband had to take turns to safeguard their child during the night. To help solve this problem, Ma worked to build a partnership between the ICF and Plug and Play to launch the Inno4Rare platform, where they tap into the types of help most needed by patients and share them on the platform for different companies to develop new products based on those needs.


Looking to the future, Ma believes that although there are challenges ahead, she and her team will not complain or passively wait but will be active changemakers to create a better future for patients.