China Continues to Amend Policies to Improve Care for Rare Disease Patients

Problems in China

Around the world there are 7,000 rare diseases currently identified. Of these, researchers have uncovered effective treatment options for less than 10%. Sadly, even when diseases have therapies, many patients never actually receive them. This is an issue which transcends country borders.

Notably, China has begun to make amendments to policies in order to minimize some of the burdens rare disease patients face.

In China, there are 121 documented rare conditions affecting 3 million inhabitants. These diseases have recently been outlined in the first medical catalog in the country that specifically covers rare diseases. This catalog was launched in May of 2018. Globally, 160 drugs have been developed to treat 74 different rare diseases but only 83 of these drugs have received market approval in China. Unfortunately, one-third of these drugs are not “adequately prescribed” because they have not been given official designations. The lack of such designations makes physicians apprehensive about prescribing these medications.

Another barrier to treatment is cost. In China, only 29 drugs for rare diseases are eligible to receive national reimbursement.

Zou Zhengtao could be considered one of the lucky ones, in terms of access to treatment. He is a 31-year-old man diagnosed with Gaucher disease. Gaucher disease is a lysosomal storage disorder that can cause many symptoms, including bone damage and shortened life expectancy. 60% of people with the condition in China are first misdiagnosed. Once properly diagnosed, many patients can’t afford the treatment which costs over one million yaun each year. The treatment is not included by most insurance companies in the country. Thankfully, Zou was able to receive the treatment, called cerezyme, through the China Charity Foundation. For the rest of his life he will not have to pay for the medication. Not everyone has this outcome.

A New Guidebook

Just this month, China has released a new guidebook concerning rare diseases. It covers all 121 conditions outlined in the aforementioned catalog. The creation of this guidebook was done under the direction of Peking Union Medical College Hospital.

A representative from the National Health Commission has explained that this book should help medical professionals in the diagnosis and treatment of rare conditions.

Report by IQVIA

IQVIA is a health data research company. They have issued a new report analyzing the state of rare diseases in China. Some of the recommendations from this report include-

  • The creation of a new office, specifically designed to manage rare disease related funds
  • An update to the rare disease catalog
  • Increased support for domestic drug development in China
  • A cap on payments to 80,000 yaun annually for patients (12,000 U.S. dollars)
  • A new national action plan specifically for rare disease (similar to those in India, France, Australia, and Brazil) which offers incentives for pharmaceutical companies that could increase the development of treatments for this population.

Other Changes

Other recent changes in China’s rare disease policy include efforts by the National Medical Products Administration to help accelerate market approval for overseas drugs. They believe this could not only help to increase the number of approved drugs but also reduce the cost of them for rare disease patients.

Additionally, the State Council has reduced tax to 3% for 21 different rare disease treatments.

Hopefully, these are just the first of many changes going into China’s rare disease policy, all working to improve the lives of those living with these conditions.

You can read more about these recent changes and China’s approach toward rare diseases here.


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