List of orphan drugs available at lower prices gets longer

BEIJING -- Another 15 rare disease drugs, also known as orphan drugs, have been incorporated into the recently updated catalog of medicines covered by China's national medical insurance, resulting in more urgently needed drugs being accessible at lower prices.

The new entries into the catalog cover 16 types of rare disease, which means a total of 80 orphan drugs now feature on the medical insurance list, including drugs needed for the treatment of myasthenia gravis and atypical hemolytic uraemic syndrome.

"The inclusion of drugs used in targeted therapy for diseases of social concern, such as myasthenia gravis, is of particular importance, as it means that more affordable target therapy drugs will be accessible," said Wu Liwen, a senior neurology physician at a children's hospital in central China's Hunan Province.

Thursday marks World Rare Disease Day.

Currently, more than 20 million people in China suffer from rare diseases. Such diseases are often very challenging, both in terms of accurate diagnosis and availability of suitable medicine.

In March 2021, a paroxysmal nocturnal hemoglobinuria sufferer was confronted with a lack of medicine. Thanks to the joint efforts of the China Alliance for Rare Diseases (CARD), the National Medical Products Administration and pharmaceutical companies, the patient gained access to new medicine, introduced from Switzerland, within three months.

Chinese health authorities have been working with social organizations, pharmaceutical companies and related research institutes to make rare disease drugs more accessible and affordable for the Chinese public.

This year, for instance, the price of the drug used for treating atypical hemolytic uremic syndrome, a rare disease affecting children, has been cut from over 20,000 yuan (about 2,815 U.S. dollars) a dose to around 1,000 yuan per dose.

This encouraging change has reignited the hopes of many patients and their families. In the foreseeable future, China's national medical insurance scheme will benefit more patients as the country has been attaching great significance to efforts aimed at the prevention and treatment of rare diseases.

A series of measures have been taken in a coordinated manner in recent years, with Chinese health authorities green-lighting and prioritizing the approval of rare disease drugs for use by medical institutions.

With the support of the government, some pharmaceutical companies have set up care centers for rare disease patients, offering one-stop professional drug services.

China has also established a multidisciplinary diagnosis platform at the national level in a bid to shorten the time needed for a patient to receive a final diagnosis. In addition, Traditional Chinese Medicine was encouraged to contribute to these efforts.

In September 2023, the National Health Commission released its second catalog of rare diseases, containing 86 such diseases falling into 17 medical specialties. So far, the catalogs have recorded 207 of the 7,000 rare diseases identified across the world.

"Consensus concerning the need to help rare disease patients secure access to drugs has turned into actions," said Li Linkang, executive director of the CARD.

China has been committed to safeguarding people's health. The initiative to increase the number of medicines covered by national medical insurance has benefited not only rare disease patients but the public in general.

The latest catalog of medicines qualifying for medical insurance reimbursement, which was released in December 2023, features 126 new medicines, with the total number reaching 3,088.

Taking into account both medical reimbursement and price-cutting, this move is predicted to save patients more than 40 billion yuan over the next two years.

"It has created new options for both doctors and patients, and will greatly relieve the financial burden faced by patients," said Li Haihai from a research institute based in Hunan Province.