Beijing releases draft plan to improve access to rare disease medications

The Beijing Municipal Medical Products Administration released a draft plan on Wednesday aimed at improving the availability of rare disease drugs and is seeking public feedback.

The draft proposes creating a "white list" system, an approved framework for the temporary import, circulation and use of urgently needed rare disease drugs, allowing domestic patients to access foreign-approved drugs that are not yet registered in China.

According to the draft, Beijing will establish three approved lists for medical institutions, urgently needed rare disease drugs and drug import enterprises. Once approved by the National Medical Products Administration (NMPA), drugs on these lists will be stocked by designated import enterprises in bonded warehouses in Beijing Tianzhu Comprehensive Bonded Zone.

Medical institutions will then be able to import and use these drugs as needed within the approved limits. This system ensures traceability and aims to shift from a situation where patients must wait for drugs to one where drugs are readily available for patients.

The draft also notes that urgently needed rare disease drugs on the "white list" will be exempt from import inspection. These drugs can be stored in the bonded warehouses of the Beijing Tianzhu Comprehensive Bonded Zone according to the needs of medical institutions, ensuring their continuous availability for patients. Beijing will also set up a traceability system for the temporary import of these drugs.

Expanded medical network and insurance list to address rare disease challenges

Rare diseases, also known as "orphan diseases," affect a very small percentage of the population, between 0.065 percent and 0.1 percent, according to the World Health Organization. A report released this year on China's rare disease sector indicates that there are now more than 7,000 identified rare diseases worldwide, impacting over 20 million Chinese patients. Each year, China sees an estimated 200,000 new cases.

These diseases have gained more attention recently due to the challenges they present, such as difficult diagnoses, limited treatment options and high medication costs, making management of these conditions particularly complex for patients.

Many rare disease drugs are prohibitively expensive, making access difficult for patients. To address this, the updated national medical insurance drug list now includes 15 previously unlisted medications for 16 rare diseases. Notable conditions that have long lacked effective solutions, such as Gaucher's disease and myasthenia gravis, are now included. Currently, the list covers over 80 rare disease treatment drugs.

In 2019, China established a rare disease medical network with 324 institutions designated by the National Health Commission. Now expanded to 419 institutions, this network reduces the time it takes for patients to receive a definitive diagnosis. To ensure that patients receive necessary medications in a timely manner, the administration has collaborated with social organizations and other departments to procure these essential drugs.