Bevy of measures announced to speed up rare disease treatment

Rare disease drugs account for over half the first two batches of drugs eligible for a fast approval track targeting medications already approved overseas and in urgent need for Chinese patients, deputy director of the National Medical Products Administration Chen Shifei said on Oct 24.

"Compilation of the third and latest batch is coming to a close and is expected to be published soon," Chen said during the opening ceremony for the 2020 China Conference on Rare Diseases held in Beijing.

Chen said the administration has placed great emphasis on addressing the demands of people suffering rare diseases, and has been reforming its drug evaluation and approval processes, upgrading registration and management systems and increasing work efficiency.

According to the administration, the first list for foreign novel drugs seeing unmet needs in China was released in November 2018 and consists of 48 drugs. The second list, published in May 2019, has 26 drugs.

In order to guide the development of rare disease treatment, the administration has also established a mechanism that facilitates communication between drug developers and drug evaluation officials, Chen added.

Faster approval of new drugs represents just one facet of improvements seen in healthcare services for rare disease patients in recent years, according to officials attending the event on Saturday.

Ma Xiaowei, minister of the National Health Commission, said it is estimated there are about 20 million rare disease patients in China.

The commission has released the country's first directory on rare diseases, set up a national cooperation network in diagnosis and treatment and promoted the use of a direct-reporting system, he said.

However, he also noted China still faces a shortage of healthcare services and drugs available to rare disease patients, and weak capability in creating innovative drugs.

To solve these problems, more efforts will be devoted to advancing a hierarchical medical system, strengthening drug supplies and deepening reform in the healthcare sector, Ma said.

"Stepping up research into pathogenies of rare diseases and intensifying development of targeted drugs represents a significant opportunity to integrate basic research in medical science with clinical applications," he said.