Rare disease seen as innovation frontier

Doctors provide rare disease medical consultation at a free clinic in Guiyang, Guizhou province. TAO LIANG/XINHUA

China's rare disease sector is entering a strategic window for biomedical innovation, as the country's expanding diagnosis network and relatively large unmet medical needs create new momentum for drug development, clinical translation and payment support mechanisms, a top rare disease expert said.

Zhang Shuyang, president of Peking Union Medical College Hospital (PUMCH), told China Daily that rare diseases should not be seen only as a public health and livelihood issue, but also as a promising field for biomedical innovation, with the potential to support new growth drivers in healthcare and advance the Healthy China 2030 initiative.

To seize that opportunity, China needs stronger policy incentives, more efficient clinical translation and a more sustainable payment mechanism to ensure innovative therapies can better help patients, Zhang said.

The challenge is acute. About 95 percent of rare diseases still lack effective therapies, leaving many patients with limited treatment options while pointing to a large unmet need for drug innovation, she said.