Biotech firm looks to bring more rare disease therapies to China

"With such guidelines, there are opportunities to bring into China rare disease therapies at the same time as those in the United States and Europe. In the past, Chinese patients had to await eight to 10 years after a drug attains US approval before they could access it," Fang said.

He noted that Shire was the first to submit a new drug application for the treatment of Fabry, a lethal rare malady which arises from a deficiency of a particular enzyme. About 10,000 patients in China currently suffer from this rare condition that has no treatment solutions.

"Over the next five years, Shire expects to bring breakthrough therapies also in Gaucher and hereditary angioedema (HAE), rare diseases that affect an estimated 8,000 and 16,000 patients in the country respectively, to address urgent and unmet needs," Fang added.

The company has for the past few years collaborated with patient organizations to accelerate the registration of patients suffering from certain rare diseases, Fang said.

For example, Shire has launched a program aimed at creating a national level hemophilia patient management system and provincial-level diagnosis and treatment pilot to help improve information management and accelerate the upgrade of hemophilia diagnosis and treatment. A total of 14,000 patients have registered at 43 designated centers, according to Fang.

In addition, the company will work closely with the patient community to ensure that people suffering from rare diseases have access to affordable treatment options.

Fang pointed out that Shire already cooperates with charity organizations, including the China Charity Federation and the China Primary Health Care Foundation, to provide the "Patient Assistance Program".

"By the end of 2017, the program has covered over 60 cities and helped more than 1,500 hemophilia patients receive best-in-class treatment," he said.