Shire China devoted to innovation in tackling rare disease treatment

Leading global biopharmaceutical company Shire said it will continue to focus on innovation and collaboration to tackle rare disease issues in China.
Ti Gong

Leading global biopharmaceutical company Shire said it will continue to focus on innovation and collaboration to tackle rare disease issues in China.

Currently a top 10 market and the fastest growing area for Shire, China has significant potential to become the top market, Peter Fang, head of Shire Asia Pacific and acting general manager of Shire China said.

"Our opportunity in China is bigger than any country in the world, with the highest number of rare disease patients — our commitment to China is significant," he said in an interview with Shanghai Daily during the 7th China Rare Disease Summit, organized by the Chinese Organization for Rare Disorders, earlier this month.

"We'll keep listening more to our patients and continue our commitment to the rare disease community in China," he said.

In China, both diagnosis and treatment rates for rare disease patients remain low.

There are currently more than 16 million rare disease patients in the country, and each year more than 200,000 children are born with rare diseases.

Industry studies estimate there are about 136,000 hemophilia patients in China, but less than 10 percent have been diagnosed.

Shire has already launched three innovative products here, offering Chinese patients with treatment options for rare diseases.

"China's releasing of the first rare disease list, which includes 121 rare diseases such as Fabry, Gaucher, hereditary angioedema (HAE), and hemophilia, is a golden opportunity for us to introduce more therapies into China under the favorable policy guidelines," he noted.

Recent regulatory improvements in terms of new drug registration rules is also expected to shorten the launch time for new treatments in China, as the country has put into place an expedited review and approval process for innovative medicines that have been marketed abroad.

Under the new regulations, Shire has already submitted the first new drug application for long-term enzyme replacement therapy in patients with confirmed Fabry disease, which has no treatment options in China at present.

"We're eying the same new drug launch time in China as overseas markets, which has been made possible by the recent guidelines to expedite the introduction of innovative therapies," Fang said.

Over the next five years, Shire China is expected to bring eight innovative and breakthrough therapies (including drugs for fatal diseases like Fabry, Gaucher and hereditary angioedema) to China to address the urgent and unmet needs of rare disease patients.

Shire currently has more than 40 products in more than 100 countries, covering six therapeutic areas. Last year, it invested US$1.8 billion in research and development, 75 percent of which went to research for rare diseases. More than 46 research programs are now underway.

It's also helping academia and healthcare authorities to set up a system and database to identify rare disease patients so that they can be diagnosed and treated.

Shire is sticking to its “patient-centric” commitment, and has been paying close attention to the pressing needs of rare disease patients, introducing more innovative therapies and helping to improve the quality of life for patients with rare diseases in China. It has also worked with over 100 advocacy groups worldwide to foster partnerships and to drive awareness of rare diseases, as well as improve diagnosis and treatment rates for rare disease patients.

During last year's CORD Summit, Shire supported the launch of the HAE China Chapter Hatching Program, providing a platform for patients to have their voices heard, promoting the registration of HAE patients in China, and facilitating the routine disease management of patients.

"To become the leader in the industry means we have to strengthen our partnerships with patient organizations, and we will continue to support them to make sure these patients’ voices get to be heard even more," Fang noted.

Shire China has partnered with local healthcare organizations for hemophilia medical system programs to help improve China's hemophilia case information management and the diagnosis and treatment of the condition in China. To date, a total of 14,000 patients have been registered at 43 designated medical centers.

Shire China has also partnered with charity organizations such as the China Charity Federation and China Primary Health Care Foundation to launch the “Patient Assistance Program”. By the end of 2017, the program covered over 60 cities and helped more than 1,500 patients receive treatment through the co-pay system.

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