China: Shortage of doctors and medicine for rare diseases leads to more overseas purchase

The Opinions on Deepening the Reform of the Review and Approval System and Inspiring Innovation of Drugs and Medical Devices issued by the General Office of the Communist Party of China Central Committee and General Office of the State Council recently proposes 36 specific reform measures, including policies to encourage pharmaceutical products and medical devices for rare diseases, such as conditional approval for marketing.

The industry considers that the cooperation among government, enterprises and public benefit community, etc. shall be strengthened for rare disease treatment to be accessible, as the problem of shortage of medicine for rare diseases involving a wide range.

The phenomenon of Chinese conducting overseas purchasing is serious as novel drugs in China are marketed 5-7 years later than in the Europe and America

Wu Zhen, Vice Minister of China Food and Drug Administration (CFDA), pointed out when interpreting the above Opinions on October 9 that there were 433 novel drugs approved for marketing in the U.S. in 2001-2016, wherein, only 133 were marketed in China, accounting for 30.7%; 29 typical novel drugs marketed in China in recent 10 years were 5-7 years later than in the Europe and America; there are other data showing that there were only 10 rare disease drugs marketed in the U.S. in 1973-1983, while there were nearly 500 rare disease drugs marketed from 1983 to 2015 after promulgation of the "Orphan Drug Act" in 1983.

The current treatment situation of the patient population with rare diseases is even worse. "Orphan drugs" are the last hope of many rare disease patients, however, most of them have no access to drugs due to factors like relatively complex cause of disease, weak enterprise R&D, and a long time taken for the marketing of overseas drugs in China.

Huang Rufang, Director of the Chinese Organization for Rare Disorders, pointed out, "It takes a long time for foreign innovative drugs for rare diseases to enter China, while the innovative R&D of drugs and medical devices in China is backward. Rare disease drugs are expensive and lacking in China." According to him, there are only over 110 drugs marketed in China among the over 500 drugs for rare diseases; many Chinese patients have to choose overseas purchasing despite the large risks; and some patients watch helplessly because of the expensive drug prices, even if drugs are there. The Beijing News reporter learned that previously many transnational enterprises introduced relevant pharmaceutical products into China through charitable drug donation, however, they were removed from the market often because of long-term losses.

Clinical trial time shall be shortened and costs shall be reduced to have novel rare disease drugs marketed ASAP

The Opinions introduced this time specially mentions "Applicants for registration of pharmaceutical products and medical devices for rare diseases may file the application for clinical trial waiver. The pharmaceutical products and medical devices for rare diseases approved to be marketed overseas can be conditionally approved to be marketed in China, and enterprises shall formulate the risk control plan and carry out research as required."

In fact, there has been a precedent for opening "green channel" to rare disease drugs before issuance of the Opinions.

As the first rare disease drug approved for relapsed or refractory peripheral T-cell lymphoma (PTCL) in China, the Chidamide developed by Chipscreen received conditional marketing. "If based on the early clinical trial that finds patient benefits and controllable safety, drugs can be approved for marketing with conditions, not only can patients receive early treatment, enterprises can also get returns sooner," said Lu Xianping, President and Chief Science Officer of Chipscreen. At that time, CFDA approved conduct of the registration Phase II clinical study for Chidamide with the purpose of marketing in China by referring to the foreign rare disease clinical development, and later granted priority and special review track to shorten the clinical study time and accelerate marketing of Chidamide.

Encouraging policies are expected to be implemented for the pharmaceutical industry to accelerate product marketing

The conditional "green light" for overseas clinical trial data means that foreign innovative drugs that account for over half of innovative rare disease drugs will speed up to enter China.

"This good news will generate strong impetus in Shire’s product R&D and lay the foundation for us to accelerate introduction of innovative products for rare diseases to China," said Cong Fan, General Manager at Shire China that is committed to rare disease drug R&D. According to him, over 75% of the 40 clinical product projects currently promoted by Shire globally aim at rare disease indications, wherein, more than half have been at Phase III clinical trial or stage of registration for marketing.

Ma Xudong, General Manager of Luoren Medical Devices (Beijing) Co., Ltd., a wholly-owned subsidiary of Singapore Lorem Vascular in China, also expressed that Lorem Group would make the best of the clinical study data of Celution regenerative medical system therapy accumulated overseas, and start the application for treatment of scleroderma with said therapy in China (the product has received the scleroderma orphan drug certification of EU). Ma Xudong hoped that more detailed interpretation on the Opinions and specific guideline for rare disease treatment product registration review and approval could be introduced, to exert the incentive function of favorable policies.

Deng Yong, Associate Professor of Department of Law of Beijing University of Chinese Medicine, suggested that guided by the favorable policies of China, Chinese enterprises should target the policy benefits, make market research, especially in foreign policies and laws for rare disease drug R&D, complete integrated survey and know themselves and competitors, and rationally make decisions in drug R&D and production.

Source: The Beijing News