The Rare Diseases and Orphan Drugs Roadshow, one of the parallel sessions in 2018 CBIIC, successfully held on the afternoon of September 20, 2018. With the publication of the national list of rare diseases and the introduction of a series of encouraging  policies, rare diseases and orphan drugs is drawing more attention from industry and society. The session was set up to gather all power from government departments, medical institutions, China Pharmaceutical Innovation and Research Development Association (PhIRDA), patients’ organizations and innovative pharmaceutical companies, to deliver as one and act jointly.

First Working Meeting of China Alliance for Rare Diseases

During the 2018 CBIIC, the China Alliance for Rare Diseases (CARD) was officially established, and the first working meeting was held as well. CARD was co-founded by PhIRDA, Chinese Hospital Association and Peking Union Medical College Hospital, up to now, more than 50 medical institutions, 30 research institutions and pharmaceutical enterprises participated in it. CARD aims to meet the urgent clinical needs of patients with rare diseases in China and even around the whole world, promote the development of orphan drugs R&D and related regulations as well. The inaugural meeting of CARD will be held in Beijing in October.

Mr. Li Linkang, Inspector of Medical Administrative Authority of National Health Commission (NHC), in his opening remark he said NHC attached great importance to the rare diseases, a great deal of work was carried out to safeguard the right of patients with rare diseases. NHC will continue to promote the standardization of rare diseases diagnosis and treatment and gradually narrow the regional gaps. He also emphasized that the Medical Administrative Authority would give full support and guidance to CARD in the future.

Mr. Li Linkang, Inspector of Medical Administrative Authority of National Health Commission

Opening Speakers

Speakers: James Xue (Left), Zhang Shuyang (Middle), Yoyo Wang (Right)

Dr. James Xue, Chairman & CEO of CANbridge Life Sciences Ltd. made opening speech on “Opportunities and Challenges in Developing Treatments for Rare Diseases in China”. He stressed that orphan drugs R&D is the “tipping point” to promote the development of rare diseases. Ms. Zhang Shuyang, Vice President of Peking Union Medical College took “The 13^th Five-Year Plan” National Key R&D Program – “Clinical Cohort Key & Special Research Project of Precision Medicine for Rare Diseases” as background in her opening speech, and explained how the national rare diseases registration system serves to facilitate orphan drugs R&D. Ms. Yoyo Wang, Secretary General & Founder of The Illness Challenge Foundation, reviewed the development and the legislative situation of rare diseases and orphan drugs in different countries including the United States, and on behalf of all patients with rare diseases, she expressed strong expectations for new R&D breakthroughs and new initiatives in China.

Roadshow Speakers

First Row: Mr. Gong Mengchun, Executive Director of National Rare Diseases Registry System of China (Left); Mr. Liu Hongyu, President of Prosit Sole Biotechnology (Right);

Second Row: Mr. Jiang Xin, International Project Manager of CoSci Med-Tech (Left); Mr. Zheng Weiyi, Chairman & CEO of Innov Pharma (Right);

Third Row: Mr. Weidong, CEO of EdiGene (Left); Mr. Zhou Zhibo, Executive Director, Strategic Development and Business Operations, WuXi NextCODE (Right)

Mr. Gong Mengchun, Executive Director of National Rare Diseases Registry System of China underlined the importance of national rare diseases registry system and shared the vision and plan in his speech. Several leading domestic pharmaceutical enterprises specializing in orphan drugs also introduced experiences from different perspectives, demonstrating the current situation and prospect of orphan drug R&D in China. As the earliest participant and advocates of orphan drugs R&D, Mr. Liu Hongyu, President of Prosit Sole Biotechnology, Mr. Zheng Weiyi, Chairman & CEO of Innov Pharma and Mr. Jiang Xin, International Project Manager of CoSci Med-Tech shared their companies’ orphan drugs developing strategies and experiences respectively.

EdiGene represents the latest cutting-edge genome-editing technique and emerging field. Mr. Weidong, CEO of EdiGene described how to bring in precise concepts in rare diseases treatment. Mr. Zhou Zhibo, Executive Director of WuXi NextCODE, pay attention on precise medicine of rare diseases from the perspective of genomic data.

Dr. James Xue chaired the panel discussion, and Mr. Song Ruilin, Executive President of PhIRDA, Mr. Li Linkang, Ms. Zhang Shuyang, Ms. Reenie McCarthy, CEO of Stealth BioTherapeutics and Ms. Zhang Yan, Partner of Pingan Ventures participated in the panel. The topic of the panel was “How to Improve the Capability and Motivation of Orphan Drugs among Chinese Pharmaceutical Companies”. The panelists unanimously agreed that the vulnerable rare diseases patient groups and the R&D on orphan drugs needs extensive attention from national authorities and society. On one hand, we need to make definition of rare diseases clear, promote data sharing among medical institutions, establish the rare diseases registry system, enhancing the ability of diagnosing and identifying rare diseases in China; on the other hand, we could promote rare diseases R&D, listing in the market and clinical use by improving the policy environmen.

Panel Discussion

First from the left: James Xue; Second from the left: Song Ruilin; Third from the left: Zhang Shuyang; First from the right: Zhang Yan; Second from the right: Li Linkang; Third from the right: Reenie McCarthy