On September 22nd, the 2019 CBIIC Rare Diseases and Orphan Drugs Roadshow was successfully held. In recent years, the Party Central Committee and the State Council attached great importance to the diagnosis and treatment of rare diseases, introduced a series of policies and guidelines to accelerate the review and approval of orphan drugs and the reimbursemen access as well as setting industrial standards such as the The List of Rare Diseases (First Batch) and the first Guidelines of Rare Disease Diagnosis and Treatment. The roadshow focused on the prevention of rare diseases and accessibility of orphan drugs, attracting the frontiers in orphan drugs research, policy makers, healthcarecare professionals and experts nationwide to share experience and thoughts.

Li Linkang, Executive Director of China Alliance for Rare Diseases (CARD), and Vice Resident of Chinese Hospital Association (CHA) emphasized in his opening remark that non-equivalence between investment and profit for the pharmaceutical enterprises exists in the field of rare disease, where the role of the CARD serves as a link among government departments, pharmaceutical R&D enterprises, medical institutions and patients, making orphan drugs become accessible, affordable, effective and qualified for rare diseases patients.

Moderator: Li Linkang, Executive Director of China Alliance for Rare Disease

Xie Junming, Member of the Expert Committee on Diagnosis, Treatment and Protection of Rare Diseases of the National Health Commission of the PRC and Chairman of Medical Insurance Branch of Zhejiang Medical Association, made a keynote speech titled “Discussion on the Prevention, Treatment and Support Model

of Rare Disease”, he stressed that rare diseases is part of social problem and the key to the orphan drug security policies is government domination and multi-channel financing and payment.

Keynote Speaker: Professor Xie Junming

Song Ruilin, Chairman of PhIRDA, chaired the panel named “Opportunities and Challenges for Access to Medicines for Rare Diseases in China”, panelists Li Linkang, Xie Songmei, Deputy Director of Office of Clinical Evaluation II of the Center for Drug Evaluation of NMPA, He Wenjiong, Vice President of China Association of Social Security (CASS) and Deputy Director of Social Science Department of Zhejiang University, Xie Junming, Member of the Expert Committee on Diagnosis, Treatment and Protection of Rare Diseases of the National Health Commission of the PRC and Chairman of Medical Insurance Branch of Zhejiang Medical Association and James Xue, Chairman & CEO, CANbridge Pharmaceuticals Inc. and Vice Chairman of CARD shared their views regarding the expectation and objectives on increasing the drug accessibility from various perspectives.

Executive Director Li Linkang expressed that, the diagnosis and treatment of rare disease requires joint efforts from multiple parties. Since its establishment in last year, CARD actively carried out a great many works, hoping that through 2-3 years of effort, can better provide policy advice for government. Deputy Director Xie Songmei briefly introduced China’s encouraging policy for the review and approval of orphan drugs, she also pointed out the challenge of China’s orphan drugs review and approval is being lack of official definitions and information about disease criteria, patients and incidence. Only by combining the basic research, policy support and healthcare security can we better improve the environment for rare diseases treatment.

Professor He Wenjiong stated that the healthcare professionals, orphan drugs and investment in rare diseases area are the key points in solving the problems for rare diseases; more attempt should be done to explore a multichannel finance mechanism in the circumstance where the relevant national system is insufficient. Professor Xie Junming took the experience of Zhejiang Province in promoting the rare disease healthcare system as an example, and noted that if we want to build rare disease pilot regions, we need to make a breakthrough in the existing structures and systems. Dr. James Xue as a representative of the industry suggested that the key factor to stimulate the motivation of pharmaceutical enterprises to develop orphan drugs is to explicate policy orientation and expectation. Meanwhile, the rare disease ecosystem should be more completed and enterprises should wholeheartedly develop orphan drugs under the social environment of helping the rare disease patients at all cost.

Chairman Song Ruilin pointed out that the practical problems of China is the thought on securing the rights of rare disease patients and the phenomenon that the relevant policies and regulations are even rarer than the rare disease itself. It requires joint efforts of all parties in society to tackle that problem. We are looking forward to bringing more benefit to rare disease patients and finding a balance between meeting the clinical needs of them and pharmaceutical innovation.

Panel of “Opportunities and Challenges for Access to Medicines for Rare Diseases in China”

From left to right: Chairman Song Ruilin (Moderator), Executive Director Li Linkang, Deputy Director Xie Songmei, Professor He Wenjiong, Professor Xie Junming, Chairman James Xue.

In the 2nd half of the session, speakers from WuXi NextCode, CStone Pharmaceuticals, CANbridge Pharmaceuticals Inc., Cosci Med-tech Co., Ltd., Shanghai Syndegen Biotech and Tongli Biomedical Co., Ltd. brought 6 splendid projects covering areas of big data, AI diagnosis, rare oncology and hematological rare diseases etc.

Big Data and AI Driving Rare Disease Diagnosis

Gu Yan, General Manager of WuXi NextCode, introduced the global standard genomic data platform built by NestCode, which uses the genome to improve global awareness of healthcare and facilitate the next generation of transformative therapies.

Reflection on the Classic Cases of Drug Innovation and Study of Rare Tumor Gene Mutations in the Era of Precise Gene Targeting

Vivian Li, Associate Vice President of CStone Pharmaceuticals, introduced the latest R&D ideas on rare tumor, gene mutations and the latest regulatory strategy development across the world in the era of gene therapy.

Delivering Life-Changing Therapies to Patients

Glenn Hassan, Chief Finance Officer & Chief Business Officer of CANbridge Pharmaceuticals Inc., made a comprehensive introduction of the pipelines of CANbridge, starting with the vision of delivering life-changing therapeutics and diagnostics to address the unmet medical needs of patients around the world.

Patient-Oriented Orphan Drug Pipeline Development

Jiang Xin, Director of Pipeline & Strategy of Cosci Med-tech Co., Ltd., introduced the latest R&D progress of orphan drugs for Phenylketonuria, Gaucher disease, Pulmonary hypertension and Glycogen accumulation etc..

Development of Biopharmaceuticals Focusing on Rare Blood Diseases

Xu Bixiong, CEO of Shanghai Syndegen Biotech, introduced the R&D pipelines layout of Syndegen, covering endocrine system, blood system and immune system.

Orphan Drug TL-010-A Broad Spectrum Antiparasitic Drug for Humans and Animals

Qian Mingxin, CEO of Tongli Biomedical Co., Ltd., introduced the TL-010 for the treatment of the parasitic diseases. The drug has got rare disease and orphan drug designated approvals from US FDA and successfully completed Phase I trial recently.

Highlights of the Roadshow Speakers

Gu Yan (Upper Left), Li Xiaomu (Upper Right), Glenn Hassan (Middle Left), Jiang Xin (Middle Right), Xu Bixiong (Bottom Left), Qian Mingxin (Bottom Right)

The roadshow received high attention from the society and industry, showing that the rare diseases has become widely noticed and become an emerging hot zone for pharmaceutical enterprises and investors. China is embracing a brand new future of rare diseases.

Plenary Meeting

Sidelights of the Roadshow