On September 28th, the 2020 CBIIC Rare Diseases and Orphan Drugs Roadshow (hereinafter referred to as the Session) was successfully held.

Since the 18th CPC National Congress, under the guidance of “National Innovation-driven Development Strategy” and support of "National Science and Technology Major Project for Major New Drug Research and Development", China's biopharmaceutical innovation has made great progress. Among all the approved drugs in 2019, the independent R&D drugs accounts for over 20%, local innovative enterprises are becoming the backbone of China's biopharmaceutical industry.

As the society pays an increasing attention to rare diseases and orphan drugs, China has successively established the National Rare Disease Diagnosis and Treatment Expert Committee, National Rare Disease Diagnosis Collaboration Network, launched the national rare disease patients registration initiative, released the China's First List of Rare Diseases, published Guidebook for the Diagnosis and Treatment of Rare Diseases (2019 Edition), accelerated the review and approval process for orphan drugs and issued series of favorable policies. The Session focused on the R&D of orphan drugs and pharmaceutical innovation development. It attracted China’s front-line researchers, policy makers, healthcare professionals and other experts to share and exchange brilliant views on site.

Mr. Li Linkang, Executive Director of China Alliance for Rare Diseases (CARD), Deputy Director of National Rare Disease Diagnosis Collaboration Network, pointed out in his keynote speech that in the field of rare diseases, the market investment of Chinese pharmaceutical companies lacks a vision of reward. CARD’s duty is to serve as a link to the government department, drug R&D enterprises, medical institutions and patients, bring all resources together and built a platform for collaborative innovation, through which could the patients get early diagnosis and treatment and easier access to affordable drugs.

Keynote Speaker: Li Linkang, Executive Director of China Alliance for Rare Diseases  (CARD)

Ms. Zhang Jie, Researcher of Department of Chemical Drug Clinical Trial I of Center for Drug Evaluation of NMPA, made a keynote speech entitled “Orphan Drugs Review and Approvals”, elaborately introduced the registration strategy of orphan drug R&D and assessment criteria and basis of clinical trial exemption approval process, indicated that incidence rate, severity, drug accessibility, basis for overseas marketing and China’s medical practice should be taken into consideration on reviewing orphan drugs.

Keynote Speaker: Ms. Zhang Jie, Researcher of Department of Chemical Drug Clinical Trial I, Center for Drug Evaluation, NMPA

Ms. Zhao Kun, Director of Division of Health Policy Evaluation and Technology Assessment of the National Health Development Research Center made a report entitled "Considerations on Health Economic Assessment of Orphan Drugs". She argued that the main difference between the rare diseases’ and common diseases’ health technology assessment was that the research on the family burden brought by rare diseases should be taken into consideration. He also mentioned how to draw lessons from international advanced experience to promote the payment of rare disease diagnosis technology innovation.

Keynote Speaker: Director Zhao Kun

Dr. James Xue, Chairman and CEO of CANbridge Pharmaceuticals moderated the panel of “Rare Disease R&D vs Drug Innovation and Development”. Ms. Yang Liu, Deputy Director of Pharmaceutical Division, Department of Consumer Goods Industry of Ministry of Industry and Information Technology, Mr. Liu Junshuai, Committee Member of National expert consultation committee of Diagnosis and Treatment of Rare Disease, Mr. Xiao Xiao, Professor of ECUST and President of BBM and Yoyo Wang, Founder and Secretary-General of The Illness Challenge Foundation (ICF) shared views about the interactions between orphan drugs R&D and pharmaceutical innovation development respectively.

As the first promoter to implement the rare diseases access policy at the local level, Mr. Liu Junshuai emphasized that rare disease is not only a problem of medical insurance, but also a problem of economy, and more importantly, a problem of sociology. It calls for all parties’ effort to jointly promote the construction of China's rare disease security system and ecology. Ms. Yang Liu, Deputy Director of Pharmaceutical Office, Department of Consumer Goods Industry, Ministry of Industry and Information Technology, stated that 2020 is the planning year of the 14thFive-Year Plan. Taking the orphan drugs’ R&D and production into consideration, China has combined rare diseases with National Science and Technology Major Project at strategic level to jointly promote the enthusiasm of enterprises and the introduction and implementation of relevant policies to address clinical needs of patients with rare diseases. As a representative of the leading gene therapy enterprise, which is prominent for its clinical trials from R&D to industrialization, he shared the main differences in rare disease related policies between China and the United States. Mr. Xiao Xiao, Professor of ECUST and President of BBM, stated that with the continuous increase of capital market investment in rare diseases, gene therapy and cell therapy in recent years, the R&D and production capacity of orphan drugs increased rapidly and gradually integrated with international standards. From patients’ perspective, Yoyo Wang, Founder and Secretary-General of the Illness Challenge Foundation (ICF) elaborated the positive role of patients and patient organizations played in the whole chain of orphan drug R&D and innovation.

Finally, Dr. James Xue pointed out that the management of rare diseases prevention and control in China started relatively late, especially in scientific research, diagnosis, treatment, rights protection etc. It requires joint efforts of all sectors to explore the benefit of orphan drug R&D on China’s pharmaceutical innovation as well as industrial capacity, facilitating upgrading of industry capacity and promoting China as a strong pharmaceutical innovation country.

Panel: Rare Disease R&D vs Drug Innovation and Development

From Left: Dr. James Xue (Host), Deputy Director Yang Liu, Mr. Liu Junshuai, Secretary-general Yoyo Wang, President Xiao Xiao

In the Phase II of the session, 7 brilliant roadshow projects were presented respectively by JS InnoPharm, R & B Biotech, China Health Group, Exegenesis Bio Hangzhou, Tianjin Happy Life Tech, Porton Biologics and CoSci Med-Tech. Projects covers areas include big data and real-world study, gene therapy, Class II new drug R&D etc.

Clinical Development of Aurora Kinase A Inhibitor VIC-1911 for Myelofibrosis

Mr. Zhang Jintao, CEO, JS InnoPharm introduced VIC-1911, the novel and highly selective Aurora kinase A inhibitor with first in class potential, and expected to be the a “first-in-class”.

Genetic medicine: rAAV gene therapy for Hemophilia B

The project introduced by Mr. Dong Biao, CEO of R&B Biotech used an independently invented scalable production system to produced a highly active Factor IX which would improve the effectiveness largely and developed a rAAV genetic drug for Hemophilia B, lower the price for more than 10 times and the drug is on CMC stage and IND will be applied in 2021.

Application of Big Data and Real World Clinical Studies to Screen New Drugs and Best Medical

Ms. Song Xuemei, Executive Director of China Health Group introduced that China Health Group is a R&D-type digital medical service group collaborated with Wanquan pharmaceutical, a research and development driven digital ecological Pharmaceutical Group, to create new drugs and clinical solutions for rare diseases and brain diseases.

Innovative Gene Therapy Platform for Rare Diseases

Mr. Wu Zhenhua, CEO of Exegenesis Bio Hangzhou Jiayin introduced their adeno-associated virus (AAV) and lentivirusbased gene therapy for ocular, hematological and CNS diseases. Exegenesis Bio is one of the few companies in China that has the manufacture process and capability to scale up GMP manufacture to 2000L and has already established a GMP facility, compliant to Chinese, American and European cGMP standards.

Real World Study Facilitating Innovative Drug Development Treating Rare Diseases

Ms. Dai Luyan, VP of Tianjin Happy Life Tech, introduced that Happy Life Tech devote itself to create intelligence technology, conduct all-round and efficient real-world study and analysis, enable the development of innovative orphan drugs and the completion of ecology of evidence chain, so as to promote the standardization of rare diseases diagnosis and treatment.

Gene and Cell Therapy CDMO in Rare Disease Drug Discovery

Mr. Kong Lingjie, CTO of Porton Biologics introduced that Porton Biologics is a CDMO company focusing its attention on developing and producing gene and cell therapy products. Porton’s services include process development, GMP manufacturing, analytical and quality control of plasmid DNA, viral vectors and cell therapy products.

CoSci Class II Orphan Drug R&D Progress

Mr. Jiang Xin, Director of Pipeline & Strategy of CoSci Med-Tech, introduced that CoSci has over 10 new orphan drugs in its R&D pipeline. KX-258 is the first medicine for Glycogen Storage Disease type I, and CoSci is going to file it to NMPA by Class 2.2/2.4 route for Clinical Investigation.

Roadshow Speakers

Mr. Zhang Jintao, CEO of JS InnoPharm (Upper Left)

Mr. Dong Biao, CEO of R&B Biotech (Upper Middle)

Ms. Song Xuemei, Executive Director of China Health Group (Upper Right)

Mr. Wu Zhenhua, CEO of Exegenesis Bio Hangzhou Jiayin (Middle Left)

Ms. Dai Luyan, VP of Tianjin Happy Life Tech (Middle Right)

Mr. Kong Lingjie, CTO of Porton Biologics (Lower Left)

Mr. Jiang Xin, Director of Pipeline & Strategy, CoSci Med-Tech (Lower Right)

The Roadshow Session was extremely popular with multiple highlights, reflecting an increasing attention of the whole society paid to rare diseases. As a hot topic in pharmaceutical industry and investment community, R&D of rare diseases and production of orphan drugs will greatly help to create an “innovation-friendly” environment, and immensely promote the development of biopharmaceutical industry.

Plenary Meeting