September 12-13, 2020, China Chengdu, the 9th China Rare Disease Summit co-organized by Chinese Organization for Rare Disorders (CORD) and West China Hospital Sichuan University was held. Mr Zhiyu YAN, Co-Founder, Chairman and CEO of Sperogenix Therapeutics, was invited to the opening ceremony and delivered a keynote speech at a seminar titled "Best pathway of orphan drug access to China market".
In his speech, Mr. Yan pointed out that there is still a big gap between China and developed countries and regions in the United States and Europe in terms of the number of research projects in the field of rare diseases, especially the relatively insufficient participation in the early clinical stage. However, China has many comparative advantages in participating in international clinical research on orphan drugs, such as abundant rare disease population, relatively complete clinical trial regulations and policies, numerous clinical research institutions and experienced clinical researchers. Early participation in international multi-center clinical studies, on the one hand, enables Chinese patients participating in the trails to have access to treatment in the earliest time, and on the other hand, the clinical data with Chinese patients participating gives regulators confidence to support orphan drug NDA in China.
He also called for closer and more active cooperation between domestic pharmaceutical companies and stakeholders such as clinical research institutes and patient organizations to promote China's participation in early international multi-center clinical research. And call on relevant government departments to improve laws and regulations to encourage international multi-center clinical trials, and to study and introduce industrial policies to encourage clinical research of rare diseases. With the improvement of China's drug discovery and development level and the improvement of clinical research capacity, China will have the chance of leading the global rare disease clinical research in the future.
Guests and attendees from government agencies, physicians and researchers, pharmaceutical companies, gene companies, patient organizations both domestic and abroad attend this conference off-line and on-line, and contribute their opinions and suggestions on rare disease research, drug development, social security policies and social support system in China.