Rare Diseases & Orphan Drugs Forum (hereinafter referred as the Forum) of the 7th China BioMed Innovation and Investment Conference (hereinafter referred as the Conference) successfully kicked off on 30th March. The Forum of this year added the theme of “pediatric drugs” based on previous forums of past 4 years. Policy makers, experts and enterprises’ representatives from drug R&D and policy research were invited to jointly discuss the security situation and gap in China, providing inspirations for improving the system security for patients with rare diseases and child patients.

Parallel Session

The parallel session includes three phases. Phase I is chaired by Mao Yuejia, senior director of Public Affairs of CANbridge.

Mao Yuejia

Li Linkang, vice chairman, Expert Committee on Diagnosis, Treatment and Support of Rare Diseases of the National Health Commission, addressed a keynote speech.

Li Linkang

Pauline Li, senior vice president of Clinical Development and Operations of CANbridge, addressed a keynote speech entitled “Clinical Development of Rare Disease Drugs Based on China for Globle”.

Pauline Li

Dr. Shawn Xiang, CEO of RareStone Group, made a keynote speech entitled “Patient-Centred Rare Disease Drug Development and Pipeline Construction”.

Shawn Xiang

Phase II was chaired by Eileen Li, chief business officer of RareStone Group.

Eileen Li

Qian Siyuan, reviewer of the Medical Review Department, Centre for Drug Evaluation II, NMPA, addressed a speech entitled “Consideration of Review Evidence of Rare Disease Drugs and Pediatric Drugs”.

Qian Siyuan

Professor Shao Rong, vice executive director of the Research Center of National Drug Policy & Ecosystem, and executive director of the Academy of Pharmaceutical Regulatory Science of China Pharmaceutical University, addressed a keynote speech entitled “Clinical Needs of Medication Accessibility vs. Unmet Needs in Patients with Rare Diseases”.

Shao Rong

Wang Xiaoling, director of Department of Pharmacy, executive deputy directory of Drug Clinical Trial Institute of Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, addressed a keynote speech entitled “Dilemma of Children’s Clinical Medication and Its Solution”.

Wang Xiaoling

Phase III was chaired by Liu Junshuai, member of Specialty Committee on Diagnosis, Treatment and Protection of Rare Diseases, vice-chairman of Medicinal Policy Specialty Committee of PhIRDA.

Liu Junshuai

Shao Rong, Wang Xiaoling, Pauline Li, Shawn Xiang, and Amy Guo, executive director head of Rare Disease of Astrazeneca engaged in the panel.

Panel