on may 25, 2019, a symposium on policy, ethics and sociology of clinical study of rare diseases was held at the lecture hall of pumch, which was co-hosted by pumch clinical pharmacology research center, cams rare disease research center and china alliance of rare diseases (card). the event was chaired by card executive director li linkang, and was attended by: zhang wenbao, division deputy chief of nhc bureau of medical administration, song ruilin, president of china pharmaceutical innovation and research development association (phirda), zhang shuyang, office director of nhc committee of experts on rare diseases and vice president of pumch, and liu junshuai, vice president of qingdao social welfare research society. participants also included representatives of rare disease societies, enterprises, members of pumch ethics committee, professors, residents and graduate students.
zhang weibao spoke highly of the efforts of card members, and encouraged card to use the platform for academic exchanges to push forward the prevention and treatment of rare diseases, and promote coordinated innovation in clinical treatment, research and orphan drug development.
dong dong, research assistant professor with the jockey club school of public health and primary care, cuhk, brought a report named “rare disease in a sociological perspective: data, field and reflections”. it showed that rare diseases is more than a medical question, it is also a profound sociological question.
yang zhimin from cfda center for drug evalsuation brought a report named “regulations on the evalsution and registration of rare disease drugs”. an experienced evalsuator, she, based on the peculiarity of rare disease drugs, talked about the technical path requirements of new drug development and policy support, principles of drug evalsuation, and also gave suggestions on clinical trial design.
bai hua, chief evalsuator of the hospital’s ethic committee on clinical trials of drugs and director assistant of clinical pharmacology research center, gave an introduction of “ethics on clinical trials of orphan drugs”. she said the ethic evalsuation now faces many challenges and certain aspects should be given special attention.
the remarks from experts from three different fields triggered hot discussions. wang yiou, secretary general of illness challenge foundation, said the symposium went beyond medical discussions and experts’ attention to related social issues was touching. zhang zhuohua, president of university of south china, said more researchers should join the development work under policy encouragement. zhang aihua, a pediatrician from jiangsu rare disease association, said primary doctors know very little of rare diseases and academic exchanges should be strengthened, and rare disease drugs should be included into the national health system so that they are accessible to patients. sui ruilin spoke highly of the discussions and suggested that policies should be further improved to encourage orphan drug development and manufacture.
the event ended with a story of a patient and his mother, who arrived at the scene. zhang shuyang called for joint efforts so that those with “orphan diseases” are not left as “orphans”.