A cross-national comparative study of orphan drug policies in the United States, the European Union, and Japan: towards a made-in-China orphan drug policy.
Rare diseases can severely impact patient life quality as well as impose a serious burden on society. But research and development for drugs to treat these disorders has stagnated because of lack of demand, insufficient knowledge of pathophysiological mechanisms, and too few patients for clinical trials. In several countries--the United States, the EU, and Japan--specific legislation has been enacted to encourage pharmaceutical companies to expedite the development of drugs for rare diseases, orphan drugs, and to assure access to them. We analyze the strengths and weaknesses of the incentives in these laws and describe the status of rare diseases in China. We offer some recommendations for orphan drug legislation in China, based on local research on rare diseases.
Bio-X Center, Key Laboratory for the Genetics of Developmental and Neuropsychiatric Disorders (Ministry of Education), Shanghai Jiao Tong University, Shanghai 200030, P.R. China.
Bao-cheng
BC
Liu
Lin
L
He
Guang
G
He
Yun
Y
He
D002681:China; D017277:Drug Approval / Q000191:economics / Q000331:legislation & jurisprudence*; D004345:Drug Industry / Q000191:economics / Q000331:legislation & jurisprudence*; D005062:European Union; D014943:Global Health; D006291:Health Policy; D006297:Health Services Accessibility; D006301:Health Services Needs and Demand; D006801:Humans; D038622:Internationality; D007564:Japan; D009965:Orphan Drug Production / Q000191:economics / Q000331:legislation & jurisprudence*; D055553:Prescription Drugs / Q000191:economics; D011634:Public Health / Q000331:legislation & jurisprudence; D035583:Rare Diseases / Q000188:drug therapy* / Q000191:economics; D014481:United States
D055553:Prescription Drugs