Clinical evaluation of drugs for rare diseases:challenges and considerations / 国际药学研究杂志

ABSTRACT

Drugs for rare diseases have always been the weakpoints in drug research. The governments of many countries have actively encouraged with the policy support the development of drugs for rare diseases, which has greatly promoted the development of related drugs. To solve the drug shortage for rare diseases and meet the urgent clinical de-mands in China, Chinese government has issued a series of policies to encourage the research of drugs for rare diseases and there are a large number of drugs being applied for clinical trials or new drug approvals for rare diseases now in Chi-na. This has brought new challenges to the clinical evaluation. This paper summarizes the problems encountered in the review of drugs for rare diseases, and discusses these issues referring to the related American technical standards in or-der to provide referential ideas for the research and evaluation of the drugs for rare diseases in China.