《2016年在研罕见病药物》.pdfVIP

2 0 MeDicines in DevelopMenT 1 3 R E Rare Diseases P O A Report on orphan Drugs in the pipeline R T presented by america’s biopharmaceutical research companies More Than 450 Medicines in Development for Rare Diseases orphan Drugs in Development* Rare diseases, when taken together, are A major area of this research targets not that rare at all. In fact, according to rare cancers, accounting for more than Application the National Institutes of Health NIH , one-third of all rare disease medicines in Submitted 30 million Americans have one of the development. Other top research areas Phase III nearly 7,000 diseases that are officially include genetic disorders, neurologi- Phase II deemed “rare” because alone they each cal conditions, infectious diseases and Phase I affect fewer than 200,000 people in autoimmune disorders. the United States. Sometimes, only Despite some recent victories, research a few hundred patients are known to 105 into treatments for rare diseases is a have a particular rare disease. daunting quest. This ongoing innovation Simply receiving a diagnosis of a rare and the hundreds of new medicines in disease often becomes a frustrating development now offer hope that physi- 85 quest, since many doctors may have nev- cians will have new treatment options er before heard of or seen the disease. for patients confronting a rare disease. This is, however, a time of great 65 progress and hope. Biopharmaceutical contents research is entering an exciting new era Innovative Orphan Drugs with a growing understanding of the in the Pipeline .........................................2 human genome. Scientific advances have given researchers new tools to Orphan Drug Approvals...........................4 explore rare diseases, which are often Challenges in Clinical Trials ......................6 more complex than common diseases. Prescription Drug User Fee Act 32 PDUFA .................................................7 28 In 2012 alone, 13 orphan drugs were appro