美国罕见病药物：独占性、定价和受治患者英文版（25页）.pdf

1、DECEMBER 2018 Orphan Drugs in the United States Exclusivity, Pricing and Treated Populations Introduction In the thirty-five years since the passage of the Orphan Drug Act (ODA) in 1983, the structure of development incentives laid out in the legislation has successfully spurred investment and innov

2、ation in rare disease therapies. Still, approximately 95% of the 7,000 rare diseases remain without any therapeutic options. Recent legislative discussion has focused on whether the ODA development incentives are working as intended or whether they are being manipulated for commercial gain. In this

3、regard, it is particularly important to understand whether the orphan designations granted have delayed generic competition. There has also been significant attention focused on the pricing of orphan drugs both at launch and over time. A persistent issuewith implications for both the pricing and lev

4、els of commercial support needed for these drugsremains that rare disease patients are difficult to diagnose, and as a result, available treatments have limited use by only a small proportion of patients with confirmed disease. This report is a companion analysis to an examination of the orphan drug

5、 market published by the IQVIA Institute in October 2018, “Orphan Drugs in the United States: Growth Trends in Rare Disease Treatments,” and brings a new perspective on the sequence of orphan and non-orphan indications approved and their associated patent and market exclusivities. It also examines o

6、rphan drug pricing relative to patient numbers and how those prices change over time. In a first-of-its-kind comprehensive analysis, the report compares current disease epidemiology to the number of treated patients to demonstrate the challenges in bringing orphan drugs to patients even after theyre