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Trends that Matter for Orphan Drug Landscapes

Posted by Matt Breese on Jun 23, 2016

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A collection of engaging infographics from healthcare influencers to capture trends in orphan and rare disease drug landscapes.

Many pharmaceutical manufacturers develop orphan drugs to address rare diseases. However, this isn't cheap... as rare disease clinical trials cost 2400% more per patient than a standard drug trial, according to Total Orphan Drugs.



While orphan drugs provide new treatment options for patients, they still create safety concerns for payers, prescribers and pharmacists due their more complex manufacturing process. However, an ISR report shows that physicians are the most concerned, with 55% believing that biosimilars should require full-fledged clinical trails.



There are more than 100 "innovator biologics" already approved in the United States. The two key driving forces behind this are improved reimbursement from payers and the high cost of reference biologics. Via DataMonitor Healthcare



A review of market access for the relatively new PCSK9 class of treatments, a group of orphan drugs, shows the variation in coverage across channels. Medicare formularies are the most restrictive with nearly half of lives restricted by a PA or step therapy policy and the remaining half are not covered on formularies entirely. Via MMIT



While the cost of development, and trials specifically, is higher for orphan drugs, legislators and the FDA provide a faster track to approval for these critical treatments to enter the market. Their time to market aftere reaching phase 2 is 18 months shorter than products without orphan status, according to Premier Research.


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Topics: Specialty, Industry Trends, Market Access