Orphan Drugs must read to all the longs!
Orphan Drugs: 'Rare' Opportunities To Make Money
An estimated 25 million people in the US alone collectively live with some sort of orphan disease. Despite the understanding that specific diseases may afflict relatively small numbers of individuals, this overall growing pool of patients is now becoming an attractive opportunity for the pharmaceutical industry. This reflects the fact that appropriate treatments for unmet medical needs that generate high levels of reimbursement.
“The high cost of therapy and attractive developmental drivers, such as government incentives (including tax credits), smaller and shorter clinical trials, extended exclusivity and high rates of regulatory success, have made top orphan drugs as equally viable as their non-orphan peers,” according to a new report from Thomson Reuters, which found the orphan drug market was worth slightly more than $50 billion worldwide at the end of last year.
Meanwhile, spending on orphan drugs currently makes up approximately 6 percent of total pharmaceutical sales, assumint a total market value of $880 billion. The compound annual growth rate of the orphan drug market between 2001 and 2010 was 25.8 percent, compared with 20.1 percent for a matched control group of non-orphan drugs. Combined with the increasing number of orphan drug approvals, the data suggests compounded annual growth will outpace other drugs.
Higher prices underscore the potential. In 2010, the most expensive drug was Soliris, which costs more than $409,000 annually and is used to treat paroxysymal nocturnal hemoglobinuria, a rare life-threatening blood disease. The med generated $541 million in sales for Alexion Pharmaceuticals. “This is a considerable achievement in terms of revenue, considering only 4,000 to 6,000 people suffer from this disease in the US,” the report states.
Moreover, repositioning such drugs to tre Less
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