The orphan drug market was worth more than $50 billion in 2011 and turns out blockbusters at the same rate as the broader industry, according to a recent Thomson Reuters analysis. This is mostly due to the high-prices of therapies developed by the industry for rare disease patients.
Even though the segment has just started to provide a great source of revenue for pharma, major challenges are already being faced to sustain it, as it will all depend on how the insurance companies will be able to continue to reimburse orphan products, especially as new therapies reach the market exponentially and access to those by patients across the world is leveraged.
Companies like NPS Pharmaceuticals are already coming up with alternative solutions to support patients with the therapies costs. NPS is better known for Gattex, its orphan drug for Short Bowel Syndrome and the company will cover out-of-pocket expenses for patients who are privately insured if the costs exceed $10 a month, and nonprofit foundations that receive financing from NPS will pay the expenses for people insured by Medicaid and Medicare. NPS will cover the full cost of Gattex for those who do not have any insurance.
Reimbursement access as well as the sustainability of insured companies' finances are two of the main topics addressed at the World Orphan Drug Congress USA 2013.
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