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Tufts study: Orphan drug approvals up, but high costs pose challenges

Orphan drugs — developed to treat rare diseases — are being approved at a faster rate, but in many cases, US patients will be paying more out-of-pocket costs, according to a Tufts University study.

During the 14-year period that ended in 2013, US regulators approved 86 orphan drugs, up from 65 approvals from 1983 to 2000, said the study from the Tufts Center for the Study of Drug Development

An orphan drug in this country is defined as that which treats a disease affecting fewer than 200,000 people in the United States. Orphan drug status can give a company a potential boost by making it eligible for such incentives as tax credits and a seven-year period of market exclusivity.


According to Joshua Cohen, the assistant professor at Tufts who conducted the study’s analysis, some drugs are prohibitively costly for patients. The most expensive orphan drugs can cost more than $500,000 a year per patient, he noted.

‘‘The encouraging news is that more orphan drugs are in development today than ever before, with more getting marketing approval --- in 2013 alone, nine orphan drugs were approved in the US, the most in a single year,” Cohen said in a statement. “But the high cost of these medicines is leading insurers to reassess their reimbursement policies, which likely will mean more out-of-pocket costs for patients.’’

Rare drugs are important to the local life sciences economy. Last month, for example, Momenta Pharmaceuticals Inc. of Cambridge said its treatment for pancreatic cancer had been granted orphan-drug status by the Food and Drug Administration.

Chris Reidy

Chris Reidy can be reached at reidy@globe.com.