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Orphan Drug Pricing And Reimbursement: Challenges To Patient Access

 
• By Joshua Cohen

The drug industry and patient advocacy groups have been leery about the imposition of price controls or restrictions on reimbursement for drugs that treat orphan diseases. A review of formulary data from the beginning of the century shows that there has been some pushback from payers about these high-priced drugs, but patient access has not been severely hampered to date.

Orphan Drug_1200

In 1983, the US government passed the Orphan Drug Act (ODA), which fosters development of treatments for rare diseases affecting fewer than 200,000 individuals. Available through the act are special research grants, a 50% tax credit on clinical trial costs, shorter Food and Drug Administration (FDA) approval times and a guaranteed seven years of patent exclusivity. In terms of spurring orphan drug development, the ODA has been an unequivocal success.

In the decade prior to 1983 only 34 orphan drugs were licensed, whereas since 1983 the Food and Drug Administration...

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