Orphan drugs for rare diseases have helped or saved hundreds of thousands of patients like 2-year-old Luke Whitbeck, but families and insurers are picking up the astronomical cost.
Drugmakers have brought almost 450 orphan drugs to market and collected rich incentives but nearly a third of those products aren’t new or were repurposed multiple times, an investigation shows.
The FDA’s drug-approval team is short more than 700 people and losing skilled staff members to the drug industry.
A voucher awarded to companies that find treatments for rare childhood diseases can be sold to the highest bidder — and then used to speed up approvals for much more common drugs.
A study in Health Affairs concludes that orphan drugs for rare diseases are not having a widespread or deep impact on health care spending.