Legislation introduced on Jan. 24 by U.S. Rep. Buddy Carter (R-GA) would modernize federal law that lets drug manufacturers block new treatment options for rare diseases from coming to the commercial market.
“Updating this bill is a common-sense measure to improve patients’ health and limit bad actors from taking advantage of rare diseases to line their wallets,” Rep. Carter said.
If enacted, the Fairness in Orphan Drug Exclusivity Act, H.R. 456, would amend the Federal Food, Drug, and Cosmetic Act with respect to limitations on exclusive approval or licensure of orphan drugs, according to the text of the bill.
The Food and Drug Administration (FDA) defines an orphan drug in the 1984 amendments of the U.S. Orphan Drug Act as a drug intended to treat a condition affecting fewer than 200,000 persons in the United States, or which will not be profitable within seven years following approval by the FDA.
According to Rep. Carter’s staff, H.R. 456 would require that all drugs having seven years of market exclusivity show that they have no reasonable expectation of recovering research and development costs through sales in the U.S., thereby closing a loophole that allows manufacturers to obtain seven-year market exclusivity by “piggybacking” on the orphan drug status of an older drug.
“Patients shouldn’t suffer because of a technicality in a 40-year-old law,” said Rep. Carter. “This legislation reaffirms the original intention of the Orphan Drug Act of 1983, to encourage research and development, without the unintended consequence of new drugs being blocked from the market.”
H.R. 456 has been referred for consideration to the U.S. House Energy and Commerce Committee.