Legislation Protects the Treatment of Rare Diseases

Two new bipartisan bills, H.R. 2672 and S. 1423, both titled Preserving Access to Orphan Drugs Act, have been introduced to safeguard the development of drugs and therapies that treat patients with rare diseases by eliminating barriers to innovation. Under the current law, most plasma protein therapies, despite being approved for marketing by the U.S. Food and Drug Administration solely for the treatment of one or more rare diseases or conditions, would not qualify for the orphan drug exclusion from the annual pharmaceutical fee. In the U.S., a rare disease or condition is generally defined as one affecting fewer than 200,000 people. The new bills would modify the law to ensure that manufacturers can exclude the sale of all drugs and therapies that are FDA-indicated solely for the treatment of one or more rare diseases from their annual fee liability.

“The majority of patients who rely on plasma protein therapies are coping with a very rare disease for which no alternative treatment exists,” said Julie Birkofer, Plasma Protein Therapies Association senior vice president, North America. “This legislation preserves access to therapies and drugs for rare disease patients and helps to ensure that research and development into new therapies for orphan diseases continues to be encouraged and remains unencumbered.”