PPTA applauds introduction of 'Preserving Access to Orphan Drugs Act'

The Plasma Protein Therapeutics Association (PPTA) applauds the introduction of bipartisan legislation, the Preserving Access to Orphan Drugs Act (H.R. 2672) and (S. 1423), which will safeguard the development of drugs and therapies that treat patients with rare diseases by eliminating barriers to innovation.

"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, senior vice president, North America, PPTA. "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."

"The Association recognizes the commitment to preserving orphan drug development demonstrated by Representative Jim Gerlach (R-Pa), who introduced H.R. 2672 with lead cosponsor Representative Jason Altmire (D-PA) and Senator Patrick Toomey (R-PA), who introduced the companion bill, S. 1423, with lead cosponsors Sens. Robert Casey (D-PA) and Ron Wyden (D-OR)," Birkofer said.

Under the current law, most plasma protein therapies, despite being approved for marketing by the U.S. Food and Drug Administration (FDA) solely for the treatment of one or more rare disease or condition, 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 less than 200,000 persons. H.R. 2672 and S. 1423 modify the law to ensure that manufacturers can exclude the sales of all drugs and therapies that are FDA-indicated solely for the treatment of one or more rare disease from their annual fee liability. The legislation is bipartisan and budget neutral.

SOURCE Plasma Protein Therapeutics Association (PPTA)