PPTA Praises Legislation that Protects the Treatment of Rare Diseases

ANNAPOLIS, MD - 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.

Plasma protein therapies, which include plasma-derived therapies and recombinant blood clotting factors (a biotechnology product), are biological products that are not interchangeable, and no generics or substitutions exist. These unique therapies are used every day to treat people with bleeding disorders including hemophilia, which causes painful internal bleeding and debilitating joint damage; primary immunodeficiency diseases, which render the body defenseless from even the most common infections, often leading to pneumonia and other serious illnesses; and alpha-1 antitrypsin deficiency, also known as genetic chronic obstructive pulmonary disease (COPD), which severely damages the liver and lungs. Additionally, a plasma protein therapy, albumin, is used in critical care settings, when treating severe trauma, burns and during major surgery.

For more information, contact Kym Kilbourne at 443-458-4682.

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