PPTA Applauds Preserving Access to Orphan Drugs Acts
The Plasma Protein Therapeutics Association (PPTA) commends the Preserving Access to Orphan Drugs Acts, bipartisan legislation that makes an important policy clarification and will remove a barrier to research and development of rare disease therapies.
Representative Jim Gerlach (R-PA), introduced H.R. 2315 with lead cosponsor Rep. Richard Neal (D-MA), and Senator Patrick Toomey (R-PA), introduced an identical bill, S. 1128, with lead cosponsors Sens. Robert Casey (D-PA) and Mike Crapo (R-ID). “Representative Gerlach and Senator Toomey have demonstrated exceptional leadership and commitment to the rare disease community with this legislation,” Julie Birkofer, PPTA Senior Vice President, North America said.
The budget neutral bills will clarify the orphan drug exclusion from the annual pharmaceutical fee by basing the exclusion on whether the therapy is solely approved for marketing by the Food and Drug Administration (FDA) to treat a rare disease or condition. Under current law, a product only is excluded from paying the annual fee if it has claimed the Orphan Drug Act tax credit. This has left many plasma protein therapies unable to qualify for the exclusion and disproportionately affects these lifesaving rare disease therapies. In the U.S., a rare disease or condition is generally defined as one affecting less than 200,000 persons.
“Claiming of the Orphan Drug Act tax credit is not the best way to define all rare disease therapies; many are left out of the exclusion despite treating orphan conditions,” Birkofer said. “The optimal way to meet the needs of patients who rely on PPTs is to clarify the provision in the Affordable Care Act to recognize rare diseases and this bill achieves that,” she added.
Plasma protein therapies include plasma-derived therapies and recombinant blood clotting factors. These unique, non-interchangeable biological products for which no generics or substitutions exist are used to treat bleeding disorders, primary immunodeficiency diseases, alpha-1 antitrypsin deficiency, and certain rare neurological disorders.
PPTA represents the private sector manufacturers of plasma-derived and recombinant analog therapies, collectively known as plasma protein therapies and the collectors of source plasma used for fractionation. PPTA member companies produce approximately 80% of the therapies in the U.S. and 60% of those manufactured in Europe. PPTA works globally to advocate for access to and affordability of therapies; engage in constructive dialogue with regulatory authorities and collaborate with patient organizations. PPTA also administers standards and programs that help ensure the quality and safety of plasma, manufacturing, donors and patients. ###
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