June 14, 2013
/PRNewswire/ -- 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.
(R-PA), introduced H.R. 2315 with lead cosponsor Rep.
(D-MA), and Senator
(R-PA), introduced an identical bill, S. 1128, with lead cosponsors Sens.
(R-ID). "Representative Gerlach and Senator Toomey have demonstrated exceptional leadership and commitment to the rare disease community with this legislation,"
, PPTA Senior Vice President,
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.