Statement from Jan M. Bult, President & CEO, Plasma Protein Therapeutics Association

Yesterday, a Congressional committee took an unprecedented step that limits access to innovative life-saving medications relied upon by thousands of patients living with rare diseases. The repeal of the Orphan Drug Tax Credit would have devastating effects for those facing chronic and genetic diseases, as it has proven to be essential to the development of hundreds of medicines, including plasma protein therapies.

Plasma protein therapies are life-saving treatments for persons facing particularly rare conditions, including Primary Immunodeficiency Diseases, Chronic Inflammatory Demyelinating Polyneuropathy, bleeding disorders such as Hemophilia, Hereditary Angioedema, and Alpha-1 Antitrypsin Deficiency. These are rare diseases as defined by the Food and Drug Administration (a condition which impacts fewer than 200,000 Americans), and the therapies which treat these diseases are considered “orphan drugs."

As tax reform efforts proceed, we encourage Members of Congress to stand with the thousands of men, women, and children living with these diseases by preserving the Orphan Drug Tax Credit. Since 1983, it has led to the approval of more than 477 orphan drugs that treat rare diseases; without it, there would be 33 percent fewer treatment options for patients facing these conditions.

PPTA urges Congress to reconsider their current position and to, instead, pass legislation making the Orphan Drug Tax Credit permanent.