PPTA Recognizes International Rare Disease Day 2014

The Plasma Protein Therapeutics Association (PPTA) joins over 300 organizations in 56 countries in recognizing the 7th annual International Rare Disease Day on February 28.

Sponsored by The National Organization of Rare Disorders (NORD) and Rare Diseases Europe (EURORDIS), this event seeks to raise awareness among the general public and decision-makers about rare diseases, impacting lives all over the globe.

In the United States, a disease is considered rare if it affects less than 200,000 people. In Europe, the definition is one that affects one in 2,000 people. Both organizations estimate that there are as many as 6000-8000 rare diseases, with about 80% being genetic, chronic and often life threatening. Those that are plasma protein disorders include bleeding disorders such as hemophilia, primary immunodeficiencies causing infections and cancer, alpha-1 antitrypsin deficiency causing lung and liver damage and other rare diseases for which plasma protein therapies are the only available treatments.

The unique nature of rare diseases, including those treated with plasma protein therapies, requires constant vigilance to promote awareness and patient access to safe and effective therapies. PPTA and its members work tirelessly to ensure patients have timely and appropriate access to the best possible therapies. In honor of Rare Disease Day 2014, PPTA hosted a Congressional tele-town hall, featuring remarks by Congressman Leonard Lance (NJ-7), Co-Chair of the Congressional Rare Disease Caucus, and Dr. Stephen Groft, Former Director, Office of Rare Disease Research, NIH.  The tele-town hall provided patients, patient advocates, and Congressional decision-makers the opportunity to discuss current and future science-driven policies and collaborative practices that intended to accelerate lifesaving treatments to rare disease patients. In both the U.S. and Europe, PPTA administers standards and certification programs, and is engaged in a broad range of regulatory issues, working to advocate and protect patient access to these therapies.

In the U.S., Rare Disease Day coincides with the 31st anniversary of the Orphan Drug Act and similar legislation has been in place in Europe for over a decade. "This legislation has advanced awareness of rare diseases and outcomes for people with rare disorders. The legislation has also provided incentives for the development of and facilitated the regulatory paths for approval for numerous medicinal products to treat rare diseases," said Mary Gustafson, PPTA's Vice President of Global Regulatory Policy.

Today, these therapies enable patients with plasma protein disorders to lead active, productive lives. As recently as twenty years ago, patients with plasma protein-related conditions faced disability and a shortened life span. The progress has been remarkable, but there is much work to be done. Rare Disease Day 2014 shines a much-needed spotlight on the important needs of these patients throughout the world.

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