Tag Archives: orphan drug act

Complexities of Rare Diseases Take the Spotlight at BIO

Orphan Drugs

Global Genes Founder & CEO Nicole Boice opened the BIO 2015 Orphan & Rare Disease Track by addressing a packed session with a simple, powerful statement: “We’re all working to eliminate the challenges of rare diseases, but patients and their advocates aren’t always equipped to fight.” Organizations like Global Genes help families affected by rare disease by connecting them with much needed tools and resources. By better understanding the definition of a “rare” disease, industry Read More >

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Expanding Access to Orphan Drugs

Orphan Drugs

At present many orphan diseases do not have any licensed treatments, and for those conditions where there is a therapy, it is often the only therapy in the class that is available. However, this is not always the case. In the field of blood-plasma proteins, therapies to treat hemophilia, alpha-1 deficiency and primary immune deficiencies have multiple innovator versions in a therapeutic class. Often one product in the class has received an orphan designation and Read More >

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FDASIA: A Step Forward for Patients

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Earlier this year, Congress passed and President Obama signed the FDA Safety and Innovation Act (FDASIA) which included a re-authorization of the Prescription Drug User Fee Act (PDUFA). PDUFA provides essential funding to the FDA by authorizing the agency to charge user fees to companies seeking to have products reviewed. Originally enacted in 1992, it must be reauthorized every 5 years. Sara Radcliffe, executive vice president of health at BIO, moderated a panel of experts and advocates Read More >

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FDA Safety and Innovation Act: A Step Forward for Patients With Rare Diseases

Peter L. Saltonstall

By Peter L. Saltonstall, President and CEO, National Organization for Rare Disorders (NORD) The Food and Drug Administration Safety and Innovation Act (FDASIA) includes the most groundbreaking measures for rare disease patients and their families since the Orphan Drug Act of 1983.  And, just as the rare disease patient community—with NORD as its unifying voice—played a major role in the Orphan Drug Act, NORD and rare disease patients have taken a leadership role in developing Read More >

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2012 Orphan Disease Forum: Addressing Today’s Opportunities and Challenges

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If all of the people with rare disease lived in one country, it would be the world’s third most populous country! Through dedicated companies such as the National Organization for Rare Disorders (NORD) and Centric Health Resources, the pressing issue of orphan diseases and its patients are being addressed. A rare disease is defined by any disease affecting fewer than 200,000 Americans. There are nearly 7,000 such diseases affecting over 350 million people worldwide.  Furthermore, Read More >

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