Tag Archives: rare diseases

FDASIA: A Step Forward for Patients


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 Originally enacted in 1992, it must be reauthorized every 5 years.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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BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases

BIO Investor Forum

The signing of FDASIA by President Obama in July reinforces the country’s commitment to fostering innovation for rare diseases. With a record 460 medicines in late-stage clinical trials, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline. In anticipation of an even more compelling opportunity, many companies are taking a step beyond and focusing on ultra rare diseases. Last week’s 11th Annual BIO Investor Forum in San Francisco hosted a panel, Raring Read More >

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New Rare Disease Miniseries


Calling all biopharma researchers! Morning show “The Balancing Act” is producing a miniseries called “Roulette Wheel of Rare and Genetic Diseases,” and they are looking for pharmaceutical partners to help educate their audience. The series is working with the Global Genes Project to help educate patients and their families about new research, clinical trials, and government policies that affect those with a rare disease. Patient groups are already excited about this opportunity to spread awareness- take 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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People With Rare Diseases Face a Host of Unique Challenges

Dennis Jackman

By Dennis Jackman, Senior Vice President, Public Affairs, CSL Behring People with rare and serious medical disorders encounter a host of unique challenges–from obtaining a correct diagnosis, to identifying an appropriate course of treatment and gaining affordable access to the care they need.  They and their families, caretakers and health care providers face a constant struggle to identify and access available resources, away from the attention of traditional media that focuses on more common disorders Read More >

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