Putting 2018 medicine approvals in context
By Andrew Powaleny | January 23, 2019
Last year, the U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved a record number of novel new medicines with 59 new molecular entities (NMEs). While...
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ICYMI: Orphan drug development brings unique challenges
By Gretta Stone | May 21, 2018
A new report from the Tufts Center for the Study of Drug Development finds that it takes 2.3 years, or 18 percent, longer to develop an orphan drug compared with medicines to treat more common...
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Rare Disease Day: Unmet need inspires innovation
By Andrew Powaleny | February 28, 2018
Today, we celebrate Rare Disease Day to both reflect on the progress made toward conquering rare diseases and recognize the continued unmet medical need for these devastating, complex conditions.
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Guest Post: Room for optimism in ALS research
By Guest Contributor | March 3, 2017
Conversations and healthy debate about issues facing our industry and the health care system are critical to addressing some of today’s challenges and opportunities. The Catalyst welcomes guest...
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Guest Post: Debra of America on Epidermolysis Bullosa
By Guest Contributor | March 3, 2017
Conversations and healthy debate about issues facing our industry and the health care system are critical to addressing some of today’s challenges and opportunities. The Catalyst welcomes guest...
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Guest Post: A Cure for Ellie on Leukodystrophy-LBSL
By Guest Contributor | March 2, 2017
Conversations and healthy debate about issues facing our industry and the health care system are critical to addressing some of today’s challenges and opportunities. The Catalyst welcomes guest...
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Guest Post: Epilepsy Foundation on rare diseases
By Guest Contributor | March 1, 2017
Conversations and healthy debate about issues facing our industry and the health care system are critical to addressing some of today’s challenges and opportunities. The Catalyst welcomes guest...
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Rare Disease Day: Accelerating innovation for patients in need
By Anne McDonald Pritchett, PhD | February 28, 2017
As we celebrate Rare Disease Day, it is important to not only reflect on how far we have come in tackling these devastating and complex conditions, but how much unmet medical remains. While more...
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New PhRMA Report: Medicines in development for patients with rare diseases demonstrate the promise of innovation
By Ieva M. Augstums | May 9, 2016
Today, there are 7,000 known rare diseases, or diseases that affect fewer than 200,000 people in the United States. But those suffering from the impact of these diseases have more hope today...
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Advancing the science for patients with rare diseases
By Allyson Funk | February 29, 2016
When you have a loved one with a rare disease, the importance of persevering to find new, innovative treatments takes on new meaning. This year on Rare Disease Day, I am excited to celebrate the...
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