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Rare Disease Day: Unmet medical need inspires biopharmaceutical innovation

By Richard Moscicki, M.D.  |    February 28, 2019
Today is Rare Disease Day, an annual reminder of the incredible community of patients living with a rare disease. In the U.S., rare diseases are classified as conditions that affect fewer than...   Read More

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...   Read More

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...   Read More

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.   Read More

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...   Read More

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...   Read More

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...   Read More

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...   Read More

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 than...   Read More

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 because...   Read More

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