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New Report: Nearly 800 new medicines in development to treat rare diseases

By Andrew Powaleny  |    December 16, 2021
Today, PhRMA released a new report detailing orphan drug development in the U.S. and the potential to meet the significant unmet medical need for patients. Rare diseases and conditions...   Read More

3 things to know about the importance of post-approval research and development

By Andrew Powaleny  |    December 6, 2021
Despite tremendous progress and knowledge gained from post-approval R&D, some in Congress still fail to recognize innovation doesn’t stop at U.S. Food and Drug Administration (FDA) approval. After...   Read More

Rare Disease Day 2020: Progress toward cures

By Richard Moscicki, M.D.  |    February 24, 2020
Today kicks off Rare Disease Week, an annual opportunity to celebrate the incredible progress that has been made to treat rare diseases and reflect on the need for continued research and...   Read More

Speaker Pelosi’s drug pricing plan could result in 56 fewer new medicines over 10 years

By Juliet Johnson  |    November 21, 2019
A new analysis from economic consulting firm Vital Transformation shows that Speaker Nancy Pelosi’s drug pricing plan, H.R.3, could result in at least 56 fewer innovative medicines for patients...   Read More

Pelosi’s radical plan would leave patients with sickle cell disease behind

By Tom Wilbur  |    November 14, 2019
Sickle cell disease (SCD) is the most commonly inherited genetic blood disorder, affecting nearly 100,000 children and adults in the United States. Three million Americans carry the sickle cell...   Read More

Pelosi’s radical plan would leave patients with rare pediatric diseases behind

By Tom Wilbur  |    November 7, 2019
There are about 7,000 rare diseases and half of these affect children. According to the National Institutes of Health (NIH), 30 million Americans, or 10 percent of the population, have one of the...   Read More

ICYMI: Fighting for a cure to ALS

By Anne McDonald Pritchett, PhD  |    September 25, 2019
Yesterday at The Atlantic Festival, I had the chance to speak with Amy, a biopharmaceutical researcher focused on amyotrophic lateral sclerosis (ALS). As a young girl, Amy’s father inspired her to...   Read More

New report shows nearly 20 innovative medicines in development for sickle cell disease

By Andrew Powaleny  |    April 2, 2019
Sickle cell disease (SCD) is the most common inherited blood disorder in the United States, afflicting nearly 100,000 Americans. Patients with SCD have sickle-shaped red blood cells, which have...   Read More

Guest post: Hearing from patients on Rare Disease Day

By Guest Contributor  |    February 28, 2019
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: 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

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