Setting the record straight on accelerated approval

As we’ve discussed on this blog before, the U.S. Food and Drug Administration’s (FDA) accelerated approval program has served as a critical lifeline for patients with serious and life-threatening...
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Celebrating 40 years of the Orphan Drug Act on Rare Disease Day

For over a decade, Rare Disease Day has been celebrated on February 28 to raise awareness and generate change for the more than 30 million patients in the U.S. living with a rare disease. This year’s...
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Recognizing National Sickle Cell Awareness Month

September is National Sickle Cell Awareness Month and a time for us to underscore the biopharmaceutical industry’s ongoing commitment to improve the lives of patients, families and communities...
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Biopharmaceutical research and development has ushered in a decade of innovation in rare disease

This year on Rare Disease Day, we join families and communities around the world building awareness around rare diseases and encouraging patients to share their stories.
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New Report: Nearly 800 new medicines in development to treat rare diseases

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

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...
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Rare Disease Day 2020: Progress toward cures

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

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

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

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...
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