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We Need Your Input: BIO Survey on Effective FDA-Sponsor Communication

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BIO is surveying members and non-members on their interactions with the FDA during the various stages of drug development prior to submitting an NDA/BLA. The survey can be accessed here. Why is it important for you to share your feedback? In an increasingly competitive and fiscally-constrained environment, it is more important than ever for all stakeholders to work together to promote effective R&D for innovative medicines. In this BIO BuzzCenter video, Alnylam’s Dr. John Maraganore Read More >

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New PhRMA Report: A Decade of Innovation in Rare Diseases

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Today, the Pharmaceutical Research and Manufacturers of America (PhRMA) released “A Decade of Innovation in Rare Diseases,” which highlights 10 years of progress in understanding rare diseases and drug development resulting in groundbreaking therapies. According to the report, more than 230 new medicines to treat rare or “orphan” diseases were approved by the U.S. Food and Drug Administration (FDA) in the last decade, and there are currently more than 450 orphan drugs in development. The Read More >

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Getting Ready for #RDD2015 (Rare Disease Day 2015)

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Rare Disease Day is just around the corner on February 28th. R&D investment into rare disease research by venture capitalists was a major focus of BIO’s recent Venture Funding of Therapeutic Innovation between 2004-2013, available for download here. One of the more interesting findings in the study is the large increase of funding by venture capitalists for rare disease R&D over the past decade, as this chart illustrates: Over the last 10 years, there has Read More >

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Biotech Could Miss a Multi-Billion Dollar Opportunity in Rare Disease

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Drug development is expensive.  Drug development is risky.  Drug development takes a long time. The key to changing this is leveraging the fact that no one is more motivated to eliminate rare disease than those who are directly affected by it. I know this because, as the founder of Give RARE, I have talked to parents of children with Tay Sachs disease who know their children will die within a few years unless something changes.  Read More >

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Op-Ed: As I Lay Dying

Former staff writer for the LA Times Laurie Becklund shares her experience ‘climbing ladders and falling down chutes’ as a metastatic breast cancer patient in this powerful op-ed. Becklund died in February, but left behind insights on how to improve the care and treatment of life-threatening diseases based on her first-hand experience. Becklund advocates for the creation of a ‘new system of data collection and an open, online, broad-range database about patient histories.’ This information Read More >

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