Real World Evidence as part of a Managed Access Program

The lack of treatment for COVID-19 , which resulted in a decision by the FDA to allow expanded access and compassionate use  for remdesivir  and Emergency Use Authorization  (EUA) for chloroquine, has brought renewed attention to these methods of getting treatment to patients with life-threatening illnesses when there is no approved drug. The FDA’s response…

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Reducing Deaths from COVID-19: What Can We Learn from Rare Disease?

The COVID-19 outbreak has been exposing many of the existing cracks in the foundations of our healthcare system.  But it is not just the lack of equipment, shortage of healthcare workers and hospital beds, and a missing plan for pandemics that are hampering our collective response.   Some patients are dying not because of the virus…

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Facing the Future of Rare Together

There are so many reasons to feel hopeful about the future of rare diseases.  Since 1983 when the Orphan Drug Act was signed into law, the number of drugs approved for rare disease has risen dramatically. According to a report released by the Center for Drug Evaluation and Research (CDER) in early January of this year,…

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Questions Around Lottery Access Strategy

“It’s a lottery where we’re leaving children’s lives up to chance. I don’t think it’s a game that any of us want to play.” This is a parent’s response to the news that Novartis has started to receive applications for a lottery-based program to give away 100 doses of its $2.1m gene therapy treatment Zolgensma for the debilitating spinal muscular atrophy (SMA) in countries where it is currently unlicensed.

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