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Rare Diseases: A New Purpose


How drug repurposing could help drive orphan drug access if pharma can accept the risks By Nader Murad, Senior Analyst Email: nmurad@partners4access.com The US National Institutes of Health describes drug repurposing as “studying drugs that are already approved to treat one disease or condition to see if they are safe and effective for treating other […]

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Webinar 2: U.S. lessons learnt from Kymriah & Luxturna

In the second of three webinars, P4A is focussing on the US environment for gene therapies. Single application cell and gene therapies in the US face health insurance challenges driven by a short-term focus, disconnection between price reporting and outcomes value, and complex distribution models…

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In our mission to understand and influence the debate on Orphan Drugs, P4A will be closely following industry and scientific conferences including attending some of them.

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