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Policy

US Announces BRAIN initiative with $100m of 2014 funding

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President Obama today announced the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) initiative and an anticipated $100M of US government funding in the next fiscal year. That is part of several hundred million more committed by private partners and foundations to this project to better understand how the brain works. http://www.nih.gov/science/brain/ NIH Director Francis Collins […]

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Fiscal Cliff & Sequestration

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Backgrounder … What is this & why do or should I care? Fiscal cliff – the effect of a series of enacted legislation which, if unchanged, will result in tax increases, spending cuts, and a corresponding reduction in the budget deficit. These laws include tax increases due to the expiration of the Tax Relief, Unemployment Insurance

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RARE Patient Advocacy Summit – 2012

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I was pleased to be the organizer and host for the RARE Project | Global Genes RARE Patient Advocacy Summit on September 29th, 2012. The day-long event with 140 in attendance and over 120 viewing via a live webcast.  Videos of the event are available below for viewing.

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PDUFA – 20 Disease Meeting followup … Firestorm – not!

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Thank you all for your response to my recent request to send letters to the FDA on the Patient-Focused Drug Development initiative last week.  Quite a number of you (dozens) responded on behalf of rare disease in general and many on behalf of MLD or lysosomal disease specifically. As I mentioned in my prior post,

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FDA … 20 Diseases Being Chosen for PDUFA-V Meetings

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Last Thursday, October 25th, I attended the FDA’s Patient-Focused Drug Development public meeting in Washington, DC. This meeting was required by the recently passed FDASIA/PDUFA-V legislation (see Abbreviation Decoder at end of post) and requires the FDA to hold 20 disease-specific meetings over the next 5 years to discuss topics such as: “the impact of the disease on patients,

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