RARE Patient Advocacy Summit – 2012

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. Continue reading

PDUFA – 20 Disease Meeting followup … Firestorm – not!

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, we will always be partial to MLD because of our roots – but it’s really the more general rare disease issues that are important to me in most of these policy discussions. Those that know me well know I have a real passion for the rare disease community.

I wanted to address one issue that has come up a couple of times including a recent blog from NORD.  They have twice described the engagement of rare disease advocates in the process as creating a “firestorm” at the FDA.

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FDA … 20 Diseases Being Chosen for PDUFA-V Meetings

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, the spectrum of severity for those who have the disease, the measures of benefit that matter most to patients, and the adequacy of the existing treatment options for patients.”

There were about 150 people in attendance and just over two dozen of us gave public testimony. The FDA is trying to get a better perspective on the needs, desires, and concerns of the patients by holding these 20 meetings.  Since we are directly impacted by the diseases we are best postured to tell them what’s most important to us.

Get involved … your letter to the FDA (see sample below) is needed by the end of the day November 1st! 

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