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Dear Member Organizations,

As you know, the FDA has recently embarked on a new Patient-Focused Drug Development Initiative, which will include convening 20 disease-specific public meetings to hear directly from patients about their perceptions of risk and benefit when it comes to new and existing treatments. FDA selected bleeding disorders as one of the disease areas of focus, based in part on advocacy from NHF, HFA and COTT, and our meeting is scheduled to occur in 2014 or 2015.

A handful of these meetings have already occurred and the patient communities involved have used different tools to gather input to share with FDA. The National Health Council has created a tool to help groups ensure that they have input from all parts of their communities. The Genetic Alliance recently released an RFP for groups to test whether its new PPRN platform, Platform for Engaging Everybody in Research (PEER), can be used to gather information for the FDA. An example of PEER can be found at: Registries for ALL – https://www.reg4all.org.

After review, HFA and NHF have chosen to not participate in the Genetic Alliance’s project at this time. We simply feel the window to apply for this grant is too short to do justice to a thorough review of the platform and whether it would benefit our community.

We commit to working with all stakeholders and consumers who wish to participate in this initiative. We will also continue to evaluate these and other tools to determine the best way to gather input from the community. Please stay tuned for more information in coming months about this exciting opportunity.

Kimberly Haugstad Signature

Kimberly Haugstad, Executive Director, Hemophilia Federation of America

Val Bias Signature

 

 

Val Bias, Chief Executive Officer, National Hemophilia Foundation

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