It was exciting to see grassroots politics in action through your 100+ calls in support of our $10 million request to create a specific review division at the FDA. The respective offices were inundated with calls from very motivated patient community leaders, and the numerous passionate callers were noticed.
While we will have to wait until June for the first draft of the appropriations bills to be released, there is still much work to be done. Members of the House and Senate FDA Appropriations Subcommittees have significant power to move this forward and we are continuing to push the issue with DC staff. We have sent out a letter for patient organizations, academic institutions, biotechnology firms and venture capitalists to endorse the request, and more than 50 organizations have already signed on.
We are also working with patient organizations to arrange constituent meetings in district offices with key Members of Congress. Additionally we will launch our CongressWeb™ function next month on our websites enabling you to automatically e-mail a letter of support to appropriate representatives in Congress.
Thanks in part to your calls, Senator Kohl (D-WI) (the Chair of the Appropriations Subcommittee) has agreed to Senator Brownback’s (R-KS) request to hold a hearing on rare disease regulatory issues in June, giving us a special forum. Senator Brownback is very interested in doing something important for rare disease patients and we should help support that goal in his last year in the Senate. Keep checking back for news on this development.
The new review Division is just a start. Combining new specialized staff with existing staff in a focused Division is an important step toward creating the new policies and guidance documents needed to ignite the development of treatments for rare diseases.
As always, thanks for all your support and please send me your thoughts.
by Emil D. Kakkis

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