PPMD Advocates Ensure Congress Recognizes Importance of Flexibility in FDA Reviews

All your calls, emails, and Hill visits during our February Advocacy Conference (in the pouring rain!) are yielding GROUND BREAKING RESULTS.


Yesterday, the Federal Appropriations Report Language for the FDA was posted and included the language submitted by our Congressional champions at the request of our PPMD community (download the full report). "Report language" is specific direction Congress gives to federal agencies when they receive their budgets.


Last year was the first time ever that we were successful in getting Duchenne-specific language included in the FDA Report Language. 

Your efforts have once again yielded success, the FY17 language reads:

"Duchenne Muscular Dystrophy.—The Committee is encouraged that the FDA has the tools, authorities, and latitude necessary to review and approve safe and effective treatments for rare diseases, such as Duchenne Muscular Dystrophy, as efficiently as possible. In particular, the Committee is aware that the use of intermediate clinical endpoints (ICE) may be an appropriate approach as it has been in similar deadly diseases with dire unmet needs, such as HIV and cancer."

And for those of you who are keeping track, you’ll recall that we submitted Report Language that pertained to a number of other federal agencies and we’ll continue to update you as more language is finalized and published.


So what does this mean? Why does this matter?


In Washington, the federal budget process is a long and detailed process that begins with the submission of the President’s Budget request to Congress on the first Monday in February. This request is often accompanied by new policies and programs that the Administration intends to conduct in the coming year, and the budget amounts serve as an indicator as to whether federal agencies will be getting support for budget increases or steady funding from the White House.


This initiates the Congressional Appropriations process where members of Congress who sit on the very prestigious Appropriations committees make determinations about how the budget funds will be directed in the coming fiscal year. In total there are 12 Appropriations Bills, each pertaining to specific federal agencies — or groupings of federal agencies. 


This is why our community’s advocacy is so critically important. 


The MD-CARE Act established critical infrastructure and programs within key federal agencies (NIH, FDA, CDC, HRSA, ACL, SSA, CMS) but all of the funding for these programs is determined annually through the Appropriations bill. So, each year we craft targeted language that reflects the specific needs of our Duchenne community — and then enlist the support of our Congressional champions to ask that they submit the Report Language and accompanying funding requests to the Appropriations Committee — on our behalf. We begin in February so that our Duchenne community is on the forefront of legislators minds as they make programming decisions (this process goes from February through about the beginning of April). And YOU – our AMAZING community – establish lasting relationships with elected officials and then continue to follow up with staffers to ensure support. With our ultimate goal being that if a federal agency gets a funding bump, a portion of that is directed towards a program within that Agency that benefits our Duchenne community in a targeted way.


Thank you to everyone who came to Washington in February and spent time on the Hill. Thank you to everyone who make calls from home. Thank you to everyone who sent emails in response to our Action Alerts. Thank you to everyone who posted alerts on social media — and encouraged your friends & family to get involved. And a huge thank you to everyone who did all of these things. 


Together, our community is amazingly powerful. Together, we played a part in helping to shape the federal Appropriations Bill so that our federal funding for Duchenne will have the optimal impact for #EverySingleOne in our community.

Read the press release.

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