With Congress decamping from Washington and not expected to return until after the November election, we thought we'd revisit one of the few bright spots of bipartisanship this Congress – the enactment of the latest FDA user fee law (PDUFA), known as the FDA Safety and Innovation Act or FDASIA. (Washington loves a good acronym).

Earlier this year PPMD helped lead an effort supported by a number of other organizations like Genetic Alliance, National Down Syndrome Society, and the Everylife Foundation for Rare Diseases to include within FDASIA key rare disease provisions intended to expedite review of potential treatments.

These provisions:

  • Modify and improve the FDA's Accelerated Review and Fast Track programs to enhance their use and provide clearer guidance on the use of surrogate endpoints; (surrogate endpoint (or marker) is a measure of effect of a certain treatment that may correlate with a  clinical endpoint)

  • Provide FDA with greater ability to engage medical and scientific experts who posses knowledge and expertise the agency may lack internally;

  • A path forward to enhance direct patient engagement with FDA in key medical product review discussions and to better understand and recognize concerns, particularly from rare diseases advocates, regarding the balance between potential benefits and risks; and

  • Establish a new Breakthrough Therapy designation designed to further expedite review of potential therapies.

PPMD is grateful that Congress included all of these rare provisions. But as all of you know, while enacting an idea into law is a major accomplishment, it's far from the end of the road, particularly on complex regulatory topics like this. Over the coming months and even years, FDA will be tasked with drafting guidance and other proposed regulations that will provide the specific details of how these provisions will be implemented.

In comparing the process to constructing a building, the provisions of the law lay out the key elements – how tall the building will be, what it will look like, etc., but the regulations and guidance will stipulate the details – floor plans, critical infrastructure, and the like – that are essential to the project.

PPMD will be working with our champions in Congress and fellow patient advocacy organizations to engage throughout the process. This work will include ensuring FDA meets its deadlines, meeting with senior agency officials, reviewing and commenting on draft documents and, ultimately, ensuring the provisions are implemented as Congress and the advocacy community intended them to be.



Ryan Fischer, Director of Outreach & Advocacy
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