Yesterday, I had the distinct privilege of representing PPMD and our Duchenne community as I sat before a full room of Senate staffers and congressional aides in the Dirksen Senate office building. Our goal was to urge them to press forward on the Innovation for Healthier Americans bill (also referred to as the ‘Innovation package’) — and to ensure that our Patient Focused Impact Assessment Act (PFIA, S. 1597) is included among the provisions in the bill. It was a compelling afternoon.
As you know, over the past two years Congress has been taking a comprehensive look at the therapeutic development process and ways to incentivize innovation and ensure that great ideas translate into access to lifesaving therapies for patients. This effort started in the House of Representatives and after amazing outreach from communities like ours – this summer – the 21st Century Cures Act (H.R. 6) passed with overwhelming support with a vote of 344-77. Attention then turned to the Senate as Senate leaders began meeting with industry, innovators, and patients communities. In June, PPMD worked with our long-time champions Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) to introduce a bill that we believe should have been included as a provision in the House 21st Century Cures package — and we are working hard to see included as a provision in the Senate Innovation package.
But recently the Senate’s commitment and focus on the Innovation package has stalled. As you know, we have a very small window to get work done here in Washington this fall. With the potential for a government shutdown, leadership changes, and elections looming — the clock is ticking and our community must press hard to ensure that the Senate understands that this bill is a priority.
Because, here’s the deal: The 21st Century Cures Act was landmark. It included innovative new programs that would directly benefit our Duchenne community, and funding to support the agencies and researchers who would implement these programs. But the 21st Century Cures Act goes nowhere until the Senate Innovation package is passed — and then the two bills must be reconciled together. So a stall in the Senate threatens all that our community — and so many others have done. Delays in this legislation are costing lives.
So yesterday, I sat alongside colleagues in the rare disease community who feel as passionately as we do. The Everylife Foundation, Research!America, NORD, the ALS Association, and staffers from two congressional offices. We discussed the bills that our organizations are leading — and we urged those gathered to sign on.
But more – as a rare disease community – we urged the Senate to put pen to paper and issue a draft of the Senate Innovation for Healthier Americans bill.
Please help us ensure that all Senators are aware of the Patient Focused Impact Assessment Act (PFIA) by reaching out today. PFIA will help us to evolve the science of patient input as we work to ensure that the many regulatory tools we as a community have developed are implemented within the regulatory framework.