On Tuesday, November 29, more than 160 people from the SMA community – including government and industry partners – gathered for the 6th Annual “Hope on the Hill” Congressional Dinner.

Several Members of Congress attended the dinner, including Reps. Sean Duffy, Bill Foster, Jeb Hensarling, Bill Huizenga and Erik Paulsen. Other honored guests included U.S. Secretary of Agriculture Tom Vilsack.

The evening included remarks from Cure SMA Board Member Greg Zerzan, Cure SMA President Kenneth Hobby and a keynote address by Whole Foods Co-CEO Walter Robb.

We thank the many generous sponsors who made this evening possible, particularly our Discovery Sponsors: Biogen, CTIA Wireless Association and Quest Diagnostics. To see a complete list of sponsors, please vist the 6th Annual Hope on the Hill Congressional Dinner website.

House Passes 21st Century Cures

We were especially proud to host this event on the eve of an important vote on 21st Century Cures. Late Tuesday, the House Energy and Commerce Committee, led by Fred Upton – a past special guest of the “Hope on the Hill” event – announced that the House would vote on a revised version of 21st Century Cures. The bill was passed by the House on Wednesday, 392-26, and the Senate is set to vote on the bill early next week.

The revised version of this bill includes many provisions that will benefit the rare disease community in general and the SMA community in particular. Our thanks to our partners at NORD (National Organization for Rare Diseases) for the following summary of some of the most significant provisions:

  • Extension of the Rare Pediatric Disease Priority Review Voucher (PRV) Program through September 2020, with an additional two years extension for products that recieved an application designation.
  • Streamlining of FDA review of genetically targeted and protein variant therapies for rare diseases.
  • Funding in the form of $4.8 billion over ten years for the NIH to fund the Precision Medicine Initiative, BRAIN Initiative, the Cancer Moonshot, and more.
  • Requirement for companies to publicly post thier expanded access policies on thier website.
  • Further expansion of the Patient-Focused Drug Development Initiative and requirements for the FDA to report on how patient experience data was used in regulartory review.
  • Creation of the National Neurological Conditions Surveillance System to better quantify the incidence and prevalence of neurological diseases, including rare neurological diseases.
  • Expansion of the Humanitarian Use Device program to include devices used by up to 8,000 individuals rather than the current 4,000 individual cap.

Uniting the SMA Community

As more SMA drugs reach late stage clinical trials and progress toward FDA approval, we remain focused on educating, involving and engaging all three groups in our community: the patient and family group, the government and regulatory group and the biotech and pharaceutical companies that are developing and testing SMA drugs. We recognize that we need the whole community working together in order to develop safe and effective treatments for SMA and get them approved as quickly as possible.

The Congressional Dinner is one of the centerpiece events for these important goals. The evening also raised more than $271,000 for SMA research and family support, bringing the six-year total of this event to $850,000!

In addition to the Congressional Dinner, the Cure SMA team and our regulatory and industry partners held several meetings on our current advocacy goals, including advocating for broad labels, insurance coverage and payment, the upcoming Patient-Focused Drug Development Meeting, newborn screening and more. Members of the SMA community are also visiting members of the House and Senate to discuss additional legislative priorities. Please continue checking our news section as we will be sharing more information on these activities in the coming days.

Pictured above: Walter Robb, Christy Vilsack, Secretary Tom Vilsack, Dr. John Day and Kenneth Hobby at the 2016 “Hope on the Hill” Congressional Dinner.