On Wednesday, July 9th, the House Energy and Commerce Committee continued its series of hearings on the 21st Century Cures initiative, which was launched earlier this year by Reps. Fred Upton (R-MI) and Diana DeGette (D-CO). The topic of Wednesday’s hearing was steps that the federal government can take to modernize clinical trials so that they become more cost-efficient and produce results that are quicker to reach the market.
In their remarks, members of the committee and witnesses to the hearing agreed that the size, duration, costs and failure rates of clinical trials are higher than ever and that this curve must be bent in the other direction. In her opening statement, Rep. Lois Capps (D-CA) offered the National Pediatric Research Network Act (NPRNA) as an example of legislation that is taking a step in the right direction. Several of the other speakers proposed standardized clinical trials as a way to reduce redundancy and maximize the data available from studies across the globe. Another popular suggestion was the creation of a national patient registry database, which would have the potential to greatly reduce the length of time needed by researchers to find volunteers for clinical trials. One witness to the hearing suggested that a database of this kind could reduce the process of finding volunteers from several years to just weeks in some cases.
The focus of this hearing was critically important to the mission of FightSMA as we continue our efforts to find a treatment. As FightSMA continues its work with the National Institutes of Health (NIH) to implement the NPRNA, we thank the Energy and Commerce committee for its ongoing focus on the importance of modernizing and improving our nation’s health delivery systems. FightSMA will continue to monitor future hearings on this important initiative, and we again thank Chairman Upton, Ranking Member Waxman, and Reps. McMorris Rodgers and Capps for their leadership in enacting the National Pediatric Research Network Act.
FightSMA is excited for the 9th Annual Ride for Corinna’s Angels event! We are so appreciative of the wonderful Calise Family for throwing this event for the 9th year! The event starts at 9:30AM on Sunday, July 13th in Cranston, RI. It is always an amazing event with lots of fun, food, and great music! If you are in area be sure to join the festivities! The funds raised benefit FightSMA and our crucial mission!
FightSMA-funded gene therapy program, led by Dr. Brian Kaspar at Nationwide Children’s Hospital, has now moved to clinical trial and formally begun enrollment. This trial will utilize chariSMA(TM), the gene therapy product licensed to AveXis for development and commercialization. “I’m thrilled to see (gene therapy product) chariSMA(TM) translate from the bench to the bedside and am hopeful that we will be able to make a difference for SMA patients,” says Dr. Kaspar.
On Tuesday, May 6, the House Energy and Commerce Committee hosted a roundtable discussion to introduce the 21st Century Cures initiative, which is aimed at exploring ways to modernize medical research and expedite critically important breakthroughs. The committee has pledged to examine how to improve the entire medical research process so that elusive treatments for the most devastating diseases can be discovered and quickly delivered. In announcing the formation of the initiative last week, Chairman Fred Upton (R-MI) and Rep. Diana DeGette (D-CO) released a video highlighting the merits of the program and cited the passage of the National Pediatric Research Network Act (NPRNA) as a model for Congress to use as it considers ways to support medical research in the future. The video can be found at http://bit.ly/about21stCC.
Each member who spoke at the roundtable, including Upton and Majority Leader Eric Cantor (R-VA), stressed the importance of shortening the time it takes to develop cures and studying ways that Congress can reduce undue regulatory burden on those conducting clinical trials. Witnesses to the hearing, which included representatives from the National Institutes of Health (NIH), the Food and Drug Administration (FDA), major universities and private research institutions, highlighted the need for a collaborative, networked approach to research in order to efficiently utilize limited funding.
FightSMA applauds the Energy and Commerce committee for creating the 21st Century Cures program and looks forward to continuing its relationship with Chairman Upton to ensure full implementation of the NPRNA, which will serve as a model for cost-effective and life-saving clinical trials.
On Thursday, May 8, 2014, the National Organization for Rare Disorders (NORD) will honor Congressman Fred Upton (R-MI) and Senator Sherrod Brown (D-OH) for their tireless efforts to pass the National Pediatric Research Network Act (NPRNA), legislation championed by FightSMA to authorize NIH to establish a network of pediatric research consortia including multisite clinical trials for rare pediatric diseases. NORD’s National Health Leadership Award will be presented to Upton and Brown during NORD’s annual Portraits of Courage celebration, at which several additional individuals, organizations and companies will also be honored for their significant contributions to the rare disease research community.
Also recognized for their contributions to the fight for an SMA cure will be Sarah and Eric Kennedy of Mattawan, MI, whose two beautiful daughters, Brooke and Brielle, live with SMA. The Kennedys will have the honor of presenting the Leadership Award to Congressman Upton, their hometown representative. The relationship established between the Kennedys and Upton during the years-long effort to pass the NPRNA serves as a case study in how constituents can effectively work with their elected officials to get meaningful legislation enacted.
FightSMA is extremely grateful for NORD’s active endorsement of the NPRNA, which gave the legislation a critical boost. Enacted in November 2013 after years of consideration on Capitol Hill, the NPRNA is a tremendous opportunity for the advancement of SMA and rare disease research. The bill simply wouldn’t have become law without the commitment by Upton and Brown to get the legislation to the President’s desk. FightSMA is so thankful for the opportunity to work with all of the NPRNA’s champions, including Sen. Roger Wicker (R-MS) and Reps. Lois Capps (D-CA) and Cathy McMorris Rodgers (R-WA).
FightSMA looks forward to working with NORD and all of our rare disease and pediatric research partners in the months ahead as we work with NIH to implement the National Pediatric Research Network Act.