Cure SMA, a nonprofit organization funding research into new treatments and a possible cure for spinal muscular atrophy (SMA), and providing SMA families with support, reports that it has been carefully monitoring U.S. Senate consideration of bill H.R. 6, the 21st Century Cures Act, on Capitol Hill.
Congress describes the 21st Century Cures act, which has bipartisan support, as an initiative to bring America’s healthcare innovation infrastructure “into the 21st Century.” The act provides new hope for patients and their loved ones, and the necessary resources for researchers to continue their efforts to uncover the next generation of treatments and cures, and to advance healthcare innovation “at lightning speed.” In a statement, the legislators observe that, from the mapping of the human genome to the rise of personalized therapies linked to advances in molecular medicine, this age has witnessed constant breakthroughs that are changing the face of disease treatment, management, and cures.
But while research is moving quickly, the lawmakers say, the federal system of drug and device approval isn’t keeping pace, and is in many ways a relic of another era, with dedicated scientists and leaders at agencies like the National Institutes of Health (NIH) and the Food and Drug Administration (FDA) held back by antiquated laws or rules that haven’t kept pace. They note that although some 10,000 diseases or health disorders known, cures and treatments exist for roughly 500 of them, an unacceptable number. For example, a House document observes that despite recent advancements in science and technology, designing and conducting clinical trials of new drugs and devices in the United States has become increasingly complex, time consuming, and expensive, and that — throughout consultations on the 21st Century Cures initiative — House members heard loud and clear that in order to get life-saving drugs and devices to patients faster, the clinical trial process must modernize.
Consequently, Congress agrees in principle that, to save more lives and keep America at the vanguard of medical innovation, it is necessary to ensure that there is not a major gap between the science of cures and the way these therapies will be regulated, and for the first time, the legislative branch is taking a comprehensive look at the steps it can take to accelerate the pace. Representatives and senators are examining the full arc of the process — ranging from discovery in basic science, to streamlining the drug and device development process, to unleashing the power of digital medicine and social media at the treatment delivery phase.
“We took our time,” the representatives affirm in the statement, “listening to the experts who know best. We’ve been working with patients, patient advocates, researchers, and innovators in the health care system to learn more about how to close the gaps between advances in scientific knowledge about cures and the regulatory policies created to save more lives.”
Cure SMA, however, suggests that lawmakers might be taking too much time, especially in the Senate. It voices concern over Congress’ lack of progress since the House of Representatives passed 21st Century Cures/Innovation legislation in July 2015, by a vote of 344-77, particularly since it had been anticipated that the Senate would quickly consider and vote on the non-partisan bill. Instead, the Senate has neither acted on the House bill nor introduced its own version of the legislation — the “Innovations for Healthier Americans Act.” Instead, the Senate Health, Education, Labor & Pensions (HELP) committee has announced that it will discuss and vote on individual bills concerning biomedical innovation as part of its “step-by-step” approach to the Innovation initiative.
Cure SMA is appealing to supporters to contact their senators, informing them that legislation promoting innovation and research is extremely important, and that the discovery of the next generation of cures and treatments for disease needs to start now.
SMA, which can affect any race and either gender, is caused by a mutation in the survival motor neuron gene 1 (SMN1) and robs people of physical strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk and, eventually, eat or breathe. SMA affects approximately 1 in 10,000 babies, and is the No. 1 genetic cause of death for infants, with about 1 in every 50 Americans a genetic carrier.
In a healthy person, the SMN1 gene produces a protein that is critical to the functioning of nerves that control muscles. Without it, those nerve cells cannot properly function and eventually die, leading to debilitating muscle weakness.
Cure SMA explains that the 21st Century Cures bill contains key provisions that could positively affect SMA patients and their families, including requirements for patient input into risk/benefit analysis, changes to FDA evaluation processes, and $10 billion in mandatory additional funding for the NIH.
According to a House Committee on Energy and Commerce document, under H.R. 6, the NIH will receive at least an additional $1.75 billion per year over five years ($8.75 billion total), and the FDA will receive an additional $550 million. H.R. 6 also reauthorizes the NIH for the first time since 2007.
Cure SMA suggests visiting http://www.senate.gov/senators/contact/ to find contact information for the senators representing your state and urging them to expedite release and consideration of the Innovations for Healthier Americans Act. They also note that Americans can also reach out to their senators through Twitter by either asking the senator’s office for his or her Twitter handle, or finding it using Twitter’s search function.
The advocacy organization explains that the Senate is taking a “step-by-step” approach to moving the Innovation legislation forward compared to the comprehensive legislative package approved by the House. This means various provisions of the 21st Century Cures act will be considered in the Senate as separate bills. They report that Sen. Lamar Alexander, a Tennessee Republican and the HELP committee chairman, plans to discuss and vote on individual biomedical innovation bills in the committee, with votes spread over February, March and April. Bills approved in the committee move to the full Senate for a vote.
The committee’s first action is planned for Feb. 9 and will include The Advancing Research for Neurological Diseases Act of 2015 (S.849), sponsored by Sens. John Isakson (Republican, Georgia) and Chris Murphy (Democrat, Connecticut). This bill addresses the expansion and enhancement of federal research into neurological diseases, and would facilitate the collection and tracking of information on neurological diseases for federal agencies, and the public, including researchers. Cure SMA says that this bill, in particular, could have a positive impact on SMA research. The group is pleased to see the Senate beginning to move it forward, and support the Senate’s commitment and dedication to advancing legislation that would directly impact the SMA community.
For more information about the House version of the 21st Century Cures Act, visit: energycommerce.house.gov/cures