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.