Earlier this month, Congress approved, and President Obama signed, the Cures Act, formally known as the 21st Century Cures Act. Along with scores of other voluntary health and research organizations, the Parkinson’s Disease Foundation (PDF) applauds this move as one that will confront diseases head-on and accelerate research seeking cures.
In this decisive, bipartisan move — too rare in these politically-divided days — the federal government has signaled its confidence that science can help us deliver on the elusive dream of drawing the curtain on diseases including Parkinson’s.
The Cures Act is a comprehensive bill designed to get better therapies more quickly to the people who need them. While it is difficult to summarize (it goes on for nearly a thousand pages!) for PDF there are three provisions in particular that resonate with our vision for ending Parkinson’s.
- One of these is the Act’s promise to provide $1.5 billion in support for continuation of the public-private partnership known as the BRAIN Initiative that was introduced by President Obama in 2013. I was proud to represent PDF (and the American Brain Coalition, which I was serving as Chair at the time) at the White House when the President launched this initiative before an audience of 250 National Institutes of Health (NIH) leaders, neuroscientists, professional organizations and lay advocates. This initiative will create a greater understanding of how the entire brain works as a complete unit. This could help us understand for example, how deep brain stimulation for PD works and provide clarity as to how the brain changes during PD and how to stop it.
- A second provision much prized by PDF is the attention the Cures Act pays to scientists in the early stages of their careers. A reality of scientific careers these days is that because of tight competition, even the most brilliant scientists are unable to win their first federal research grant until they are in their late 30s or early 40s. PDF has seen good scientists leave Parkinson’s as they try to provide for themselves and their families in an uncertain funding environment. PDF has tried to fill this gap — to ensure creative young scientists bring their talents to bear on Parkinson’s — by creating the Stanley Fahn Junior Faculty Awards. Similarly, the Cures Acts includes measures to increase opportunities for early career scientists, including the establishment of an office at the NIH to focus on that goal. A new infusion of funding could give young scientists a shot at winning their first grant years earlier and help us find cures more quickly.
- And a third provision, close to our hearts at PDF, is strengthening the patient’s role in medical decision-making and the use of data that captures their experience. Patients will have an enhanced role in the assessment of new medicines and devices. In addition, for drugs approved by the US Food and Drug Administration (FDA) moving forward, the FDA must include a statement on how “patient experience data” was used to evaluate the drug. This type of information is critical to understanding how new therapies impact people’s lives in the real world. PDF itself has been investing $500,000 a year in groundbreaking patient advocacy efforts through which we have mobilized a network of more than 300 patient leaders. This part of the Cures Act provides another step in the right direction.
In addition, the Cures Act sets asides funds for initiatives such as the Precision Medicine Initiative (now called “All of us”) and puts measures into place to streamline clinical trials.
As in all legislation that makes its way to the desk of the President, political compromise was a necessity. To pay for provisions in the Cures Act, funding was cut in the Prevention and Public Health Fund that promotes disease prevention. And, there is valid concern that while the Cures Act puts in action core components that will ensure that new therapies get to patients faster, it did not explicitly tackle how we as a nation will address the high price of drugs and therapies and the impact this has on the lives of patients and their families.
Yet overall, the Act is a landmark triumph for great science, a dramatic demonstration of belief in effective public-private collaboration, and a shining example of good old-fashioned American optimism in getting big things done. For PDF, it is a terrific down-payment on a realizable, world-enhancing dream. And for me personally, as I prepare to step down after more than two decades leading one of PDF, it is an exhilarating, life-enhancing moment.