House of Representatives passes the 21st Century Cures Act – December 1, 2016

Yesterday, the US House of Representatives passed the 21st Century Cures Act, a sweeping bill to revamp medical research funding and the FDA’s drug and medical device approval process. This is more than two years in the making – the thousand-page bill has been in development since April 2014, and passed the House by an overwhelming margin of 392 to 26. The bill dedicates more than $6 billion to key medical research initiatives, including $4.8 billion to the NIH and $500 million to the FDA. On the device and drug development front, the bill supports reforms in (i) modernizing clinical trial design, (ii) better incorporating patient voices into the FDA decision-making process, and (iii) improving the FDA’s ability to hire and retain leading scientific experts. Although the bill has been criticized for potentially weakening the standard for evidence at the FDA, it has also garnered wide support from advocacy groups, drug and device companies, and the Obama administration itself, which released an official statement saying that “this legislation offers advances in health that far outweigh these concerns.” A Senate vote on the 21st Century Cures Act is expected early next week – if the bill passes there as well, it could be approved by President Obama by year-end.

Notably, the House of Representatives passed an earlier version of the 21^st Century Cures Act in July 2015 – it was rejected by the Senate, and revisions of the bill are geared to ensure that this time, it passes. The changes are not insignificant. NIH funding has been cut and has gone from guaranteed to discretionary. Still, we anticipate that the Act will have positive implications for people with diabetes and obesity. We eagerly await the Senate’s decision.

• The Act dedicates >$6 billion to key research initiatives, including $4.8 billion to the NIH. This funding will be delivered over the course of 10 years to support programs such as the Obama administration’s Precision Medicine Initiative, the BRAIN Initiative, and the Cancer Moonshot. The bill also includes $1 billion in funding to combat the prescription opioid and heroin epidemic, and offers grants for the improvement of mental health services. In our view, this research funding is a clear win for patients awaiting better treatment and overall care. These funds are supporting innovation in currently under-resourced research programs, which is great to see. That said, there are noticeable changes from earlier versions of the bill, which allocated $10 billion in mandatory funding to the NIH. In contrast, the bill as it stands only allows the NIH access to its smaller pool of money if Congress reauthorizes the appropriation of these funds each year. In light of the uncertainty as to President-Elect Trump’s precise plan for healthcare, the current bill cannot guarantee funding for all 10 years.
• One of the Act’s key aims is to shorten the time it takes new drugs and devices to reach the market. Provisions of the bill encourage FDA approval of breakthrough therapies to be based on early-stage safety and effectiveness data, allowing additional clinical trials to occur after the drug or device is already available to real-world patients. The Act further allows drug companies to submit “summary-level reviews” instead of raw data for new indication approvals. On the plus side, these provisions will bridge the gap between innovative research and patients in-need – as we see it, this gap is far too wide currently for many disease areas and definitely in diabetes. In type 1, for example, there is great potential for stem-cell based therapies and artificial pancreas devices, and we’d certainly be happy to see faster patient access to advanced treatments.
  
  • We acknowledge the concern that the FDA may loosen standards to make this happen, but also see how the bill could incentivize more investment in promising drug development and ultimately, could lead to better drugs. In a recent JAMA editorial, noted cardiologist Dr. Rita Redberg presents the trade-off: “In our rush to find new effective treatments, we should not harm our patients with ineffective toxic ones.” Members of congress, most notably Elizabeth Warren (D-MA) and Bernie Sanders (D-VT), have also expressed worry that these provisions give too much power to drug and device companies by limiting FDA access to primary data. Importantly, the Act promotes faster approvals with the end goal of better drugs. Drug and device developers play an undeniably vital role in healthcare by bringing the next generation of therapies to market, and a shorter approval process for the US market could incentivize and maybe even accelerate innovation. And, if the FDA is given more resources, there is no reason for evidence standards to fall.
• The Act calls for a structured framework for consideration of patient experience data in the review process. We’ve been waiting for this one for a long time – a patient-centered approach to the drug development and review process. In the context of diabetes, the recent FDA workshop on Outcomes Beyond A1c was an excellent step in the right direction – so many players, spanning from regulatory to industry, acknowledged that we need to more systematically consider what patients care about most (including quality of life measures) in evaluating therapies. If the 21st Century Cures Act passes, we are optimistic that the future holds even more discussion of patient-centered outcomes.

-- by Abigail Dove, Payal Marathe, and Kelly Close