The grand bargain has a long tradition in American history – the Compromises of Missouri and 1850, New Deal, and 2009 stimulus come to mind, but these landmark achievements also belie thousands of lesser-known legislative compromises over the decades. On December 13th, we added one more to this list: the 21st Century Cures Act. The Act enjoyed widespread bipartisan support, passing the House 392-2 and sailing through the Senate 94-5 before being eagerly signed into law by President Obama. President Obama praised passage of the bill, stating “the bipartisan passage of the 21st Century Cures Act is an example of the progress we can make when people from both parties work together to improve the health of our families, friends and neighbors.”
The Act boosts funding for medical and drug research and development, streamlines approval for new treatments, and allocates resources to opioid treatment and mental health care. It also safeguards some of President Obama’s signature science initiatives such as the BRAIN initiative, Precision Medicine Initiative, and Vice President Biden’s Cancer Moonshot.
And yet, the Act is also a complicated standard bearer for the Obama administration’s science legacy. The bill will introduce major changes to the FDA’s approval process for drugs, devices, and biologics, making it easier for treatments aimed at orphan diseases to reach the market without having to prove their efficacy beforehand. Consumer and patient advocates are worried that the expedited approval process will bring products to the market that aren’t properly vetted, or worse, may present a potential danger to patients.
Much of this is due to provisions requiring the FDA to consider “real-world evidence,” or RWE, such as insurance claims, pharmacy data, and patient medical records instead of relying solely on traditional clinical trials. Because such data is more observational and weakly supported, there is a real concern that it may be unreliable and fail to highlight product risks. “That’s a much lower level of evidence,” says David Carome, Director of the Health Research Group. However, this is being praised by those who see the loosened regulation as an opportunity to bring additional options to patients who in some cases have no treatment options. Gregory Daniel, head of the Brookings Institution’s pharmaceutical and medical-device policy group, argues “We’re making a lot of benchtop, pre-clinical breakthrough discoveries. But we don’t see a lot of breakthroughs translating into therapies.”
Whether a net boon, or setting a dangerous precedent, the full impact of the 21st Century Cures Act won’t be clear for years. However, it is without a doubt one of the largest investments in medical and science research in years. Here are some key provisions included in the bill:
- $4.8 billion in funding over 10 years for the NIH, which will help it to advance biomedical research. NIH Director Dr. Francis Collins has argued, “Our nation has never witnessed a time of greater promise in biomedicine,” and yet for every research proposal the NIH funds, more than four others go unfunded, as Marc Casper and others wrote in The Hill.
- Increased funding opportunities for young researchers, who often struggle to secure grants over more established scientists. As Fortune has explained, “In 1980, new investigators got their first major NIH grant, on average, at age 36; today, the average age is 42. Back then, more than 40% of research project grants went to principal investigators (PIs) under 40; the share in 2016 is less than 10%.”
- A new approval pathway for antibiotics allowing for smaller clinical trials with the aim of getting more products onto the market. A separate provision allows for the quicker approval of drugs specifically treating life-threatening bacterial or fungal infections, as well as regenerative advanced therapies utilizing stem cells.
- “Breakthrough devices” that treat small patient populations (8,000 or less) will be eligible for an expedited approval process without having to demonstrate its effectiveness in treating the disease.
- Almost $500 million in funding over 10 years for the FDA. However, this is far less than the agency has asked for, and some within the agency feel that the additional responsibilities placed on the agency, coupled with decreased approval time frames, will overstretch FDA staff.
- $1 billion allocated to the states to boost opioid treatment, as well as additional funding for mental health initiatives.
- $3.5 billion in cuts from the Prevention and Public Health Fund, which works to prevent Alzheimer’s disease, hospital-acquired infections and other ailments.