After three years of stalling in the House and Senate, President Obama signed into law the biggest health reform bill since the Affordable Care Act.
The $6.3 billion 21st Century Cures Act is a controversial, bipartisan effort that’s been in the works since April 2014. As its name would suggest, it’s been touted as legislation that’ll help get medical cures to patients faster by boosting health research funding and speeding up the drug regulatory process.
But health researchers have called the Cures Act "lipstick on a pig" and the "19th Century Fraud Act" for good reason. The bill offsets research spending by cutting public health spending. And critics worry the bill could seriously damage the regulatory authority of the Food and Drug Administration.
The funds promised for research aren’t guaranteed
At first glance, it’s not hard to see why the latest version of the law passed the House and Senate in a landslide. It has some genuinely helpful features.
At a time when the health research community is desperate for funds, the Cures Act increases money for the National Institutes of Health (NIH) on the order of $4.8 billion over 10 years. These funds would support President Obama’s signature research programs, including the Brain Research and Precision Medicine initiatives.
The bill also includes $1 billion in funding over two years to help state governments battle the opioid epidemic, and it would expand Medicaid to children with severe mental illnesses. This is all good news.
Yet there are significant strings attached to this money.
For one, the NIH funds aren’t actually guaranteed — they will only materialize if future Congresses sign off on them.
Second, nearly half of the funds for the legislation are going to be paid for cutting $3.5 billion from public health efforts like immunizations and tobacco prevention. We already underfund public health in the US. As of 2012, more than 17 percent of US GDP was spent on health care — drugs, doctors, hospitals — while only 3 percent of the health budget went to public health measures.
So we have a bill that’s giving us medical cures by cutting some of the basic public health funding dedicated to disease prevention.
The bill does worrisome things to the FDA
The main reason health researchers worry about the Cures Act, however, has to do with what the bill could do to the FDA. They say it'll further erode the standard of evidence the agency uses to decide whether drugs are safe and effective.
Right now, before a new drug can be marketed for a particular use, drugmakers need to present high-quality evidence in the form of randomized clinical trials (the gold standard) to the FDA. The bill moves the agency in the direction of allowing drugmakers to submit "real-world evidence" as proof their drugs work when they're seeking approval for marketing a new indication (or use) for an already existing drug. So companies may soon be able to put forward safety monitoring data or observational studies as evidence in these cases.
Now, this would allow the FDA to incorporate more types of evidence than just the randomized controlled trial into its decision-making. But the worry is that, unlike clinical trials, these types of evidence aren't very rigorous. The researchers I spoke to said this opens the door for data dredging, and that there’s really no way to know whether a drug is safe or effective based on that kind of research.
The Cures Act will also allow FDA medical reviewers to use "summary-level reviews" written by drug companies when making decisions about new indication approvals, instead of raw data. Researchers expect that because the FDA is starved for resources, officials will wind up using these potentially biased summaries more often than doing their own analyses.
So there are some good things in the Cures Act, some good-seeming things, and some worrisome things. It's no surprise that more than 1,400 lobbyists pushed for the bill's passage.
Clarification/correction: I clarified/corrected details about the "real-world evidence" and "summary-level reviews" provisions. The "real-world evidence" provision is a stepping stone for the agency to use this lower-quality data; the "summary-level reviews" will accompany raw data, but the FDA is not required to review it.