Bernie SandersElizabeth WarrenstatementWarren
They are right to be concerned. Researchers who study the FDA have long pointed out that, tucked away in the 996-page bill, are provisions that will weaken the regulator's authority and, potentially, compromise patient safety.
Critics have long worried that the bill weakens the standard of evidence at the FDA
While boosters of the Cures Act see it as a much-needed FDA overhaul, critics have pointed out that the bill — and its previous iterations — includes provisions that will erode the quality of evidence the FDA uses to evaluate new drugs and devices, making it easier for companies to bring substandard or ineffective medicines and devices to market.
As Rita Redberg, editor of the journal JAMA Internal Medicine, wrote in a comment, this could amount to a dangerous trade-off: "In our rush to find new effective treatments, we should not harm our patients with ineffective toxic ones."
One major concern is that the bill includes language that moves the agency in the direction of allowing drugmakers to put forward "real-world evidence" to support the approval of a new indication (or use) for an existing drug or for a new drug that was approved through an accelerated regulatory pathway.
In the past, only rigorous clinical trials would do in these cases. But under this new provision, companies can submit safety monitoring data or observational studies as proof that a drug works. As Joseph Ross, a Yale researcher who has been studying the FDA, said, "This raises all sorts of red flags to me. You don’t know 100 percent how well something works unless you’re studying it in a trial."
Another major concern is a provision that allows drug companies to submit "summary-level reviews" for new indication approvals, in addition to raw data (which they currently have to submit in the FDA process).
Researchers think the resource-starved FDA may rely on these potentially biased summaries — created by pharmaceutical companies — instead of crunching the data themselves. "This undermines the integrity of the regulatory review process, and it limits transparency," Ross said. "In today’s day and age, we’re moving toward greater transparency, more access to data, more data sharing."
Faster approvals don’t mean better drugs
Proponents of the bill say it'll bring cures to patients faster by streamlining the FDA process. But critics have also taken issue with the basic premise that a sluggish FDA process is what’s blocking innovation. (I’ve written about this in detail here.)
"There's no evidence the FDA blocks innovation or makes innovation harder or makes it more costly," said Harvard's Aaron Kesselheim, who has studied previous iterations of the bill. "The goal in drug development isn't merely innovation — it is innovation that works to help patients."
Right now the FDA is actually the fastest regulatory system in the world, with several expedited approval pathways for important drugs — but this wasn’t always the case. The FDA under Ronald Reagan in the 1980s was underfunded and sluggish in approving new drugs, and arguably held back innovation.
What you see in the graph above is that the annual approval rate for new drugs has mostly held steady since the 1950s, with a (short-lived) boost in the 1990s, after the Prescription Drug User Fee Act (PDUFA) was passed. The act allowed the FDA to collect fees on pharmaceutical companies in order to increase funding for the drug approval process, dramatically shortened approval times for drug applications, and led to more drugs coming to market.
But the boost from PDUFA was only temporary. During the late 1990s and 2000s, as drug review times kept falling to all-time lows, there was no increase in new drug approvals. Today, the FDA approves drugs much more rapidly than it did in the 1980s — again, it's the fastest regulatory agency in the world. But the number of new drugs coming to market each year has fallen back to historical averages.
For some, though, the bar is still too high. As Kesselheim told Vox, "There are a lot of powerful financial interests that would like to see reduced hurdles toward getting their products on the market because it can be expensive to run tests to show that your product works."
In a New England Journal of Medicine article, Kesselheim and his colleague Jerry Avorn argued that there are already numerous — and popular — pathways in place for drug companies to seek faster approvals:
A third of new drugs are currently approved on the basis of a single pivotal trial; the median size for all pivotal trials is just 760 patients. More than two thirds of new drugs are approved on the basis of studies lasting 6 months or less — a potential problem for medications designed to be taken for a lifetime. Once the Food and Drug Administration (FDA) starts its review, it approves new medications about as quickly as any regulatory agency in the world, evaluating nearly all new drug applications within 6 to 10 months, an impressive turnaround for such complex assessments.
Daniel Carpenter, a Harvard professor who wrote a history of the FDA, has called the bill the "19th Century Frauds Act." He continued: "The clauses on using purely observational data for drug approval and what amounts to anecdotal evidence for devices are deeply anti-scientific and would undermine the credibility of the American market for drugs and medical devices. ... This act would seriously undermine that credibility, and by extension the market as a whole."
The bill also boosts spending on potentially lifesaving research
Even critics acknowledge that, alongside the provisions that erode FDA authority, there are some very alluring boosts for medical research, as well as funds to address mental health issues and the opioid crisis.
The bill would increase much-needed funding for the National Institutes of Health (NIH) in the order of $4.8 billion over 10 years to help support Obama’s medical research programs, including the Brain Research Through Advancing Innovative Neurotechnologies Initiative and the Precision Medicine Initiative. The bill also includes $1 billion in funding over two years to help state governments with opioid painkiller abuse prevention and treatment programs, and $500 million for the FDA.
The bill's Mental Health on Campus Improvement Act would give universities access to grants for the funding of mental health services on campuses, and expand Medicaid to children with severe mental illnesses.
But there's a catch with this money. While a previous version of the bill included $10 billion in mandatory funds to the NIH, the latest version includes a condition that the NIH will only get the more modest pool of funds if future Congresses sign off on appropriating them. The bill is also paid for by cutting $3.5 billion in public health spending.
"There are lot of good things in the bill," Ross said, "but they are all bungled together with things that undermine the FDA’s regulatory authority." Now, it'll be up to the Senate to decide whether the trade-offs are worth it.