Republican presidential candidate Sen. Ted Cruz (TX) and Sen. Mike Lee (R-UT) just introduced a new bill that would completely overhaul the Food and Drug Administration.
The main idea: The senators want to expedite approvals for lifesaving drugs, give Congress space to intervene in FDA decisions lawmakers don't like, and expand the drugs and devices Americans have access to by allowing products approved in other countries onto the market.
More specifically, here's what the Reciprocity Ensures Streamlined Use of Lifesaving Treatments Act, or RESULT Act, would do:
- Allow for "reciprocal approval of drugs, devices and biologics" from "trusted, developed countries" like the European Union, Israel, Australia, Canada, and Japan. This means that if a drug or device is approved in one of these countries, citizens here could access it — and vice versa.
- Create a 30-day window for FDA review of lifesaving drug and device applications, in order to speed them up.
- Instruct the Health and Human Services secretary to approve a drug, device or biologic if "the FDA confirms the product is lawfully approved for sale in one of the listed countries; not a banned device by current FDA standards; there is a public health or unmet medical need for the product."
- Give Congress the power to overrule FDA decisions about promising applications for lifesaving drugs that the FDA rejected.
On its face, this looks like a win for patients
There's definitely an argument to be made for more efficiency in the global drug approval system. Why should all these different national drug and device regulators replicate each other's work by combing the same data sets and coming to similar conclusions about which medicines make it to market?
By streamlining this process, Americans could have access to more treatment options from other jurisdictions, including potentially lifesaving medications that are already approved in other countries that they can't access here. Patients and doctors in other countries — many of which rely on FDA data before making their own decisions — would also expand their medical arsenal.
That seems to be why economist Alex Tabarrok was excited about the bill. As he has argued, "There is an invisible graveyard filled with the bodies of people who would have lived if more new drugs had been available and sooner." Anyone who has had a loved one struggle with a disease with few medical options to turn to knows why a more open system is compelling.
I've also talked to drug developers in other countries, such as France, Germany, and Italy, who have complained about what they view as the FDA's bias toward American-made drugs. I haven't seen any studies on this question (please email me if you know of them!), but these developers all provided examples about drugs in their respective fields that haven't gotten the same treatment by US regulators as lesser drugs made by American companies. Maybe a reciprocal agreement could fix that.
But drug and device regulation is extremely variable by country
On the other hand, drug and device regulation can be extremely variable depending on the country. Consider the problem of medical devices in the EU. It's well-known that device makers in Europe, for example, sometimes go to countries with more lax regulatory standards in order to get a device (of potentially dubious quality) onto the market everywhere in the EU.
If this bill passes, then what if this type of gaming of the system happens on a global scale? The regulatory news service Regulatory Focus noted that the FDA is considered the most stringent approval system in the world, so it's not clear that regulators here would suddenly accept drugs or devices from countries with lower standards. But I'd argue that it could open the gateway for that, unless protections are put in place to avoid a "race to the bottom."
"This would completely marginalize the FDA," said Aaron Kesselheim, a professor of medicine at Harvard Medical School. "It would allow the decision-making in other countries to have full power over the market in the United States."
Still, the reciprocity part could be the least worrying aspect of the bill. More baffling is that decisions about drug approvals — which right now involve doctors, clinical trials experts, toxicologists, and epidemiologists poring over reams of detailed clinical data and statistical analyses — would be handed to Congress to overrule.
Health professionals and scientists comprise fewer than 10 percent of Congress members. Are these lawmakers really equipped to make medical regulatory decisions? This could further expose scientific decisions about drug safety to political influence and lobbyist meddling. This is probably the most dangerous idea in the bill.
Is the regulatory process really slowing things down?
Like the House-passed 21st Century Cures Act, the underlying premise of the RESULT Act is that a clunky regulatory process is delaying cures from getting to patients.
But it's not clear that this legislation can solve the biggest problem here — the lack of promising treatments in the pipeline. In other words, a faster approval process can't fix a dearth of innovation from labs themselves.
"There's no evidence the FDA blocks innovation or makes innovation harder or makes it more costly," said Kesselheim. "The goal in drug development isn't merely innovation, it is innovation that works to help patients. When drugs are shown to be effective and safe, the FDA is the fastest regulatory agency in terms of approvals of new drugs in the world."
Right now the FDA is actually the fastest regulatory system in the world, with several expedited approval pathways for important drugs. Last time we looked, the agency had approved 96 percent of the applications that came its way in 2015. (You can read more about that here.) So, again, how this new legislation — which further speeds up approvals — will solve the real reason better medicines aren't getting to patients remains to be explained.