This weekly column offers opinions on the latest pharmaceutical industry news.
Imagine being prescribed a medicine when neither your doctor nor the manufacturer has any clue whether it will actually work — because the government never required it to be tested for effectiveness.
That’s not how things are done now, because federal law requires drugs to undergo clinical trial testing to gauge benefits and risks. But the incoming Trump administration may seek to undo a decades-old standard of evaluating drugs for effectiveness — to the detriment of every American who takes a prescription medicine.
One of two people being vetted as the next Food and Drug Administration commissioner gave a speech two years ago in which he suggested the agency require only safety testing for new drugs. After that, good luck.
“We should reform FDA so it is approving drugs after their sponsors have demonstrated safety and let people start using them at their own risk,” said Jim O’Neill, a managing director at Mithril Capital Management, a venture capital firm run by Peter Thiel, the billionaire Trump donor and transition team advisor. “Let’s prove efficacy after they’ve been legalized.”
He is not the right person for the job.
O’Neill is not a scientist or physician. Most FDA commissioners have had such a background, because the training involved in both careers makes it easier to understand the myriad issues involved in moving medicine forward.
So what does he offer? Basically, O’Neill is a policy wonk with good connections and a steadfast belief in the power of free markets. A former policy adviser at the Department of Health and Human Services, he now funds early-stage biotech companies and likes to lambaste the “cartelized” health care system for hindering the development of health care products.
His outlook is better understood in the broader context of a growing movement to revamp the FDA in order to get more drugs to patients more quickly. Of course, the notion of getting medicines out the door faster is a wonderful idea. But lowering or eliminating standards in the process would be dangerous.
“Waiting to figure out whether a drug works after it’s available is naive, because drugs don’t work like the markets for pens or so many other products,” said Daniel Carpenter, a political scientist at Harvard University who has studied the FDA. “We would be turning the clock back nearly a century, and the legitimacy of the American medicine cabinet would crater.”
Indeed, O’Neill’s notion would be like letting the proverbial genie out of the bottle. In some cases, patients may benefit from the famous placebo effect — maybe they think a drug is working simply because they took it. More important, it is likely to be very difficult to organize proper clinical trials to determine effectiveness once a drug is widely used.
The retort to such concerns may be that regulators can rely on what is called “real world evidence,” such as observational studies, insurance claims data, and patient registries, among other things. To be sure, such information is useful, but it also has limits and is not an automatic substitute for randomized controlled trials, the gold standard for safety and effectiveness in drug development.
Real world evidence is gaining ground, though. The concept was included in the 21st Century Cures Act that Congress passed last week by huge margins; the bill gives the FDA leeway to use that standard of evidence to approve additional uses of existing medicines.
Being required to do fewer upfront studies would save drug makers big money. So one would expect the pharmaceutical industry would be enthusiastic about O’Neill’s suggestion, right? After all, why not get behind a proposal that slashes costs — and may ring registers sooner?
But there are some caveats here.
Consider the case of an Alzheimer’s treatment developed by Eli Lilly. Three years ago, test results were disappointing, but the drug appeared safe. Moreover, there was enough of a positive response in patients with a mild form of the disease for the company to pursue a narrower study focused on just those patients. But last month, that test also flopped.
“Under a guy like O’Neill, the Lilly drug would have been approved back then because it looked safe and there was a trend toward improved cognition,” said John LaMattina, a former head of R&D at Pfizer and now a senior partner at PureTech Health, a venture capital firm. “But that would have been a waste, and it’s an example of why his idea is horrible.”
But you may ask, “What’s the harm? If the drug is safe, why not let people try it?”
Well, most drugs are not safe, actually. They’re approved because clinical trials have shown that on balance, they help more than they hurt.
Moreover, this assumption ignores the immense cost that such a scheme could impose on the health care system. Either insurers, taxpayers, or individual patients would have to pay for all those new drugs, without any idea whether they’re likely to be effective. Plus, approving ineffective drugs would likely spark a political and legal backlash. That could sting drug makers.
“Yes, patients and physicians are crying out for something, but if a drug doesn’t work, they will become livid,” LaMattina said. “Companies will get billions in their coffers and, meanwhile, it will look like they foisted snake oil on the public. Payers will go nuts and there will be lawsuits. And this will only kill the industry’s reputation, which is already bad enough. I don’t think anybody would benefit.”
Keep in mind that even if Trump appoints O’Neill and he’s confirmed as the new FDA chief, he cannot unilaterally overhaul the drug approval process.
The 1962 Kefauver-Harris amendments to federal law require trials to demonstrate adequate safety and effectiveness. So Congress would have to get involved to change the system. Still, that seems entirely possible given the Republican penchant for deregulation.
Naming someone with such radical ideas as FDA commissioner would not serve the public. If Trump does tap O’Neill, consider it a prescription for bad health.