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.

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  • The FDA is a complete disgrace and blight on the Americn people.They accept millions in bribes from drug companies every year to approve and “fast-track” dangerous poisonous chemicals disguised as medicine. The problem is that the whole system is rigged and has been for over a hundred years. Big Pharma’s poisons don’t CURE anything!!!(except for antibiotics). There’s no money in cures.The whole pharmaceutical model needs to be replaced. It’s high time.It’s time to put to bed the whole “patent medicine” paradigm.This evil that has been foisted upon us has run it’s course. Enough lives have been sacrificed in the name of corporate greed. I hear that Jim O’neill “hates” the FDA,how it operates and what it stands for. I hope that is a fact. I sure do. It’s time for NATURAL CURES to have their day. I certainly hope that that is what this is leading us to.

  • You can’t separate safety from efficacy. You state in your article that most drugs are not safe. Actually all drugs have adverse effects. The question is whether a drug is worth the risk. It might be worth risking death if a drug can cure your cancer. But what if the drug is not effective? You need to know if the drug works in order to decide whether to take the risk. I am also not convinced it will be so easy to prove a drug is not effective using real life data. Not only is there the placebo effect, but there are also the vast propaganda resources of the pharmaceutical industry. Even with more stringent testing there are lots of drugs and procedures being used today that have little or no efficacy.

  • As far as this group is concerned if the drug generates a profit then it is effective.
    Everyone knows profit is more important then health or safety

  • Hopefully the next head of the FDA will recognize electronic cigarettes as harm reduction, and allow the industry to continue offering a product that is proven better than combustion cigarettes. The recent regulations placed on “vaping” products by FDA will kill innovation of a product that can save millions of lives.

  • The asininity of this article is perfectly exhibited in the first sentence. “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.” The fantastic thing about this sentence is Silverman’s use of the passive phrase “being prescribed.” It clouds the issue of who actually is doing the prescribing. In this hypothetical, your doctor is the one prescribing the medication. Your doctor is in a good position to know about the effectiveness of a new drug. If he had “no clue” whether it would actually work he would not prescribe it to you unless he was completely incompetent. Imagine your doctor prescribing a medication to you saying “I have no idea whether this will actually work.” Would you accept that explanation and take the medication? I don’t think so.

    • You are the one being naive. According to a study cited by Dr.Atul Gawande, FORTY FOUR percent of oncologists admit to prescribing cancer drugs that they know won’t work because the patient DEMANDS the drug against all odds. Those drugs aren’t being prescribed based imagination, they are being prescribed for real.

    • How is a doctor in “a good position to know about the effectiveness of a new drug”? If you don’t have adequate information you can’t be in a good position to know anything even if you do have an MD after your name. Many physicians get most of their information about drugs from pharmaceutical company reps or drug company sponsored studies that they don’t critically analyze.

    • How is a doctor in “a good position to know about the effectiveness of a new drug”? If you don’t have adequate information you can’t be in a good position to know anything even if you do have an MD after your name. Many physicians get most of their information about drugs from pharmaceutical company reps or drug company sponsored studies that they don’t critically analyze.

  • On further reflection, I would like an engineer to lead the FDA, and especially, the device unit. They do not let planes fall out of the sky, or build bridges that collapse. They understand materials safety. They understand the concept of Precision Devices and Precision Medicine (testing to insure “right for you” and your allergies, immune system and genetics), making sure you get the right kind – not the wrong kind – of gas in your car. They look at the big picture and study all the data – the wall built between medical and dental records and health impacts would come down. Imagine the possibilities!

    In addition, FDA needs to reboot the Expert Advisory Panel system where specialists in an organ or body part review and make decisions about devices that are inserted in that organ or body part. To understand systemic impacts, all Panels should also include an allergist, immunologist, geneticist, functional medicine physician, and biologic dentist. You need all of the elephants in the room to consider benefits, risks, and ways to modify and limit risks of installing devices with 24/7/365 impacts on a genetically and medically diverse population.

  • I’m not sure how you would define the “right” person for the job. After all, Les Crawford was a horse doctor and a stock swindler.

    Let’s keep this party going!

  • The FDA has abrogated its responsibility to properly evaluate new drugs for efficacy, although it doesn’t do a bad job on safety. No new initiatives are required. Just dust off an oldie but a goodie as Marty McFly would say. Between 1938 and 1962, because of the massive number of new drugs approved and due to personnel shortages at the agency the FDA subcontracted efficacy reviews to the National Academy of Sciences under the DESI review program. Under this program drugs were classified as either effective, probably effective, possibly effective or ineffective. If the drug was found to be less than effective the manufacturer could submit additional data (“real world evidence”) to the New Drug Evaluation Office. If the decision was made to upgrade the product to “effective” status a notice was published in the Federal Register. If the drug was classified as “other than effective” the company was provided with a Notice of Opportunity for a hearing before an administrative law judge.

    Although the intent of the DESI review was to retroactively remove ineffective products from the market (almost 900 products were removed between 1968 and 1980) it could be re-engineered to approve drugs in a prospective way, with the FDA basically assuming the role of a rubber stamp agency with regards to efficacy.

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