What Ted Cruz Gets Wrong About The FDA: Tough Regulation Can Spark Innovation

Last October, Republican presidential hopeful Senator Ted Cruz laid out a plan to fix the Food and Drug Administration in The National Review, a conservative weekly. “We need to tear down the barriers blocking a new era of medical innovation, and the primary inhibitor is the government itself.”

Cruz’s main example that the FDA is being too tough? A multiple sclerosis drug called Lemtrada, developed by Sanofi . Cruz claimed the FDA was refusing to approve the drug because studies of the drug did not fit the agency’s “placebo testing requirements.” One problem there. When Cruz wrote his piece, Lemtrada had been approved for an entire year. That error didn’t keep Cruz from introducing a bill that would force the FDA to honor approvals made by regulators in Europe or Japan.

In reality, the FDA is approving 9 of 10 new drug applications that are filed. Drugs for serious diseases are making it from test tubes to the market faster than ever before. What Cruz, and most FDA critics, miss is that there is a delicate balance to what the FDA does. True, tough regulations stifle innovation, keeping innovators from even trying. But they also can spark it, setting the bar high enough that patients get the effective drugs they want and need–the ones that will save their lives.

Sometimes, breakthroughs happen because the FDA demands them.

Like what? The first diabetes drugs ever shown to prevent heart attacks and strokes, for one. Last November, results were published in the New England Journal of Medicine showing such a benefit for Jardiance, from Eli Lilly and Boehringer Ingelheim . Last week, Novo Nordisk announced that its Victoza also prevented the combined endpoint of heart attacks and strokes.

It’s likely neither of these studies would have even been run had it not been for FDA–and controversy. In 2007, Steven Nissen, the chairman of cardiology at the Cleveland Clinic, was on the warpath. He’d raised concerns that Avandia, at the time the most widely used diabetes pill in the world, actually caused heart attacks.

Nissen’s proposal: If a company wants to sell a diabetes drug, it needs to do a study to try and prove that the drug reduces heart attacks and strokes. Even if the study fails on that goal, it’s likely to show that the drug is safe. Industry representatives initially rejected to the idea, but it got a major boost when Nissen’s rival, Duke cardiologist Robert Califf, also supported it. Califf is now the FDA’s commissioner.

To make sure that the new goalposts didn’t slow diabetes drugs too much, the FDA used a compromise also proposed by Nissen. Drugs could be approved when studies were halfway done, so long as the data were kept secret between the drug maker and the company until the trial was finished so as not to mess up the scientific validity of the study.

And here’s where we see that forcing the FDA to do what they do in Europe or Japan, as Cruz would want, doesn’t make sense. This way of testing new drugs for safety was so successful with diabetes medicines like Jardiance and Victoza that the FDA exported it to the field of obesity drugs. One of the main reasons to lose weight is to reduce one’s risk of heart attack and stroke. But, again, obesity drugs have a history of doing the opposite.

One obesity drug maker, Orexigen, hired Nissen himself to run the heart trial for its drug, Contrave. But Orexigen's executives broke their promises both to Nissen and to the FDA by releasing data from the study prematurely, first to 100 people connected with the company and then, after the drug was approved, to the world, in a patent, that claimed Contrave did prevent heart attacks and strokes. An FDA official went public calling the premature results "misleading" and "wrong," and Nissen himself issued a press release showing that more complete data showed no such thing. Results published today in the Journal of the American Medical Association confirm that Contrave had no such benefit.

Approving Contrave was a mistake, possibly encouraged by the FDA's recent permissiveness. This may mean we never know what the effect of the drug on the heart is. Results are not expected until 2020. Do we really want the FDA to have to make a decision like that because they did it in Japan?

Companies might even benefit from a  tougher standard. New cholesterol drugs launched by Amgen and Regeneron/ Sanofi last year were supposed to be blockbusters. But because they were approved before big studies that would prove they prevent heart attacks and strokes completed, insurers have been able to limit their use. The companies might have been better off waiting for more evidence.

Are there examples of drugs the FDA should have approved faster? Sure. Ted Cruz couldn't manage to find one, but I can: Roche's Esbriet, a Roche drug to treat scarring of the lungs that was kept in regulatory purgatory for years. And hurdles for new pain drugs that work by keeping nerves from growing, or for new antibiotics, may have made it difficult to address public health crises like the opioid epidemic or the crisis of antibiotic resistance. But there are drugs the rest of the world wrongly approved a drug and FDA didn't. (Example: Sanofi's weight loss pill Acomplia, which caused suicidal thinking.)

The problem is that a regulator like FDA doesn't need to be lax or tough. It just needs to be right. And the best way to do that is to base decision-making on science.