“It’s at least possible that if I know we’ve got to show actual, substantial benefits, or that it’s got to be novel in some way, that I might push harder,” says Dr. Steven Joffe, a pediatric oncologist at the University of Pennsylvania.

Dr. Gottlieb and others say that patients facing long odds and potentially fatal diseases don’t have time to wait for more clinical trials. That’s a fair point. Reasonable people can disagree over where the fulcrum between speed and evidence should be placed. But a new drug is only innovative if lives are extended or improved, and we can’t know if they will be without more data.

Based on the data we do have, the thousand-plus cancer drugs now in clinical development are quite likely to help only a handful of patients, and only a very little bit: According to one recent study, targeted cancer therapies will benefit fewer than 2 percent of the cancer patients they’re aimed at. That reality is often lost on consumers, who are being fed a steady diet of winning anecdotes about miracle cures. Those stories are heartening, especially if you or someone you love is one of the people battling the long odds who could be helped. But they omit a lot, including the number of people who aren’t saved — or even helped — by a given drug, and the likelihood that any given success would have occurred even without the new medication.

The Food and Drug Administration is still the world’s leading regulator of medicine by far, setting the bar for countries around the world. Lest it lose that standing, the agency should demand more of the drugs (not to mention medical devices) it approves.

Requiring at least two successful clinical trials for any drug — as the government did until recently — would be a great start; it would sharply reduce the odds of false positives (drugs that show benefit but only by pure chance). It would also help to set minimum benefit standards, requiring that a drug improve patients’ lives and health by a certain, measurable amount.

Another thing federal officials can do is to use independent cost-benefit analysis to set a drug’s list price. The United States is the only developed country in the world that doesn’t do this, and the result is that the prices patients pay for medications often have little to do with how much benefit is derived from them.

Officials in the past have rejected this approach because of concerns that consumers would be robbed of choice, either because an insurance company would force them to switch from their current medication to one that it determined was fairly priced, or because pharmaceutical companies would choose not to market drugs that they thought were not priced generously enough.

But consumers, who now rank steadily climbing drug prices at the top of their list of health concerns, might be willing to make these trade-offs. At the very least, people who rely on medications deserve a better sense of what they’re paying for.