Recent approval of three new cardiovascular drugs by the U.S. Food and Drug Administration (FDA) has raised criticism from some who think the approvals were premature because not enough was known about the drugs. They express concern that widespread use of these drugs will drastically increase health care costs and put patients at risk for long-term side effects that have not yet been evaluated.

The two new drugs for cholesterol reduction (Repatha and Praluent) are injectable drugs that dramatically reduce the blood levels of so-called bad or LDL cholesterol. They were approved before there was proof that the drugs reduce the risk of heart attacks and strokes, a benefit that has been well-established with the current, cheap, generic statin drugs.

The new drug for heart failure (Entresto) is a combination pill that combines a previously approved angiotensin-blocking drug, valsartan, with a new compound that blocks neprilysin, an enzyme that inactivates some beneficial vasodilator substances. This new drug combination exerted a greater survival benefit in patients with heart failure than the comparative strategy of using a generic ACE inhibitor, which has been the standard first step therapy for heart failure.

Criticism of the approval process fails to recognize the laws and precedents under which the FDA functions. Approval of a drug’s use does not imply it is better than other drugs, nor even as good. It means only that the sponsor demonstrated that the drug achieved a targeted goal compared with another agent or a placebo and that it demonstrated adequate safety to justify its use to attain the stated goal.

The problem is the stated goal. With cholesterol, the FDA long ago made a compromise with its traditional standards. Whereas the approval of drugs has traditionally required demonstration that the drug either makes the patient feel better or live longer, the FDA made an exception for cholesterol reduction. For reasons with which I have long disagreed, the FDA determined that LDL cholesterol level was so intimately related to heart attacks that it accepted its safe reduction as a suitable goal for a drug. This measurement has in recent years proved to be an unreliable guide to health and a questionable goal for therapy, but the FDA policy has been difficult to change because it has served as a target for pharmaceutical company drug development.

Use of mortality reduction for approval of a heart failure drug is a powerful end point, but it must be recognized that only a small fraction of patients with heart failure die during the duration of any trial, so the absolute number of people benefiting from the new drug may be modest, and the benefit may not apply to all patients. Only further comparative studies can adequately determine who needs the new drug and who doesn’t.

The only simple solution to concern about the use of expensive new drugs rather than cheap, older and better-studied drugs is to place the responsibility where it belongs: the prescribing caregiver. Drug approval results in usage only if providers prescribe the drug. There may be a place for the new and expensive cholesterol-lowering medications in selected problem patients, and use of the expensive heart-failure medications may currently be justified in a very limited patient population. But the major gap in medical care today has not been the lack of new drugs. The problem is inadequate use of long-approved, powerful agents that can inexpensively prevent cardiovascular morbid events and prolong healthy life. Better education of the medical and lay community is far more important than the FDA in improving cardiovascular health care.


Dr. Jay N. Cohn is a cardiologist and professor at the University of Minnesota Medical School.