The FDA has begun approving drugs in a way that results in knowing less about a drug's effectiveness when it hits the market.
The FDA has begun approving drugs in a way that results in knowing less about a drug's effectiveness when it hits the market. - 

New drugs can literally save a person’s life. Dr. Jay Edelberg believes a new drug his company, Sanofi, has developed may end up doing that by lowering cholesterol, what he calls LDLs.

“The incredible relief that so many of our patients have had knowing that for the first time in their lives, or for the first time after a heart attack, their LDLs are actually at treatment goal has really been stunning,” he said.

Enter new drugs known as PCSK9 inhibitors. Earlier this year, the FDA approved applications from Sanofi and Amgen. Each listed prices running at more than $14,000 a year. Some clinicians believe for select people, this drug may be their only option to lower their bad cholesterol. 

“It sounds like it should be a slam dunk,” said Dr. Steven Pearson who runs the Institute for Clinical and Economic Review. “But we don’t know yet for sure whether they will reduce heart attacks and strokes. And in the meantime they are very expensive.”

Recently, the Food and Drug Administration has begun to approve drugs with more evidence based on what are sometimes called surrogate outcomes. That means when a drug hits the market, insurers, doctors, patients and even the drug companies know less about a product’s effectiveness. Using the PCSK9 drugs as an example, Pearson said it’s more difficult to determine a drug's actual value.

Sanofi said it may take 24 months before it can say whether someone is less at risk for a stroke or heart attack. 

“The price can’t be considered a stagnant proposition. It needs to be a vibrant, living thing,” said FDA consultant Peter Pitts.

Pitts said what must change is that insurers and drugmakers must accept if the drug works, then it should be rewarded. If it doesn’t, he said, don’t pay for it.

Follow Dan Gorenstein at @dmgorenstein