Where Are The Widespread Wonder Drugs?

Dan Hurley investigates. Why a focus on genetics hasn’t paid off as much as hoped:

Gleevec, used since 2001 to treat chronic myelogenous leukemia (C.M.L.) and a variety of other cancers, is often pointed to as one of the great gene-to-medicine success stories. Its design followed logically from the identification of an abnormal protein caused by a genetic glitch found in almost every cancer cell of patients with C.M.L.

Many of the drugs developed through target-based discovery, however, work for only single-mutation diseases affecting a tiny number of people. Seventy percent of new drugs approved by the F.D.A. last year were so-called specialty drugs used by no more than 1 percent of the population. The drug Kalydeco, for instance, was approved in 2012 for people with a particular genetic mutation that causes cystic fibrosis. But only about 1,200 people in the United States have the mutation it corrects. For them it can be a lifesaver, but for the tens of millions of people suffering from more widespread diseases, target-based drugs derived from genomics have offered little.

However, he acknowledges that an “overreliance on genomics is not the only factor slowing down the discovery of new drugs”:

One challenge is that the industry is the victim of its own previous successes. In order to thrive, it must come up with drugs that work better than blockbusters of the past. After all, old drugs don’t fade away; they just go generic. Scannell and Warrington have dubbed this the “Better Than the Beatles” problem, as if every new song in the recording industry had to be bigger than “Hey Jude” or “I Want to Hold Your Hand.

At the same time, the demand for proof of safety and efficacy, not only from the F.D.A. but also from trial lawyers and the public at large, is far higher than in years past. The days when drugs like the original insulin could be sold within a year of their discovery by chemists are long gone, and rightly so.