The New Prominence of Comparative Drug Trials

Roche announced on Dec. 2 that its experimental diabetes drug performed better in a trial than Merck's (MRK) $2 billion-a-year blockbuster Januvia. This gave the Swiss giant potent bragging rights—and it was part of a pattern. Six weeks earlier, Roche trumpeted data showing its treatment was superior to a diabetes drug from Eli Lilly (LLY). Next year it will wrap up two more trials, pitting its drug against diabetes products from sanofi-aventis (SNY) and Japan's Takeda.

Drug companies have always been ambivalent about this kind of head-to-head trial. Executives fear they'll spend millions on such tests only to find their drugs don't stack up against the competition. But the health-care reform debate is forcing them to face a new reality: Washington wants proof that expensive therapies are as good as or better than cheaper alternatives. A bill being debated in the U.S. Senate proposes setting up a nonprofit institute to perform "comparative effectiveness" research, which would pit therapies against each other in controlled trials.

EASY TO SPINSo drugmakers are learning to live with comparative trials—and are even flaunting the results. They're weaving the information into marketing messages, much to the chagrin of longtime industry critics. Spinning data is relatively easy because companies are not always required to publicize trials that don't go their way. And if the results are mixed, drugmakers can tweak the message to accentuate the positive. Who should sort all this out? "There have to be rules of evidence in a neutral forum that can be trusted," says Sean Tunis, founder of the Center for Medical Technology & Policy in Baltimore.

Buried in Roche's diabetes trial announcement is the information that patients taking its drug sometimes reported nausea and vomiting. But no details are provided about how the drug compared with Merck's Januvia on side effects. That makes Barclays Capital (BSC) drug industry analyst Tony Butler suspicious. "I think it will bear out that Roche's side effects are high," he says. Roche won't reveal further details until the trial is accepted for presentation at a medical meeting. In a written statement, Merck says: "We look forward to reviewing the complete results."

The drug industry has long been under pressure to prove its new treatments are worth the expense to patients and payers. Some overseas regulatory agencies won't approve drugs without head-to-head tests. And health insurers around the world yearn to compare popular treatments in crowded categories such as diabetes and cardiovascular disease.

In the U.S., insurers are designing their own head-to-head trials, in part to eliminate drugmaker bias in the comparisons. Pharmacy benefits manager Medco recently started a three-year trial comparing Plavix, a blood thinner sold in the U.S. by Bristol-Myers Squibb (BMY), with a newer rival, Eli Lilly's Effient. The Medco study will be completed in 2012, right about the time Plavix loses its patent protection and becomes an inexpensive generic option.

Felix W. Frueh, a Medco vice-president, says that the Effient vs. Plavix study will examine how genetic variations affect patient response to the drugs. He says identifying the people likely to benefit from one drug over the other will be a plus for everyone. Medco has slotted 10 such trials in 2010 alone to analyze the effectiveness of various treatments at the behest of insurance company clients. But some critics worry such trials could become a tool to deny coverage on drugs that only help small slices of the population.

Health-care reformers largely support comparative effectiveness but say it delivers on its promise only when all the data is transparent. That may be difficult in a world where drugmakers control the information, says Dr. Jerome P. Kassirer, a professor at Tufts University School of Medicine. "It's naive to think companies are doing anything but marketing," he says.

Before it's here, it's on the Bloomberg Terminal.