Consider Actimmune. A trial was ended abruptly for the drug, which was being tested for a fatal lung condition. No meaningful statistical difference was found - among 826 patients, 14.5 percent given Actimmune died, compared with 12.7 percent given a placebo.

To the 80,000 or so Americans with idiopathic pulmonary fibrosis, an often fatal scarring of the lungs for which there are no approved meds, this is disappointing. “There still, unfortunately, have been a lot of people who have been taking it on an off-label basis,” Michael Rosenzweig, president of the Pulmonary Fibrosis Foundation, a patient advocacy group, tells The New York Times (registration required).

Investors were also upset: InterMune stock fell 18 percent on the news. The drug generated $90 million in sales last year, but sales derived from two approved uses would bring in just a fraction of that amount. So Intermune didn’t seem bothered about off-label use, even though last fall it agreed to pay $36.9 million to settle charges of off-label promotion.

There was a lot of hope surrounding Actimmune and pulmonary fibrosis, because a 2003 trial of only 18 patients suggested it improved lung function. A larger trial then found the drug didn’t improve lung function, so InterMune began studying prolonged survival, the trial that just ended. Meanwhile, doctors and patients seized on the small study and off-label use grew. Some doctors say hope was misplaced.

“It’s unfortunate that there was so much hype about the drug and that physicians were willing to accept the results of a preliminary study that resulted in many patients being placed on this drug at a very high cost without obvious benefit,” Jonathan Orens, associate director of pulmonary and critical care medicine at Johns Hopkins Hospital in Baltimore, tells the paper.

There are no easy answers to this dilemma. Drugmakers are widely criticized for off-label promotion as a means to boost sales improperly. Henry Waxman just opened an investigation into the practice. But the Abigail Alliance, an advocacy group, says FDA rules are restrictive and is fighting in court. Should someone facing death be denied an experimental med? How should that be decided? And who should decide?

[tags]InterMune, Off-Label Promotion[/tags]