As a philosopher of science and statistics, as well as a sometime trader in (those dangerous) biotech stocks, I realize that what is warranted inferentially need not follow what appears to be licensed/unlicensed by the straight and narrow path of officially sanctioned statistics. Understanding the background theories, history, detailed data, and assorted rulings are relevant for evidential grounds, which (despite what we might sometimes think) are rather different from legal grounds. (Grounds for stock trading decisions take one to yet a third and different world, but there are intersections). I only heard of the particular (Actimmune) episode mentioned in my previous blog entry from reading Schactman’s recent post[i], and have only a smattering of the background—some of which might shift the initial impressions of readers. As I’m about to leave London (not even time for a pic), I’ll just post the controversial press release itself, posted on (Dr. Barbara Martin’s) website PATHOPHILIA[ii]:
INTERMUNE ANNOUNCES PHASE III DATA DEMONSTRATING SURVIVAL BENEFIT OF ACTIMMUNE IN IPF
—Reduces Mortality by 70% in Patients with Mild to Moderate Disease—
BRISBANE, Calif., August 28, 2002—InterMune, Inc. (Nasdaq: ITMN) announced today that preliminary data from its Phase III clinical trial of Actimmune® (Interferon gamma-1b) injection for the treatment of idiopathic pulmonary fibrosis (IPF), a debilitating and usually fatal disease for which there are no effective treatment options, demonstrate a significant survival benefit in patients with mild to moderate disease randomly assigned to Actimmune versus control treatment (p = 0.004). These data confirm the survival benefit seen in the Phase II trial presented earlier this year at the 98th Annual Conference of the American Thoracic Society. There was also approximately a 10% relative reduction in the rate of progression-free survival associated with Actimmune versus placebo, the trial’s primary endpoint, but this was not a statistically significant difference.
“We are extremely pleased with these results, which indicate Actimmune may extend the lives of patients suffering from this debilitating disease,” said W. Scott Harkonen, MD, President and CEO of InterMune. “Actimmune is the only available treatment demonstrated to have clinical benefit in IPF, with improved survival data in two controlled clinical trials. We believe these results will support use of Actimmune and lead to peak sales in the range of $400-$500 million per year, enabling us to achieve profitability in 2004 as planned.”
“The mortality benefit is very compelling and represents a major breakthrough in this difficult disease,” said Ganesh Raghu, MD, Professor of Medicine, University of Washington in Seattle, and the Phase III study’s lead principal investigator. “Interferon gamma-1b is the first treatment ever to show any meaningful clinical impact in this disease in rigorous clinical trials, and these results would indicate that Actimmune should be used early in the course of this disease in order to realize the most favorable long-term survival benefit.”
[i]Although it is not unfamiliar when it comes to information intended for investors (as opposed to published items)…
[ii] A chronology: http://bmartinmd.com/2008/03/intermune-chronology.html