Pharmalot... Pharmalittle... Good Morning

The Vertex story illustrates how studies are now designed to meet the expectations of the Wall Street power brokers. Here's the quote:

"The 119 investors surveyed, on average, expected to see a 6.7% improvement in placebo-adjusted lung function from the high dose VX-809 arm of the study. This is precisely what Vertex delivered."

Traditionally the delta for designing the Phase III study (expected degree of improvement) is predicted by the Medical Director of the pharma company based on Phase II data and other avaible evidence without any outside help. He/she then sits down with the biostatistician who provides the required sample size based on the anticipated delta, the desired Power (usually set at 80-90%), with the significance level usually set to 0.05 for efficacy. From there the sample size goes into the protocol and the study proceeds.

It seems now that The Street is telling the company what the delta should be in order for the drug to make a splash. The logical next step to me is to have the guys from Morgan, Goldman Sachs, et al actually sit on the Protocol Development Committee instead of seeking outside input. Would be a great time saver, IMO.

Another superb reading list, Ed. Thanks!

Why do we need a new drug for vaginal dryness? My friend the Wyeth rep sent me a case of Premarin Cream when my ball and chain hit the BIG M. Seemed to work fine for her and it's on the generic market.

A little dab will do ya!

Let's ditch these equivocal CF drugs and go full bore with gene therapy. Initial single dose studies in children using liposomally delivered transgenes have shown promise, and now it's on to multidose studies. See below:

http://www.cfgenetherapy.org.uk/publications/daviesnacfc2011.html

@oii who opined, "...It seems now that The Street is telling the company what the delta should be in order for the drug to make a splash. The logical next step to me is to have the guys from Morgan, Goldman Sachs, et al actually sit on the Protocol Development Committee instead of seeking outside input. Would be a great time saver, IMO...."

You mean this "Street"? The ideology whose DOGma consists of a nihilistic "wholesale rejection of empirical observable facts"? Talk about IMAGINARY metrics for the bench scientists! Always set up to FAIL....

Cut and pasted:

“The crowning achievement of the academics who devised modern free market fundamentalist ideology was the wholesale rejection of empirical observable facts . One of the most extreme permutations of this school of thought, The Austrian School of Economics, even rejects the scientific method. You can’t make this stuff up!

Instead of facts, they embrace false “axiom” systems, which are basically unsubstantiated opinions on human nature that are treated as though they were empirical facts in order to guide their ideas. That’s why most mainstream economists don’t take the Austrian School Seriously, even though a less extreme forms (like the Chicago School of Economics, which inspired a US-backed military dictatorship in Chile) have dominated US Economic policy & global economic development for the past 30 years.”

dz I prefer the Chicago school of thought myself, you know the one where students must include the phrase "Greed is Good" in their morning and evening prayers.