Just so I can remember it

The Devil's drug development dictionary. Warning, off-colour jokes.

Is it just me, or is Eli Lilly asking for a torcetrapib-like epic fail?

From Lilly's press release:

INDIANAPOLIS, May 21, 2009 /PRNewswire-FirstCall via COMTEX News Network/ -- Eli Lilly and Company (NYSE: LLY) today announced it will begin enrolling patients this month in two separate but identical Phase III clinical trials of solanezumab(i), previously referred to as LY2062430, an anti-amyloid beta monoclonal antibody being investigated as a potential treatment to delay the progression of mild to moderate Alzheimer's disease. The trials, called EXPEDITION and EXPEDITION 2, will each include a treatment period that lasts 18 months and are expected to enroll a total of 2,000 patients age 55 and over from 16 countries.

Ok, fine. I'm sure the Lilly solanezumab team is excited and nervous, as this is their moment in the spotlight. But then later on:

Alzheimer's disease theory suggests that amyloid beta clumps together and eventually kills brain cells. Solanezumab binds specifically to soluble amyloid beta and thereby may draw the peptide away from the brain through the blood. In short-term clinical studies, solanezumab appeared to have dose-dependent effects on amyloid beta in blood and cerebrospinal fluid. The clinical studies to date have been too short to evaluate any potential delay in the progress of Alzheimer's disease.

Ok, hit the brakes. I am acquainted with some of the statisticians over at Lilly, and they are very bright people who have contributed a lot to the field. I can't help but think they are more nervous than the people who made the decision to run with a long-term endpoint in Phase 3 based on a short-term endpoint in Phase 2. I've known a couple of epic fails falling into the same category. Throw on top of that that the theory behind it, while still king of the hill, is starting to be thrown into doubt, and Alzheimer's is a graveyard anyway. (Just read this series to see the associated problems.)

It may be standard practice in some circles to run a large trial on a long term endpoint based on a shorter term endpoint or a biomarker (or even 2), but, while I hope this trial succeeds (we definitely need an effective Alzheimer's treatment), I can't put my money on it at this point. I wish them luck.

Deep thought of the day

Biostatisticians should be involved much more than we currently are in the forming of a data collection strategy in clinical trials. Too often we are left in the margins of case report form design, and so we have to deal with the consequences of decisions made by those who don't live downstream of the data collection process.

I have a suspicion that clinical trials isn't the only place where this principle applies.

New edition of Jennison and Turnbull

I just found out that there will probably be a second edition to Jennison and Turnbull's book on group sequential designs (with a slightly different title) coming out next year. Rock on.

(Pharmaceutical) SAS programmers

I've seen more SAS programmers work on short term projects than any other field in the CRO/Pharma/Biotech industry. I don't know how they do it.

I'm all for studying comparative effectiveness, but ...

Eye on FDA explains very well my concerns with comparative effectiveness research.