GTCB’s Non- Non Sequitur
[More about the U.S. trial design from today’s CC.]
When I heard Dr. Cox say on today’s conference call that the comparator arm of the planned U.S. ATryn trial will use a “prospective historical control,” it made my head spin. After all, how can something be both prospective and historical?
It can’t, but the terminology actually makes sense, as explained to me by Tom Newberry from investor relations this afternoon.
The historical appellation applies because the patients who will comprise the trial’s control arm have already been treated with plasma-derived antithrombin. The prospective appellation applies because the investigators charged with selecting these control patients will not have access to the patients’ outcome records; rather, the patient-selection clinicians will see only the entrance work-up reports that were prepared when these patients checked in to the hospital for surgery or childbirth.
This, the clinicians will have no opportunity to cherry-pick patients for the control arm to make ATryn look better by comparison. Moreover, the patient-selection clinicians will not be the same individuals as the clinicians who will evaluate the patient outcomes, creating yet another layer of blindness to assure the integrity of the results.
I must say that I am impressed that GTCB was able to negotiate this trial design. Only a year-and-a-half ago, the FDA was insisting on a conventional, large, randomized trial that would have taken forever and a day to enroll patients.
Assuming that things proceed with the FDA as GTCB says they will, a great deal has been accomplished. The detractors of Dr. Cox may be dead wrong in their assessments of the CEO’s management and negotiation skills.
“The efficient-market hypothesis may be
the foremost piece of B.S. ever promulgated
in any area of human knowledge!”
