Replies to post #3353 on GTC Biotherapeutics (fka GTCB)

[keitern]

Good info, and thanks.

[alia]

re off label market, additional numbers
i just realised i left out one step of my thinking in my earlier post which might be relevant to the question of off label market size to you though: among those 4000 university hospital ICU patients, I have 50 that get antithrombin and 30 that by ICD code might be HD or AD, my assumption (based on prevalence data of HD and what else is wrong with them) is that it is AD for all or most of these 30 patients. These patients were randomly selected among all incoming ICU patients entering these ICUs (mainly surgical, but also internal medicine and neuro ICUs) within the study period of 2 months in 2005 to provide a reasonably representative snapshot of care during a particular (random within the year) point in time.
So, extrapolating wildly, let's assume I have 1 patient among those with HD, that leaves me 29 patients with AD who got antithrombin and another 20 on top of it receiving antithrombin which probably do not fall into either the HD or AD category since more than half of all patients in this database came from centers of care with PDMS systems which automatically assign (lots of ) ICD codes and not just one or two main ones. So, taking these numbers with a pinch of salt, the potential off label market in German hospitals does not look insignificant compared to the HD indication, provided Atryn is priced reasonably (see previous post) and provided it is marketed well (personal view for Germany: don't mention the cuddly goats but focus on a competitive price).

[shears]

If Atryn is priced lower than plasma derived ATIII then one would assume a pressure on hospital formularies to stock the lower priced and 'safer' version and substitute it whenever the plasma product is prescribed? Just a thought. I know hospitals do that all the time in the US for conventional drugs. Why not for Atryn? Just thinking out loud, but it makes sense to me that hospitals in EU face the same economic pressures. Also, as data starts coming out on aquired AT deficiency more off label use may ensue. I'm thinking if plasma derived ATIII is currently used in aquired deficiency states Atryn (particularly if less costly) might be substituted. Perhaps just wishful thinking. Does anyone have access to the plasma derived ATIII list of indications?

[DewDiligence]

Reimbursement in Germany came up on NVS’ CC this morning with respect to Lucentis. Here’s what was said in reply to a question:

“In Germany and certain other countries, until full reimbursement is granted they’re managing this process through the hospitals and they just want to make sure the patient has been appropriately diagnosed and that specialists are administering the drug correctly.”

In other words, it’s possible for a clinician to request off-label or pre-approval reimbursement for an individual patient, but there is a considerable amount of red tape involved.