Biotech Values

[DewDiligence]

VRTX – Does the FDA’s mandate include concern about off-label use?

Officially, it does not; this can be ascertained by reading the FDA statutes, where the only mandate is to assure the safety and efficacy of approved drugs. On the other hand, the potential for off-label use is front and center in many of the FDA’s actual product reviews.

Xarelto is a case in point:: about 45 minutes of the Xarelto advisory panel last week was devoted to a debate of the harm that might be inflicted by potential off-label use for longer than the approved duration of treatment. The FDA could easily have cut off this debate by telling the panel that off-label use was immaterial to the panel’s raison d’etre; that the FDA did not do so reveals the degree to which potential off-label use has been incorporated into the FDA’s mindset.

In short, the point raised by Thomas in #msg-36471993 is spot-on. If the FDA does not approve Telaprevir for second-line treatment prior to the completion of the phase-3 program, concern about off-label use in the first-line setting will likely be the reason. Regards, Dew