There is yet another twist in the ongoing and always interesting Provenge saga. The Centers for Medicare & Medicaid Services, which last June issued a surprise decision to conduct a National Coverage Analysis, which will be used for coverage guidance by Medicare contractors, has released the questions to be answered at a Nov. 17 panel meeting and the focus is on on-label efficacy.
The NCA, for those who may not recall, stunned all sorts of people because such reviews do not occur very often (see here and here). Dendreon execs subsequently downplayed the move by telling analysts NCA was sparked by concerns of off-label use, although CMS has been cryptic about its reasoning other than to point to the fact that Provenge is the first in a new class of treatments that use a patient's own cells to stimulate the body's immune system in an approach known as immunotherapy.
In any event, at least one analyst noted the questions are unexpectedly about approved usage. "Surprisingly, many of the questions focus on EFFICACY in the ON-LABEL indication. It asks the panel to rank efficacy on a scale of 1-5, leaving the possibility of an ambiguous outcome," writes ISI Group biotech analyst Marc Schoenebaum. "This is the first time we’ve seen Medicare analyze the efficacy of a drug."
However, RW Baird analyst Chris Raymond notes the quetions "aren't unprecedented." Of the nine questions, he agrees that the focus is on efficacy, "but multiple questions also focus on whether the evidence is applicable to off-label use." He then adds several recent such meetings contained similar lines of questioning.
Among other things, the panelists are asked to rate their confidence in Provenge's ability to improve overall survival, control disease related symptoms and minimize the burdens associated the vaccine while maintaining overall survival and control of disease-related symptoms. You can see the list of questions here.