RE:Papillary Tumors The "disease free survival" rate (which is a time to event endpoint) is not considered as an appropriate metric to evaluate, in a single-arm trial, the efficacy of an adjuvant therapy. Remember that papillary-only tumors are resected before starting any intravesical treatment. Therefore, the patient is considered "disease-free" at the start of the (adjuvant) treatment.
That being said, there is still a chance the FDA say yes and keep on-label the papillary only tumors. But in regards in the briefing document, that chance appears pretty thin.
To my knowlegde, papillary tumors are not an advanced type of CIS tumor. But it could well be the opposite : CIS tumors grow after non-well treated papillary tumors. Remember that patients with CIS containing tumors are ALWAYS categorized in the high-risk group...not true for T1 and Ta papillary only tumors.
Like Dr O'Donnell said at the Adcom, it is believe that only 40 % of CIS tumors is seen with standard cystoscopy, as shown with the introduction of blue light cystoscopy (in the last years). And still, as determined by the FDA review, 80 % of the 129 patients cohort (high-risk patients only) of study 301 had CIS containing disease. My point is a high majority of NMIBC high risk patients have CIS tumors. And the newer technology is now even better at visualizing them.
Those who think that the indication of "CIS-containing disease only" is very limiting are wrong. It probably include something around 80 % of the patients targeted by the "larger" indication. Same proportion that what study 301 showed.
M80