Well Isn't That Special: Overall Survival Still Oncology Gold Standard

Every biotech that has invested a decade of blood samples, sweat equity and investor tears into developing a drug thinks their “baby” is special, worthy of an exception to the rules. But there’s a world of difference between thinking your kid is the smartest in class, and selling the principal on the idea.

And so the FDA’s cancer drug czar, Richard Pazdur, found himself once again lecturing oncology companies who think their drug should be approved on something other than the gold standard of overall survival (OS), like the faster/cheaper route of progression-free survival (PFS).

A few years back, the FDA issued an oncology guidance document stating that overall survival is usually preferred, but PFS may be appropriate in some cases. Now the agency is being flooded with PFS applications, especially in the wake of last year’s PFS-backed accelerated approval of Genentech’s Avastin (bevacizumab) in breast cancer.

But as Pazdur told BioWorld Insight at the time, the Avastin decision did not signify a new trend. Avastin really was special: It was already approved for other indications, providing a wealth of safety data, and it was being used off-label for breast cancer anyway — something the FDA preferred to control by granting approval.

For most biotechs, PFS trials introduce risky subjectivity and bias unless the FDA is totally on board with the trial design (and ‘on board’ doesn’t mean the agency said “we’d really rather you used OS but we suppose we’ll look at PFS if absolutely everything checks out perfectly.”)

As Pazdur told BioWorld Insight:

Please do not come to me with the statement that ‘We’ve got three clinical experts, Dr. Pazdur, and they all agreed that an eight-week difference in PFS is a benefit.’ Not buying it . . . especially when they are put up at the Ritz-Carlton Hotel.