Has FDA ever issued a waiver for a medical device? Here there are great reasons. 1) Initial USA safety (limited) pilot study completed years ago. 2) Two Rigorous independent Italian studies completed. 3) Publication in presitigous journals 4) Followup longer term study demonstrating safer, even life-saving outcomes 5) Dealing with a one-and-done medical device, not a pharmaceutical with possible long-term effects nor with a device utilized long-term, such as an implant, pacemaker, artificial hip, bone, vein, artery, vein, skin, etc., that requires long-term use to discern potential for detrimental effects and failures. 6)Without waiver, no other prospect for immediate domestic introduction of this *life-saving* device. 7)With recent study demonstrating long-term postive outcomes and avoidance of otherwise negative outcomes, postponement of implementation of this device means loss of life, greater health complications, greater monetary larger expenditures for health mainenance. Hence, by granting a waiver FDA is allowing zero demonstrated risk with this device and by refusing waiver it will be causing death and disease, etc.
I agree that from a public safety risk/reward profile, this product is exceptional: however, didn't see a clearly defined process for clinical testing waiver of standard procedures for medical devices:
I know that several prescription products were "fast tracked" and "emergency use waived" on a case by case basis, but that is usually reserved for the "do this or watch them die" scenario. While RG does prevent CIN in a given percentage of at risk patients, it would be difficult to clinically say with certainty that "patient A" would not have died had we used RG.
I think a "fast track" for approval would be more suitable here. You would still need to perform a statistically relevant number of patients, but given the safety profile, the bar (total sample size) could be lowered in the interests of public health.
FDA is under a lot of pressure to toughen up in the device space. They have ruled that a lot of devices were essentially the same as older ones (and therefore needed minimal testing), and too many of those have come back to haunt them. So they can't really bend device rules these days.
On the other hand, PLC's trial design is adaptive: in effect, if results look good half-way through, we can declare victory and go home. That isn't something you want to predict WILL happen, but it can.
Frankly, it makes so much sense for a device company to buy PLC outright that I have trouble understanding why it hasn't happened yet. Mr Tauscher doesn't sound like he's having much fun and the rest of us would probably take 45 cents and say "thank you." And at that price it tould be a steal.