April 28, 2021
I’m Dr. Diana Zuckerman, president of the National Center for Health Research. Our center is a nonprofit think tank that scrutinizes the safety and effectiveness of medical products, and we don’t accept funding from companies that make those products. I am trained in statistics, clinical trial design, epidemiology, and public health and was a faculty member and researcher at Yale and Harvard and a Fellow in Bioethics at Penn. I’ve also worked at HHS.
The details differ but in both cases our statistical and research analyses support the FDA findings that the data do not confirm the indication.
That’s especially important because both Tecentriq and Keytruda cause substantial adverse events and an alternative treatment has shown clear benefit!
FDA grants accelerated approval with requirements for post-market RCTs to evaluate overall survival to ensure clinically meaningful benefit. But the randomized clinical trials conducted did NOT show benefit. How could FDA continue to offer accelerated approval for any drugs in the future if post-market RCTs results are ignored?
Most of you are clinicians and you’re used to trying different types of treatment in hopes that something will work. But the rules for FDA approval are different. Shouldn’t cancer patients be eligible for free treatments in clinical trials instead of paying for treatment that isn’t proven to work – and that has risks?
Can other studies be used to confirm the indication? FDA explained the problems very clearly.
Not appropriate to use studies with data based on patients that aren’t for the same indication: #1) PD-L1 high and #2) not eligible for cisplatin or other options.
Merck is not a start-up company – they should conduct well-designed studies. If less appropriate studies are accepted as alternatives, does this create disincentives for all companies to do the well-designed studies they agreed to do?
Our analysis agrees with FDA that neither progression-free survival not overall survival were clinically meaningful or statistically significant. What is the justification for keeping the indication for years while awaiting data that might or might not support approval?
FDA also points out that Real World data must meet scientific standards to confirm meaningful benefit.
Patients deserve treatments that provide meaningful benefits that outweigh meaningful risks and they need to be able to trust that FDA approval confirms that.
As a cancer survivor myself, I know that patients want hope.
What are the ethics of your decision today? FDA approval has always meant rigorous evidence – it isn’t supposed to be based on wishful thinking or speculation about cross-over data or post hoc manipulation of data. I respectfully urge you to listen to what the FDA scientists told us repeatedly in their memo — the evidence DOES NOT support continued approval and using other studies can’t provide the data needed.
In conclusion, patients have suffered from taking cancer drugs that aren’t proven to work. Other treatment options are proven to work. Physicians can still choose whatever treatments are on the market, but treatment decisions shouldn’t be based on the mistaken belief that these drugs are proven effective for advanced urothelial carcinoma.