So, who wants to dismantle the FDA, you ask? Some patient advocacy groups, among others, aided by a few senators:
We need the FDA to be more insulated from these forces. Instead, every few years, legislators offer bills that amount to death by a thousand cuts for the agency. The latest is the Promising Pathways Act, which offers “conditional approval” of new drugs, without even the need for the preliminary evidence that accelerated approval requires (i.e., some indication that biomarkers associated with real outcomes like disease progression or survival are moving in the right direction in early drug studies).
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This bill is being pushed by powerful patient groups and has the support of Democratic senators like Kristin Gillibrand and Raphael Warnock, who should know better.
The bill would codify using “real-world data” and unvalidated surrogate endpoints for something called “provisional approval”, a level below the already tenuous accelerated approval.
I can see how it may appeal to patients: you may get a promising new drug for your life-threatening, debilitating disease sooner via this pathway. On the other hand, there are already mechanisms in place that enable access to these: a clinical trial, for one. Or expanded access (a.k.a. “compassionate use”) for those who may not be eligible for a trial.
So how would “provisional approval” help? If anything, wouldn’t it transfer the risks and — importantly — costs of drug development from the drug manufacturer/sponsor/study investigator to the patient?
Ultimately, the reason why there aren’t many cures for rare, terminal diseases is not because the big bad FDA is keeping the already developed drugs away from patients but rather because they are devilishly difficult to develop at our current level of technology. Wouldn’t it then make more sense to work on advancing the technology The careful reader will note that the opposite is being done, and I write this as no great fan of AI. that would lead to those new cures? I worry that the Promising Pathways Act would solve a problem that doesn’t exist by adding to the already skyrocketing costs of American health care. But that could be just me.
(↬Derek Lowe)