Recently BIO released a white paper on post-PDUFA FDA reform. (Full details can be found here.)
Many good ideas – but one that gets the blood flowing (whether you’re for or against): “progressive approvals.”
Sounds good (at least in theory) to many. But there are some tough questions. As an FDA insider opined, What exactly would the standard for progressive approval be? And would every one of the progressive approvals come with some sort of access control?”
Attempting to address some of these unknowns (via an op-ed in the Wall Street Journal) are Michael Boldrin (chair of the economics department at Washington University in St. Louis) and S. Joshua Swamidass (medical professor at Washington University).
They write:
“We can reduce the cost of the drug companies' bet by returning the FDA to its earlier mission of ensuring safety and leaving proof of efficacy for post-approval studies and surveillance.”
I’m not sure where they get the “earlier mission” statement, but let’s allow them to continue.
“In exchange for this simplification, companies would sell medications at a regulated price equal to total economic cost until proven effective, after which the FDA would allow the medications to be sold at market prices.”
Leaving the difficulty of determining what such a “regulated price” might be (and don’t for a minute believe that the devil isn’t in the details), an even tougher question (and a real rabbit hole of one) is the issue of “until proven effective.”
What does “effective” mean? Does it mean “cure” or “remission?” Does it mean "cost effective?" Or extension of life? And, if so, for how long? 5 years? 5 months? 5 minutes? And who makes the call?
According to Boldrin and Swamidass, “Doing so will improve all of our lives, decrease the cost of health care, and unleash the next wave of medical innovation.
It’s a weak argument in support of an important discussion.
Do we really want to open the door for tacit price controls and healthcare technology assessment in return for a very questionable upside?
Safety without efficacy? Really?
Better to pursue a path last publicly discussed in April 2009 when Merck agreed to peg what the insurer Cigna pays for the diabetes drugs Januvia and Janumet to how well Type 2 diabetes patients are able to control their blood sugar.
Now that’s progressive.
