PDUFA Christmas Tree Ornament for Children

O Tannenbaum, o Tannenbaum,
wie treu sind deine Blätter!

At CMPI’s PDUFA without the Politics conference, each panelist was asked to bring one ornament to hang on the reauthorization Christmas Tree.  Tim Franson (President of the USP Convention and one of the “Fathers of PDUFA”) offered a little lifeboat.

What can we do for those with life threatening unmet needs? One thing, as was done in PDUFA IV, is adding renewal of the Best Pharmaceuticals for Children’s Act. That piece of legislation has done more for pediatric drug development than anything in the past. It’s been good for American children and it should be made permanent. I think the better policy argument is to make it permanent, because companies don’t invest in five-year cycles. (Note to reader: The Act is currently renewable every five years.)

If I’m looking for a return on an investment, I need certainty well in advance of five years on types of studies I should be conducting and I need to know the benefit I’m getting at the end. I think five years is far too short a period of time.

Here’s what Vince Ventimiglia (a former Assistant Secretary for Legislation at the US. Department of Health and Human Services) had to say when asked about renewal vs. permanent authorization:

The argument I heard when we first set the five-year term, and that I heard again when we tried to make it permanent in subsequent years, was that we want to hold these guys accountable. It’s more to use it as a tool against industry -- and hold out the prospect that it could be removed.

To which Father Franson replied:

There’s an accumulated body of evidence, of companies fulfilling their commitments. Maybe its time we make it permanent. I think this point of holding up renewal like the Sword Damocles isn’t very persuasive even to sponsors.

O Tannenbaum, o Tannenbaum!
Das soll dein Kleid
mich lehren.