Taking Pediatric Medicines Development off the 5 Year Plan

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  • 04/04/2012

We called it.

At CMPI’s November 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.

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.

Well, a bipartisan House bill now proposes to permanently reauthorize two pediatric-drug statutes that include marketing incentives and clinical-study mandates, which the drug industry supports, while also giving FDA recourse for unfinished pediatric studies and requiring drug sponsors to submit their plans to the agency earlier, measures advocated by pediatricians.

Reps. Ed Markey (D-MA), Anna Eshoo (D-CA) and Mike Rogers (R-MI) introduced the bill to permanently reauthorize the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act.

The bill would require drug makers to submit an initial pediatric-study plan by the end of phase 2 clinical trials. Currently, companies must submit the information when they submit an application or supplement to the agency. FDA must promulgate a rule and guidance on the new timing requirement, according to the bill. The plan should include an outline of proposed studies and any request for a waiver to defer the requirements.

Lawmakers backing the bill said the pediatric laws have led to pediatric studies for 180 new drugs and more than 400 updated drug labels.

“Government and private industry have worked together to fill an important void,” Eshoo said in a statement. “The laws have provided greater transparency and accountability in the programs, while improving communication between FDA and companies. I’m proud of how far we’ve come and look forward to a day when we know how all medicines will affect children.”

Now sponsors are looking for a little HELP.


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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