Isaiah 2:4 and Fear of Commitment

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  • 01/16/2012

According to PhRMA:

Biopharmaceutical research companies are working to continue this progress and to meet new health challenges specific to children. These include:

• 54 for cancer, which, despite significant progress, is still the leading cause of death by disease among American children.

• 49 for infectious diseases, resulting in more than 164 million missed school days annually in American public schools due to the spread of infectious diseases.

• 48 for genetic disorders, including medicines for cystic fibrosis, which affects 30,000 American children and adults.

• 25 for neurologic disorders, including medicines for epilepsy, which affects more than 300,000 school children under age 14 in the United States

In addition to creating new medicines specifically for children, biopharmaceutical research companies are testing many existing medicines to determine safe and effective dosage levels for children. In 2007, Congress reauthorized both the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA), highly successful programs that generate new knowledge about medicines for use in children. PREA and BPCA are set to expire on October 1, 2012 unless reauthorized or made permanent by Congress.

BPCA and PREA have led to hundreds of pediatric studies covering more than 16 broad categories of diseases that affect children.  The research conducted as a result of BPCA and PREA has led to nearly 425 pediatric labeling changes since 1998, according to the FDA.

Crucial information as Congress considers reauthorization.

As Tim Franson (former regulatory guru at Eli Lilly & Co. and currently President of the USP Convention) said at the Center for Medicine in the Public Interest’s Capitol Hill conference on PDUFA:

These companion pieces of legislation have done more for pediatric drug development than anything in the past. I recall being involved in this when I was doing clinical trials. Very difficult to get companies to do studies on young children. This has stimulated that. It’s been good for American children and it should be made permanent.

Why not make them permanent? Here’s Vince Ventimiglia (former HHS Assistant Secretary) on that topic (also from the same CMPI event):

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 Tim Franson added:

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.

Considering the success of these two programs, perhaps it’s time to take that political sword and beat it into a public health ploughshare.


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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