Do the Rewards of Pediatric Exclusivity Outweigh the Benefits? Researchers Discuss

Regulatory NewsRegulatory News | 26 September 2018 |  By 

The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) have increased the number of pediatric studies conducted, though the costs of running the studies are dwarfed by the rewards from additional exclusivity, a new study in JAMA Internal Medicine found.

The researchers identified 54 drugs that received pediatric exclusivity extensions between 2007 and 2012 and for which there were 141 trials enrolling 20,240 pediatric patients, costing the companies $4.9 billion in 2017 dollars, with a median of $36.4 million per trial.

Of those trials, the researchers from the Program on Regulation, Therapeutics, and Law (PORTAL), Brigham and Women’s Hospital and UC-Berkeley found that 31 drugs (57%) demonstrated safety and efficacy for children in at least one study, while 29 (54%) extended the age range for an existing adult indication and three (6%) obtained a new clinical indication.

But the pediatric exclusivity provided pharmaceutical manufacturers with a median net return of $176 million and a median ratio of net return to cost of investment of 680%.

Overall, the researchers said that the pediatric exclusivity extension “has generated new pediatric indications for several drugs, but it has also led to substantial rewards to pharmaceutical manufacturers. Over the long term, direct federal funding of pediatric research may be less expensive for consumers and have similar—or greater—public health benefit.”

And should policymakers determine that the costs to consumers for pediatric exclusivity extensions are excessive, the researchers say that an alternative would be to “set a fixed or predetermined award amount for each requested study, claimable on successful completion of pediatric studies.

“Such an approach would not require companies to wait several years to recoup capital invested in pediatric research, and it would be less expensive for the public, particularly for products with substantial revenues, in which the extension of the monopoly creates the largest mismatch between the incentive and the cost,” they wrote

Labeling Changes and Costs for Clinical Trials Performed Under the US Food and Drug Administration Pediatric Exclusivity Extension, 2007 to 2012


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