Study finds ‘substantial revenues’ for cancer drugs in granted pediatric exclusivity

Cancer drugs in a pediatric clinical trial network granted pediatric exclusivity cost between $20 million and $75 million to develop and generated revenue between $259 million and $454 million, according to a recent research letter published in JAMA Pediatrics.

“This cohort study of 4 cancer drugs granted pediatric exclusivity based on trials conducted with pediatric clinical trial networks found that exclusivity may generate substantial revenues,” Ameet Sarpatwari of the Program on Regulation, Therapeutics, And Law (PORTAL) at Brigham and Women’s Hospital and Harvard Medical School in Boston, and colleagues wrote.

Under the Best Pharmaceuticals for Children Act, manufacturers that complete pediatric trials requested by the US Food and Drug Administration (FDA) are granted 6 months of additional exclusivity. A recent draft guidance released by FDA notes that the agency intends to stop granting these extensions for written request (WR) studies under the Pediatric Research Equity Act, an issue that most stakeholders responding to the draft guidance said would reduce the number of pediatric studies conducted and the number of WRs sought for pediatric indications (RELATED: Commenters push back on FDA’s plans for PREA compliance, pediatric exclusivity, Regulatory Focus 19 July 2023).

Sarpatwari and colleagues used data from the Children’s Oncology Group (COG) to analyze the value of conducting pediatric trials and the revenue generated from a drug being granted pediatric exclusivity. There were 254 drugs were granted pediatric exclusivity between 2002 and 2023, and 42 (17%) were cancer drugs including 9 associated with COG trials. Of these, COG had complete data available for four cancer drugs (sunitinib, dasatinib, eribulin, and ruxolitinib) that received pediatric exclusivity between January 2010 and June 2023. Data were available for 12 pediatric trials for the four drugs, including five trials for dasatinib, three trials for eribulin, two trials for ruxolitinib, and two trials for sunitinib. COG conducted 9 of 12 trials (95%) in the analysis.

The researchers evaluated the trials leading to pediatric exclusivity for each drug, payments made to execute trials led by COG, and costs associated with trials requested by FDA. Cost of investment was estimated using a 10% cost of capital, and in sensitivity analyses, the researchers used cost of capital estimates of 5% and 15%. Pediatric exclusivity revenue estimates were determined by identifying an entry date for a generic drug or an expected entry date after the exclusivity window, and revenues from exclusivity were assessed with a 55% market-share erosion rate, with market-share erosion rates estimated at 40% and 70% in sensitivity analyses.

Overall, manufacturers invested $156 million into pediatric trials, for a mean $13 million investment per trial and $39 million invested per pediatric exclusivity status. The total revenue across all cancer drugs granted pediatric exclusivity was $1,236 million, or a mean $309 million per cancer drug given exclusivity. Revenue generated by cancer drugs ranged from $42 million in the case of eribulin to as high as $741 million in the case of ruxolitinib, researchers said. When conducting the sensitivity analyses, the researchers noted total cost of investment per cancer drug granted exclusivity had a range of between $20 million and $75 million, and a range between $259 million and $454 million.

Sarpatwari and colleagues said their study was limited by relying on COG for estimating trial costs, projected revenue, and market-share erosion. The study also focuses on cancer drugs and may not be generalizable to other types of drugs, they noted.

“Nonetheless, this study estimates the potential value of data generated by pediatric clinical trial networks, which could be leveraged to support further advances in pediatric care,” they said. “This finding highlights an opportunity to ensure that the value of pediatric exclusivity to manufacturers is commensurate with benefits to pediatric research programs. For example, manufacturers could invest a portion of returns in pediatric research enterprises, which have historically been underfunded.”

JAMA Pediatrics Sarpatwari et al

Study finds ‘substantial revenues’ for cancer drugs in granted pediatric exclusivity

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