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Patent exclusivity provides lucrative market incentives for companies in the biopharma space to innovate. Companies that receive a patent for their drug from the U.S. Patent and Trade Office (USPTO) have a limited monopoly for 20 years from the date of filing the patent. The economic incentives are so lucrative that Amgen built a patent portfolio that resulted in a thirty-seven-year patent term through patent thickets (multiple patents that cover a single drug) for its most profitable drug, Enbrel.

Beyond the standard twenty-year patent exclusivity for each patent relating to the drug, companies may receive a patent extension of 6 months through pediatric exclusivity granted by the FDA. Pediatric exclusivity under the Best Pharmaceuticals for Children Act (BPCA) uses strong market incentives to encourage pharmaceutical companies to provide rigorous clinical data to improve drug label instructions for the pediatric population. Currently, many drugs are used off-label in pediatric patients due to the lack of pediatric data available in medicine. Currently, roughly 50% of medication labels lack guidance for medical providers on a drug’s use in children.

Pediatric data is sparse because drugs must go through multiple trial stages in testing drugs and pediatric trials may occur only after trials reach later stages when evaluation for efficacy, acceptability and adverse effects occur. Moreover, parents and doctors are generally reluctant about involving children in trials because of uncertain treatment effects on children, resulting in a negative feedback loop. With a small sample size, treatment effects are difficult to ascertain with statistical significance. Thus, market incentives may serve as a powerful motivation for pharmaceutical companies to provide rigorous clinical data.

Although granting even more patent exclusivity may seem excessive given the twenty-year patent term, data suggests that the BPCA has generally been successful in meeting its overarching goal of gathering rigorous clinical data. Since 2002, when the BPCA was enacted, a total of 313 drugs received exclusivity determinations from the FDA. Furthermore, under Section 505(a) of the Federal Food, Drug, and Cosmetic Act, written requests for pediatric studies require detailed assessments of pediatric patients including: sample size, age groups, drug information, dosage form, route of administration, drug-specific safety concerns to be monitored or assessed, statistical information, and pediatric labeling that may result from the studies.

In practice, a popular antibiotic, Clindamycin was relabeled to include clinical pharmacology and dosage information for children with obesity in March 2020.  The sample size included 23 participants and additional clindamycin pharmacokinetic study data to develop a population pharmacokinetic model of 220 children. Furthermore, in December 2021, Clindamycin was relabeled again to include safety data for infants with complicated abdominal infections. The sample size included 260 participants. Clindamycin’s relabeling practices with a robust pediatric sample size illustrates BPCA’s success in promoting the accumulation of useful pediatric data to further guidance for medical providers in prescribing drugs to pediatric patients.

Despite pessimism towards extending beyond the twenty-year patent term, which functionally serves as a limited monopoly for pharmaceutical companies, the BPCA has been generally effective in pediatric labeling. Although BPCA research occasionally includes research on adult drugs such as Pomalyst, a treatment for melanoma, with no safety and effectiveness established in pediatric labels, the outlook on pediatric data seems even more encouraging with the enactment of the Pediatric Research Equity Act (PREA) in 2003. PREA gives the FDA the authority to require drug manufacturers to complete studies in children when the drugs are expected to be used in a substantial number of children. In the case of PREA, the pessimism surrounding patent incentives is abated as there is none. Where patent incentives of the BPCA cannot incentivize pharmaceutical companies to provide useful pediatric data for pediatric labeling, PREA may fill the gap in knowledge.

 

Suggested Citation: George Lee, Incentivizing Pharmaceutical Pediatric Data with Patent Extensions, Cornell J.L. & Pub. Pol’y, The Issue Spotter (November 27, 2023), http://jlpp.org/blogzine/incentivizing-pharmaceutical-pediatric-data-with-patent-extensions/.