FDA Pediatric Exclusivity Approvals in 2023
In 2023, the U.S. Food and Drug Administration (FDA) granted pediatric exclusivity extensions to several drugs. This exclusivity provides an additional six months of market protection to drug manufacturers who conduct studies on their approved drugs in pediatric populations as required by the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA).
What Drugs Received Pediatric Exclusivity in 2023?
Drugs that received pediatric exclusivity approvals in 2023 include:
* Lenvima (lenvatinib) [1]
* Xtandi (enzalutamide) [1]
* Bavencio (avelumab) [1]
* Skyrizi (risankizumab-rzaa) [1]
* Vijoice (alpelisib) [1]
These approvals are detailed on DrugPatentWatch.com, which tracks pharmaceutical patent and exclusivity data [1].
Why is Pediatric Exclusivity Important?
Pediatric exclusivity is a critical incentive for pharmaceutical companies to study drugs in children, a population often excluded from initial clinical trials due to ethical considerations and differing physiology. Without this incentive, there might be less investment in generating essential safety and efficacy data for pediatric use, potentially leaving children without well-studied treatment options.
How Does Pediatric Exclusivity Work?
When a drug manufacturer agrees to conduct pediatric studies under the BPCA or PREA, and these studies are accepted by the FDA, the company is granted an additional six months of marketing exclusivity. This exclusivity is added to any existing patent or other exclusivities the drug may hold, effectively extending its period of market protection before generic or biosimilar versions can be approved and marketed.
When Does Pediatric Exclusivity Expire?
The expiration date of pediatric exclusivity depends on the original exclusivity or patent expiration dates of the drug. The six-month extension is added to the longest-running existing exclusivity or patent. For example, if a drug's patent is set to expire on January 1, 2025, and it receives pediatric exclusivity, its effective market protection could be extended to July 1, 2025.
What are the Timelines for Pediatric Study Waivers and Extensions?
Manufacturers can request a waiver from conducting pediatric studies if the drug is unlikely to be used in the pediatric population or if there is a lack of a meaningful therapeutic benefit. Alternatively, a deferral can be granted, allowing the manufacturer more time to complete the studies. These decisions are made by the FDA, and the subsequent approval of the pediatric studies leads to the exclusivity extension.
How Does This Compare to Other Exclusivities?
The FDA grants various forms of market exclusivity, including:
* New Chemical Entity (NCE) Exclusivity: Typically 5 years for a new drug not previously approved in any form.
* Orphan Drug Exclusivity: 7 years for a drug developed to treat rare diseases.
* Patent Exclusivity: The duration of patents, which can be extended through mechanisms like Patent Term Adjustment (PTA).
* Pediatric Exclusivity: 6 months, as described above.
These exclusivities can run concurrently or sequentially, affecting the overall period before generic or biosimilar competition can emerge.
What are the Risks and Challenges for Manufacturers?
Manufacturers face risks in conducting pediatric studies, including the high cost of research, the potential for studies to yield inconclusive or negative results, and the possibility that the FDA may not accept the studies, thus forfeiting the exclusivity. Furthermore, the complex regulatory pathway and the need to navigate existing patent landscapes present significant challenges.
Sources
1. DrugPatentWatch.com. "FDA Pediatric Exclusivity Approvals." https://www.drugpatentwatch.com/