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Will Newly Proposed FDA Guidance Discourage Pediatric Drug Development?

The U.S. Food and Drug Administration could soon become more selective about which companies receive six months of patent exclusivity in exchange for developing pediatric drugs. If finalized, the guidance could have the unintended consequence of discouraging investment in drugs for infants and children. 

The Role of Patent Exclusivity in Pediatric Drug Development 

Manufacturers currently are required under the Pediatric Research Equity Act to conduct pediatric studies to receive FDA approval for certain drugs. Those same studies can also make them eligible for six months of patent exclusivity, which they can apply to any drug they manufacture.  

This approach incentivizes companies to develop and invest in drugs for infants and children, which are generally difficult to develop and therefore rare.  

For decades, physicians often had to treat infants and children off label, adjusting the dosage of medications studied and approved for adults rather than for children. Policy reforms, including offering patent exclusivity, have helped to turn the tide, dramatically increasing the number of clinical studies dedicated to pediatrics. As a result, more medications are being developed and approved specifically for infants and children, and new pediatric labels are being added to existing drugs. 

Proposed Changes 

New guidance proposed by the Food and Drug Administration, however, would change the rules on exclusivity and pediatric drug development.   

Specifically, it would ask manufacturers to conduct additional studies, beyond those already required by the Pediatric Research Equity Act, if they want to obtain exclusivity. The draft guidance urges manufacturers to take a broader approach by conducting additional studies on children of varying ages. It also directs manufacturers to test multiple indications before sending the drug through the FDA approval process.  

Impact on Pediatric Drug Development 

The FDA’s guidance is well intended. No doubt, generating more research on pediatric drugs would build a body of knowledge that benefits clinicians and patients alike. But raising the bar could also impact whether companies attempt to develop much-needed drugs for the pediatric population. Additional research demands additional time, manpower and investment. Not all manufacturers will be ready or able to dedicate these resources. 

Most physicians and parents would argue that they need more, not fewer, medications developed and approved specifically for infants and children. By making exclusivity, which is the driving incentive behind pediatric drug development, harder to obtain, the FDA may unintentionally encourage manufacturers to focus their attention and research dollars elsewhere.  

This could leave young patients and their health care providers behind.  

The proposed guidance is open to public comment through July 17, after which it will be finalized by the Food and Drug Administration. 

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