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FDA Leadership Changes Tune On Pediatric Priority Review Voucher Benefit

 
• By Sarah Karlin-Smith

As the reauthorization deadline nears, CDER and CBER leaders stressed that sunsetting the program would hurt pediatric rare disease development, while endorsing a new Democrat-proposed stick for rare disease research. 

older female doctor talks to young child and parent
FDA leaders have a new message on the value of the rare pediatric PRV program with the reauthorization deadline nearing. • Source: Shutterstock

US Food and Drug Administration leadership recently stressed the importance of the rare pediatric disease priority review voucher program, a notable shift for the agency, which once said the program had adversely impacted its ability to set public health priorities and manage its workload, just as the program heads toward its reauthorization deadline.

Key Takeaways

  • The CDER and CBER directors said the rare pediatric disease priority review voucher is an important incentive, a notable shift for an agency that once wanted to scrap the program.

  • FDA leaders also pushed for removing the orphan drug exemption from the Pediatric Research Equity Act to spur more pediatric rare drug development

Center for Biologics Evaluation and Research Director Peter Marks and Center for Drug Evaluation and Research Director Patrizia Cavazzoni stressed that they were not lobbying, but conveying the feedback they have received on the potential reauthorization when they spoke at the 16 July

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