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Emerging Gene Therapy Concern: A Child Joins A Clinical Trial, But Must Be Followed Into Adulthood

 
• By Derrick Gingery

The long-term follow-up requirements expected for many gene therapies could raise consent and drop-out issues, which could hinder efforts to track safety and efficacy.

gene therapy
Decentralized trial designs could help patient retention in long-term post-approval follow-up for gene therapies. • Source: Shutterstock

Pediatric patients will change a lot in the decade or more after receiving a gene therapy, which has raised concerns about postmarketing tactics to ensure safety and efficacy tracking can continue in the long-term.

US Food and Drug Administration officials are advising sponsors that postmarketing follow-up for gene therapies could last up to 15 years or longer

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