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US FDA’s Marks Issues ‘Provocative’ Call For Target Gene Therapy Profile That Includes Costs

 
• By Sarah Karlin-Smith

CBER Director Peter Marks said recent leaps in gene therapy science have not been matched by more affordable manufacturing technology and that the field needs to set a cost-effectiveness target. 

scientist holds 3d rendering of DNA molecule in hands.
The FDA's Peter Marks wants to lower the cost-effectiveness of gene therapy. • Source: Shutterstock

The US Food and Drug Administration’s top gene therapy regulator issued what he acknowledged is a “provocative” call to consider setting a “target product profile” for gene therapy that includes not just what is expected from a clinical perspective, but “what we need to expect out of them from an economic perspective and from a clinical benefit perspective versus cost perspective.”

Key Takeaways

  • The FDA’s Peter Marks suggested the gene therapy field needs a target product profile that would consider cost and effectiveness so the industry can strive to develop more affordable products.

The FDA, by virtue of legal mandates and historical precedent, tends to avoid discussing the costs of novel medical interventions....

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