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 medical 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
- FDA’s Peter Marks suggests the gene therapy field needs a target product profile that would consider both cost and effectiveness so it can strive to develop more affordable products.
FDA, by virtue of legal mandates and historical precedent, tends to shy away from discussing the costs of novel medical...
