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Considerations driving the choice in clinical trial design of cell and gene therapy products: weighing convenience versus necessity

5 Jun 2025

Abstract excerpt

Because of small patient populations, lack of effective therapies, and complicated treatment protocols, advanced therapy medicinal products (ATMPs) for rare diseases are often licensed based on evidence generated outside of a "gold standard" double-blinded, randomized controlled trial (RCT). The lack of important bias-reducing features in non-RCTs can present uncertainties for regulators and health technology...

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PMID: 40542809
DOI: 10.1016/j.jcyt.2025.06.001

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