Pivotal trial design considerations for new and next generation cell and gene therapies

Résumé fourni par la source

The past decade has seen significant advancements for cell and gene therapies (CGTs). FDA lists more than 30 approved CGTs on their Approved Cellular and Gene Therapy Products website as of August 2024. With the promising treatment effect brought by the currently approved CGTs, there are noticeable limitations, such as, manufacturing delays and durability of response, that encourage continued development in this field. New CGTs can potentially benefit patients by enhancing efficacy or addressing the limitations of currently available therapies. However, the development of new CGTs faces unique challenges as highly efficacious approved first-generation products could be potentially established as the current standard of care (SOC). When the approved CGT products are recommended as the comparator, questions arise, especially, is it feasible to set up a head-to-head comparison between the new and the approved CGT products? What could be the feasible study design options to evaluate the effectiveness of new CGT products? In this article, we introduce the challenges of developing new CGTs in conventional randomized control and single arm trials, as well as discuss some possible strategies for pivotal trials such as hybrid study designs. We believe that more dialogue about this topic among all stakeholders involved in drug development is crucial and hope that this article will contribute towards facilitating such a dialogue.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.