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Expedited access of cellular and gene therapy products: The EU versus the US

Post thumbnail Expedited access of cellular and gene therapy products: The EU versus the US

There is some variation in the eligibility criteria and features of breakthrough therapy designation (BTD), regenerative medicine advanced therapy (RMAT) designation, and the PRIority MEdicines (PRIME) scheme, as will be explained in this review. The expectations that the FDA and the EMA have for RMAT and PRIME, respectively, are distinct because some products were granted the designation/scheme by either the FDA but not the EMA, or vice versa. Therefore, denial of an RMAT or PRIME status should not preclude a request of the “counterpart” from the other agency. Although RMAT designation has only been available as a mechanism to expedite drug development and approval for two years (authorized by the US Congress in December 2016, implemented early 2017), it shows great promise in increasing sponsor access to the FDA review division. There are not sufficient data available yet to confirm this statement, as none of the cell and gene therapies already authorized by the FDA had an RMAT designation, but some products are approaching the stage of development from which a biologic license application (BLA) will soon emerge. Recently, the FDA introduced the initiation of a new programme called INitial Targeted Engagement for Regulatory Advice on CBER ProducTs (INTERACT). INTERACT meetings replace the former pre-pre-investigational new drug application (pre-pre-IND) meetings and they can no longer be requested. The INTERACT programme will also provide sponsors who are developing cellular and gene therapy (CGT) products much earlier access to the FDA for assistance in designing nonclinical safety programmes, taking a practical approach that allows sparing of animals and early collaboration on the clinical strategy to streamline development, much like the pre-pre-IND meetings.


Published on: February, 2019
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    publication by

    Emmanuelle Sabbah-Petrover
    Emmanuelle Sabbah-Petrover, PhD
    Director, Complex Biologics
    As Director at Voisin Consulting Life Sciences, Emmanuelle manages projects involving cellular and gene-based medicinal products; from early stages of…
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    Mark Gauthier
    Mark Gauthier
    Senior Director, Regulatory Science, Drugs & Biologics
    As Senior Director, Regulatory Science, Mark is responsible for assisting clients to define the regulatory strategies for the global development and…
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