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.
Emmanuelle Sabbah-Petrover, Director Complexe Biologics
As Associate Director at Voisin Consulting Life Sciences, Emmanuelle manages projects involving cell, gene & tissue-based medicinal products (“ATMPs”); from non-clinical to clinical phases, both in Europe & the US. Emmanuelle has a particular expertise in GMO requirements for experimental drugs in Europe. Emmanuelle brings 12 years of experience in the biotech industry. Her Ph.D. in science added to R&D experience allows her understanding the constraints & challenges that developers faced daily.
Gopalan Narayanan, Vice President Disruptive Biologics
He provides advice and guidance on the global development and regulatory strategy for ATMPs and complex disruptive biologics based on substantial experience in the field as previous medicines regulator in Europe.
Mark Gauthier, Senior Director Regulatory Science Drugs & Biologics
Mark has a proven history of building and maintaining good relationships with FDA personnel to facilitate development and commercialization of new products (Orphan Products, PDUFA meetings, CBER and CDER Review Divisions including OTAT). He has managed post-approval submissions of all types, including advertising and promotional materials, drug product listings, CMC and labeling supplements.