Benefits of RAT Designation
RAT designation affords the following benefits to sponsors:
Expedited development and review by FDA (i.e., the same actions as for breakthrough therapy designation);
Early interactions “to discuss any potential surrogate or intermediate endpoint to be used to support the accelerated approval of the product;” and
Eligibility for accelerated approval under current FDA preapproval standards but with new postapproval requirements.
Signaling for Expanded Use of Accelerated Approval
The new postapproval requirements are a key differentiating factor from the breakthrough therapy designation. Sponsors of RAT-designated products approved via the accelerated approval pathway have additional options to meet post-approval requirements beyond the standard, controlled clinical trial. Post-approval requirements[color=red][/color] can be met through:
Clinical evidence, clinical studies, patient registries, or other sources of real world evidence, such as electronic health records;
The collection of larger confirmatory data sets; or
[color=red]Postapproval monitoring of all patients treated with such therapy prior to approval of the therapy.
The new designation, therefore, encourages FDA to further develop and utilize the Subpart H/accelerated approval pathway. For a comprehensive review of FDA’s use of accelerated approval, refer to our paper, here.
Notably, the January 2015 House version of 21st Century Cures at section 2041 addressed regenerative medicine from a different angle. It directed FDA to issue a guidance document on surrogate and intermediate endpoints for accelerated approval of regenerative medicine products. Although this original approach was less ambitious, it also highlighted the need for accelerated approval to bring to market products in the budding field of regenerative medicine.
