Comparator isn't necessary. One of the benefits of RMAT is that superiority isn't required. Superiority would be needed for the breakthrough designation. For RMAT, the amount of data for preliminary clinical evidence would vary based on indication. Our RMAT was for an indication with a high placebo response. We originally submitted data for 32 subjects from a randomized, double blinded study and it wasn't enough. After completion of an additional study, we then submitted a data set for about 120 subjects and was granted the RMAT designation. When gathering intel for our RMAT application, we found that many companies are trying to file for RMAT for theoretical applications of their product. Product X is approved for indication Y and the company thinks product X could be used for indication Z, so they apply for RMAT with indication Z. To do so, you have to have controlled clinical data with product X for indication Z. Data for indication Y will not support the RMAT application in this case. Hope this helps.
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Stephen Westover ASQ
Director of Regualtory Affairs
Bloomington IN
United States
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Original Message:
Sent: 18-Feb-2021 11:12
From: Anonymous Member
Subject: RMAT Designation
This message was posted by a user wishing to remain anonymous
With 155 requests submitted to FDA as of Jan 2021, and only 59 granted, I am wondering what people know about what is necessary to meet the FDA standard for preliminary clinical evidence? Other than what is stated in the guidance, which is somewhat vague. What are the issues that caused denial? Not enough follow up? Lack of appropriate comparator? Any thoughts?