Entering the RAT race? FDA issues instructions for new cell therapy designation

By Dan Stanton

- Last updated on GMT

Image: iStock/lzf
Image: iStock/lzf

Related tags Food and drug administration Medicine Fda

Drugmakers have been invited to submit preliminary clinical evidence for cell and gene therapies under the new US FDA Regenerative Advanced Therapy designation

The 21st Century Cures Act, signed into US law in December​ by former President Obama, introduced the Regenerative Advanced Therapy (RAT) designation offering personalised medicine makers an “efficient development program for, and expedite review of, [their] drug if the drug qualifies as a regenerative advanced therapy.”

And last week the US Food and Drug Administration (FDA) gave industry instructions as to how it can apply for the new designation either concurrently with submission of an Investigational New Drug application (IND) or as an amendment to an existing IND.

“We generally do not expect you to submit primary data (data sets), but your request for designation as a RAT should describe the preliminary clinical evidence,”​ the Agency said.

“Please include a brief description of any available therapies for the disease or condition, the study design, the population studied, and the endpoint(s) used; and a description of the study results and statistical analyses (e.g., subgroup analyses).”

The FDA added it would not grant a RAT designation if an IND is on hold or is placed on hold during the RAT designation review.


According to Section 3033​ of the 21st Century Cures Act, a product is eligible for RAT designation if:

  1. The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations;
  2. The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
  3. Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition

The FDA also referred drugmakers to its May 2014 guidance on Expedited Programs for Serious Conditions – Drugs and Biologics​ as to its nterpretation of whether a disease or condition is serious or life-threatening and whether a drug is intended to treat a serious disease or condition.

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