Super-speedy cancer drug reviews: FDA unveils new draft guidance to help sponsors apply
How does a cancer drug cross the FDA finish line 3-5 months before its PDUFA date? That’s where the Real-Time Oncology Review comes in.
For the last five years, FDA’s Oncology Center of Excellence under Rick Pazdur has been quietly tapped into RTOR to allow sponsors to provide even earlier, segmented submissions of critical efficacy and safety data, thereby enabling these faster evaluations of applications.
On Friday, OCE provided its first draft guidance on the RTOR program, explaining how sponsors can apply, what to submit if accepted, and how RTOR is different from rolling reviews, in that rolling reviews require complete sections (e.g., the complete clinical module) to be submitted prior to a complete application submission.
“At the time top-line results of a pivotal trial(s) are available and the database has been locked, an applicant may apply for review under RTOR by submitting a request via email,” the agency explains. “The applicant should include their top-line results and a written justification explaining how their application demonstrates that it is appropriate.”
Generally, about 20 business days later, the FDA review division will decide if an application can be accepted.
While many of the drugs that have gone through RTOR so far have shaved significant time off their PDUFA dates, the FDA also makes clear in Friday’s draft, that “this may not be feasible for all applications due to specific issues that may be identified with the application or overall workload considerations.”
Generic Drugmakers and CRLs
Another hot-button issue for the agency is the fact that generic drugs often go through multiple cycles of reviews before winning approval.
To help combat these long cycles of CRLs and re-submissions, the agency on Friday released final guidance explaining how if an ANDA applicant fails to fully address all deficiencies identified in a CRL, “within 1 year after issuance of the CRL (or take either of the other two actions prescribed by regulation and that are described above), FDA may consider this failure to be a request by the applicant to withdraw the ANDA.”
As far as changes from the draft issued on September 29, 2020, the agency said:
Changes from the draft to the final guidance include adding an appendix that provides [nine] examples of factors that FDA could consider as the basis for concluding that an applicant’s request for an extension of time to respond to a CRL is reasonable. In addition, editorial changes were made to improve clarity.
Remote Regulatory Assessments Outside the Pandemic
The agency on Friday also issued a new Q&A-based draft guidance on remote regulatory assessments, which have been an important resource for the agency during the pandemic, and will continue to be of use after the pandemic.
While the agency explains how the FDA does not consider an RRA to satisfy the legal requirements of what an inspection is (i.e. in-person assessment of a facility), it says they are still valuable, particularly as FDA has used them to help with approval decisions, verify corrective actions for establishments with an acceptable compliance status, and gain compliance insight into establishments that FDA has been unable to inspect.
“The Agency believes that FDA’s use of both voluntary and mandatory RRAs, as applicable, for all types of FDA-regulated products is in the interest of the public health, and the Agency is issuing this guidance to provide further transparency to stakeholders about the circumstances in which RRAs may be used both during and beyond the COVID-19 pandemic and promote greater consistency in the way RRAs are conducted,” the draft says.