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When FDA published the guidance, Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases in August 2024, I read it with great interest. At first, I thought that it was a draft guideline, as this was the first time I had seen a guideline addressing dose selection in the oncology setting. But I quickly realized that this final guidance implements the information gathered in the FDA’s Project Optimus.

In this blog, I will be referencing data in the public domain to compare and contrast the studies submitted to FDA to support label expansions for two marketed products and the similarities and differences in the FDA review and outcomes. By way of disclosure, PROMETRIKA has not worked on either of the two products discussed and all information presented is in the public domain (the October 5, 2023 Oncologic Drugs Advisory Committee (ODAC) FDA slides and ODAC vote for the first and the April 1, 2022 Clinical Review and Evaluation and Statistical Review for the second).

As many of us are well aware, while single arm trials (SATs) historically have been accepted by the US FDA as primary evidence of efficacy for marketing approval in the setting of accelerated approval (AA) of new oncology agents/indications, their acceptance in other therapeutic areas including rare diseases has been more selective.

Draft Guidance - Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics

This guidance issued in March 2023 has been the subject of much talk. While in my opinion, it does not provide any new thoughts on the part of FDA regarding how they handle requests for approval using the accelerated approval (AA) pathway, it does put in writing the policies they have implemented in recent reviews and communications with sponsors.