FDA Revises Guidance on Oncology Endpoints

Regulatory NewsRegulatory News
| 19 December 2018 | By Zachary Brennan 

Replacing a guidance from 2007, this latest guidance from the US Food and Drug Administration (FDA) clarifies how certain oncology endpoints can serve different purposes based on the context of use, and it provides the agency’s current thinking on factors considered in making these determinations on which endpoints are used in what context.

The new updates made to this guidance also expand on the information with respect to oncology endpoints and provide updated resources, references and examples of regulatory approvals.

Other updates include the addition of examples of emerging oncology endpoints and the addition of intermediate clinical endpoints in the discussion of accelerated approval.

“We continue to encourage companies to engage with FDA early in the drug development process so that we can work with them to apply modern and efficient trial designs, as well as explore novel endpoints, such as minimal residual disease, which recently formed the basis of approval for a new treatment for a type of leukemia,” FDA Commissioner Scott Gottlieb said.


In addition to discussing the statutory and regulatory requirements for effectiveness, the 19-page guidance discusses endpoints based on tumor assessments, endpoints involving symptom assessment, blood or body fluid-based biomarkers and emerging endpoints.

Under the header of clinical trial design and analysis considerations, the guidance also touches on single-arm studies, randomized studies designed to demonstrate noninferiority, trial designs for radiotherapy protectants and chemotherapy protectants and trial design and analysis issues.

“Over time, larger improvements in tumor reduction and delay in tumor growth have been seen, and tumor measurement endpoints have been used to support both traditional and accelerated approval. Improvement in disease-free survival (DFS) has supported drug approval in selected adjuvant settings, in which a large proportion of patients were expected to have cancer symptoms at the time of recurrence,” the guidance says.

“Durable complete response has supported traditional approval in leukemia, where complete response (CR) is associated with less infection, bleeding, and blood product support,” FDA adds. “A large improvement in progression-free survival (PFS) or high, substantiated durable ORR has been used to support traditional approval in select malignancies, but magnitude of effect, relief of tumor-related symptoms, and drug toxicity should also be considered when making the approval decision.”

 Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics Guidance for Industry


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