So our Citizen Petition has been submitted to the FDA on need and rationale for
supplementary comparison of Quality of Life-related patient reported outcome (QoL-PROs)* for surrogate endpoints based on tumor assessments.
full text: http://www.lymphomation.org/Citizen%20Petition-QoL-feb2022-fin.pdf
We waited for Congress to approve a commissioner and avoided advancing methods (thanks to guidance provide here) for capturing, comparing, or reporting QoL-PROs.
Opening statement:
To amend the Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics Guidance for the Industry to require or strongly urge supplementary comparison of Quality of Life-related patient reported outcome (QoL-PROs) for the following surrogate endpoints in randomized controlled clinical trials:*
Endpoints Based on Tumor Assessments
Disease-Free Survival (and Event-Free Survival) | Objective Response Rate
Complete Response | Time to Progression and Progression-Free Survival (PFS) | Time to Treatment Failure
What I hope to see is a universal set of QoL-PRO in key domains applied across all studies and reported in a way that promotes physician and patient understanding.
We anticipate this change in industry registration trials would provide a good many benefits (see text of petition) and would be an aid to regulatory decision making in close calls:
3 scenarios:
Major improvement in time to relapse with modest impairment in QoL.
(Approval could still be justified)
Modest improvement in time to relapse with impairment of QoL
(Longer followup justified)
Modest improvement in tumor response with improvement in QoL
(Approval could still be justified)
Suggestions on models, instruments, analysis, and reporting in plain language most welcome!