RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:Panc Biomarker Presentation at ASCO GI 18-20 Jan 2024Pazdur's 2022 comments have been superceded by the FDA's move towards biomarkers and surrogate endpoints in early Phase1/2 clinical trials to facilitate quick Accelerated Approvals for orphan and rare diseases, like advanced/metastatic pancreatic cancer (mPDAC), that are followed with confirmatory trials, in contrast to drugs to treat common diseases, which are approved once confirmatory trials are underway.
“We've [got] a better idea over time over the nature of the data that we want to see that can help us feel that there is a compelling case that a given intervention will potentially be associated with a positive clinical outcome,” said Marks, who serves as director of the FDA's Center for Biologics Evaluation and Research, in a July 2023 interview with Fierce Biotech.
His words come amid calls from patient advocates and drug developers, both in the U.S. and globally, to more rapidly expand treatment options, especially as gene therapies become more mainstream. One of the ways that can happen is through accelerated approvals, a regulatory pathway whereby drug developers get earlier approval based on data that's likely to predict clinical benefit for serious conditions. Those data points, called surrogate endpoints or biomarkers, differ depending on the disease, but are easier to accrue than clinical endpoints.
https://www.fiercebiotech.com/biotech/peter-marks-says-fda-now-has-better-sense-rare-disease-biomarkers-will-more-accelerated