FDA’s Pazdur and Blumenthal: The March of Checkpoint Inhibitors Will Continue
More approvals of new immune-checkpoint inhibitors, targeted and personalized therapies, as well as the use of real-world evidence are coming in the near future, Richard Pazdur, director of the Office of Hematology and Oncology at the US Food and Drug Administration (FDA) and associate director Gideon Blumenthal wrote in a comment published this week in Nature.
Those expectations follow 2016 oncology approvals for FDA that included five new molecular entities and 17 efficacy supplements, including six accelerated approvals, 17 priority reviews and 11 approvals of breakthrough-designated therapies. FDA also approved five companion diagnostics, including a liquid biopsy test to assist in patient selection. And one new anti-PD-L1 antibody was approved, along with six supplementary approvals of anti-PD-1/PD-L1 antibodies.
“In the coming years, we are likely to see the continued expansion of the therapeutic landscape of immune-checkpoint inhibitors, targeted therapies, and novel companion and complementary diagnostics, including the further development of multiplex genomic testing platforms (including novel tissue-based or blood-based NGS assays),” Blumenthal and Pazdur write. “Short-term and longer-term FDA oncology priorities include the use of ‘real-world evidence’ (RWE) to enable evidence generation outside of clinical trials.”
Such RWE can be generated from multiple sources, they say, including electronic health records, patient registries, mobile health applications and social media.
“Such evidence could be useful for the expansion of labelled indications to patients with rare cancers, potentially enabling a better understanding of patient populations not typically studied in pivotal trials, and of the various dosing and safety issues that arise in clinical practice,” they added. “Other FDA oncology priorities include facilitating a culture of data sharing to ensure that clinical trial data that currently exist in silos can be more broadly used by the biomedical community to rapidly translate basic and clinical observations to improve patient care.”
FDA will also support the expansion of patient eligibility criteria in oncology trials. These developments come as FDA last June created a new Oncology Center of Excellence, which Pazdur will head, to better focus agency resources and better integrate regulatory expertise across FDA review centers.