Beyond Phase I: Expansion Cohort Trials Bring Efficiencies, Regulatory Challenges
First-in-human, expansion cohort trials are on the rise in oncology, but protocols for such studies can be highly problematic, lacking clear objectives, well-defined populations and independent oversight.
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“One Continuous Trial” In Oncology: US FDA Offers Guidance And Encouragement
One of the hallmarks of the “breakthrough” era has been the rapid expansion of first-in-human trials into pivotal studies for efficacy submissions. In a new draft guidance, FDA offers some initial thoughts on safeguards to make hyperfast development pathways successful.
At Tofersen Panel Review, US FDA Shined A Light On Internal Differences Of Opinion
Agency’s presentations on Biogen/Ionis’ ALS drug included differing viewpoints from the statistical and clinical pharmacology teams; the transparency and emphasis on a multidisciplinary review approach may reflect lessons learned from missteps at the Aduhelm panel review in 2020.
BrainStorm’s ALS Treatment NurOwn, Filed Over Protest, Will Get US FDA Panel Review
A November 2022 refuse-to-file letter cited clinical and statistical issues for the stem cell therapy, which failed its Phase III clinical efficacy endpoint. However, BrainStorm has consistently asserted that NurOwn demonstrated benefit in patients with less advanced disease at baseline, and it is encouraged by regulatory flexibility FDA recently has shown for the neurodegenerative disease.