This study of all FDA approvals granted Breakthrough Therapy designation from 2012 through 2017 suggests that pivotal trials supporting these approvals commonly lacked randomization, double-blinding, and control groups, used surrogate markers as primary end points, and enrolled small numbers of patients...
Additionally, blood-based biomarker detection can potentially be used to monitor disease progression5 and therapeutic efficacy6, as well as surrogate endpoints7, thus streamlining the translation of research advancements to real-world applications. Blood-based biomarker driven cl...
Using preliminary evidence, rather than a full series of clinical trials, it’s possible that, in many cases, the supporting evidence for the product would be based on surrogate endpoints used to determine clinical efficacy (Accelerated Approval). This would require post...