CER Daily Newsfeed

Integrating Trials Into Clinical Practice: US FDA Guidance Outlines Limited Use Cases

(9/18, Sarah Karlin-Smith, Pink Sheet) reports “...Outcome assessments like clinical-reported outcomes and patient-reported outcomes may be less appropriate for use in integrated trials as these evaluations often require research-specific instruments and timing of assessments that may be more challenging to integrate into clinical practice. However, the guidance notes some ways that these outcomes might be made more feasible for an integrated trial, such as by using clinical outcomes assessments that are most consistent with information collected in the context of a routine clinical practice visit.” Subscription Required

EMA Finalizes Their Perspective of the Use of Single-Arm Trials for Regulatory Decision-Making

(9/19, Joanne Walker, The Evidence Base) reports “...The paper provides detailed guidance on when and how SATs can be used to support regulatory decisions in situations where RCTs may not be feasible. The EMA places the onus on manufacturers to justify the use of SATs instead of ‘gold standard’ RCTs, requiring them to demonstrate why a SAT is appropriate to provide robust evidence in the given clinical context.” Full

UK: Daiichi Sankyo’s Vanflyta Recommended by NICE for Acute Myeloid Leukaemia

(9/19, Emily Kimber, PMLiVE) reports “Daiichi Sankyo’s Vanflyta (quizartinib) has been recommended by the National Institute for Health and Care Excellence for certain cases of acute myeloid leukaemia (AML). The drug has been specifically recommended for routine NHS commissioning in England and Wales as an induction, consolidation and maintenance therapy for newly diagnosed FLT3-ITD-positive disease.” Full