OBJECTIVES: To study the concordance and disagreement between regulatory and reimbursement agencies’ evidence requirements for clinical decision making across EU5 in acute myeloid leukemia (AML).

METHODS: Regulatory labels from the European Medicines Agency (EMA) and health technology assessment (HTA) reports from G-BA and IQWiG (Germany), HAS (France), NICE and SMC (United Kingdom), HU Virgen de las Nieves and GENESIS (Spain), and CF AVEN – Emilia Romagna (Italy) were reviewed for drugs approved for the treatment of AML to identify the clinical studies.

RESULTS: Eight EMA labels and 29 HTA reports were reviewed. One recently approved drug (gilteritinib) had no available HTA reports. Variation was observed across HTA agencies for the following assessed drugs: decitabine (100%; assessed by all 8 agencies), gemtuzumab ozogamicin (62.5%); midostaurin (62.5%); daunorubicin-cytarabine (50%); arsenic trioxide (37.5%); azacitidine (37.5%); and histamine dihydrochloride (25%). HAS and SMC reviewed all 7 drugs whereas HU Virgen de las Nieves and CF AVEN – Emilia Romagna only reviewed 1 drug. A total of 22 unique studies were identified (from 2 to 5 studies for each drug). Of these, only 59.1% were used by EMA and HTA agencies. However, 18.2% studies were only used by EMA and not by HTA agencies whereas 22.7% studies were only used by HTA agencies but not by EMA. The differences in the considered studies could be attributed to population (pediatric studies) and study designs (non-randomized studies, exploratory dose-finding studies, etc.) considered appropriate by the regulatory and reimbursement agencies. Further, differences in study use for each drug were also observed between HTA agencies.

CONCLUSIONS: Considerable variability was observed across evidence considered by regulatory and reimbursement agencies. Understanding the evidence requirement by regulatory and reimbursement agencies for clinical decision making would help pharmaceutical manufacturers pragmatically decide their evidence-generation strategy and could possibly result in quicker patient access to innovative treatments.