Unraveling innovation potential in the real-world setting: eighteen novel agents with twenty-six approved European indications, in the management of leukemias, lymphomas, and multiple myeloma

Introduction: Real-world effectiveness of hemato-oncology pharmaceuticals may not necessarily mimic clinical trial efficacy results, mainly due to demographic and clinical practice variability. The aim of this review was to systematically assess the availability of real-world evidence (RWE) and the transferability of clinical trial (CT) efficacy results to real life, for novel agents recently approved to manage lymphomas, leukemias, and multiple myeloma. This is the largest cross-indication review comparing RWE to CT results, aspiring to inform clinical practice and decision-making when funding hemato-oncology pharmaceuticals.

Areas covered: The review methodology focused on identifying all novel agents that entered EU landscape between 2012 and 2016 by using European Medicines Agency (EMA) database, while conducting a systematic PubMed literature review of RWE in the specific hematological malignancies, in order to compare RWE versus CT efficacy endpoints.

Expert opinion: In total, 18 international nonproprietary names (INNs) that received EMA approval for any indication were included and the registrational efficacy results are presented. Eight (44%) INNs proved to have relevant RWE generated in at least one approved indication. The analysis of findings revealed high variability in terms of RWE availability and transferability of CT results to relevant real-life experience among the disease areas investigated.