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 Journal Article
@article{Petrakis2019-uw,
title = {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},
author = {Ioannis Petrakis and Christos Kontogiorgis and Evangelia Nena and Kostas Athanasakis and Vasiliki Gougoula and Ioannis Kotsianidis and Theodoros C Constantinidis},
year = {2019},
date = {2019-09-01},
journal = {Expert Rev Hematol},
volume = {12},
number = {12},
pages = {1063--1075},
address = {England},
abstract = {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.},
keywords = {Hematological malignancies; leukemia; lymphoma; myeloma; real-world evidence},
pubstate = {published},
tppubtype = {article}
}
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.
