Structured benefit--risk assessment for enoxaparin, based on real-world
evidence in the context of its label extension, for the extended
treatment of deep vein thrombosis and pulmonary embolism, and prevention
of its recurrence in patients with active cancer
Abstract
Purpose Guidelines recommend low-molecular-weight heparins (LMWHs) for
patients with cancer associated thrombosis. However, until recently,
only dalteparin and tinzaparin were approved in the European Economic
Area (EEA) for these patients. This study compares the benefit–risk
profile of enoxaparin with dalteparin and tinzaparin, based on
real-world evidence (RWE) for the extended treatment of deep vein
thrombosis (DVT) and pulmonary embolism (PE), and prevention of
recurrence in adult patients with active cancer. Methods A
semi-quantitative structured benefit–risk assessment was conducted for
the label-extension application of enoxaparin employing the following
steps of the benefit–risk action team descriptive framework: define
decision context; determine key benefit and risk outcomes; identify data
sources; extract data; interpret results. Results The key benefits were
defined as reduced all-cause mortality and VTE recurrence (including
symptomatic DVT, fatal PE and non-fatal PE); the key risks were major
and non-major bleeding of clinical significance. When compared with
other EEA-approved LMWHs (dalteparin, tinzaparin), enoxaparin
demonstrated a trend toward favourable effects for the reduction of VTE
recurrence and all-cause mortality. There was a trend in favour of other
LMWHs for major bleeding, and a trend in favour of enoxaparin for
non-major bleeding with no evidence of significant difference between
enoxaparin and the comparator groups. Conclusions The assessment using
RWE demonstrated a favourable benefit–risk profile for enoxaparin
similar to that of other EEA-approved LMWHs for the treatment of DVT and
PE and the prevention of recurrence in patients with active cancer and
thus supported the label-extension approval.