Element card for assessment element D0001 in HTA Core Model Application for Pharmaceuticals (2.0) application

D. Clinical Effectiveness


What is the expected beneficial effect of the intervention on overall mortality?


Mortality is the preferred, objective endpoint for assessments of life- threatening conditions. Overall mortality refers to all-cause mortality. It is expressed either as mortality rates (incidence in given population, at given time point and usually risk standardised), or survival (number of people alive for a given period after an intervention). Several methods are used to adjust mortality rates and survival curves, e.g. relative survival (observed versus expected survival), which can be quite misleading; and hazard ratio (derived from a statistical method comparing the median survivals in the two groups). Note that progression-free survival is not a mortality endpoint; it describes the time from the beginning of an intervention until a patient shows signs of disease progression. Consider separately absolute mortality (compared to placebo or waiting list) and mortality relative to the comparator. See also Methodological guideline for REA of pharmaceuticals: Endpoints used for relative effectiveness assessment of pharmaceuticals, clinical endpoints http://www.eunethta.eu/sites/5026.fedimbo.belgium.be/files/Clinical%20endpoints.pdf

Systematic reviews of trials, trials, both placebo- controlled and with active control. In the absence of head to head trials, studies with indirect comparison (see Methodological guideline for REA of pharmaceuticals: Direct and indirect comparison, http://www.eunethta.eu/sites/5026.fedimbo.belgium.be/files/Direct%20and%20indirect%20comparisons.pdf). If these are not available, non-controlled studies and respective systematic reviews. Health care register data. Modelling studies.

Submission file, SPC, EPARs,




Hochman 2011, Black 2002