GetReal case studies

The GetReal project aimed to show how robust new methods of real-world evidence (RWE) collection and synthesis could be adopted earlier in pharmaceutical research and development (R&D) and the healthcare decision-making process. To attain this goal, case studies were drawn from the portfolios of EFPIA partner organisations to explore new analytical methods (WP4, WP2, WP1) or study designs (WP2, WP3), or to explore stakeholder views of these methods or study designs (WP1), in specific disease areas.

Some case studies were more historic in nature – to test analytical methods on medicines that have already passed through regulatory and health technology assessment (HTA) that might have been useful earlier on in the process or stakeholder views of these methods (WP1, WP2, WP4). Other case studies included medicines in development that were thought to have a particularly large efficacy-effectiveness gap (see Clarifying the Issues for a definition) or a difference in expectations between regulators and payers (WP2, WP3).
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Drivers of effectiveness in
Hodgkin’s lymphoma

Drivers of effectiveness in diabetes

Patient level drivers of effectiveness in schizophrenia

Healthcare setting-level drivers of effectiveness in schizophrenia

Detecting channeling bias after launch in anticoagulant medicines

Detecting channeling bias after launch in antihypertensive medicines

Detecting channeling bias after launch in diabetes

Early pragmatic trials in chronic obstructive pulmonary disease

Adjusting for drop out from cohort multiple randomised controlled trial in cardiovascular disease

Modelling and simulation of a population enrichment RCT in schizophrenia

Methods for network meta-analysis using individual participant
data in depression

Incorporating non-randomised studies in NMA of RCTs in schizophrenia

Using RWE to connect ‘disconnected’ networks of evidence and inform second-line treatment effects in rheumatoid arthritis

Using RWE to estimate relative effectiveness and inform trial design in multiple sclerosis

Predicting effectiveness based on RCT efficacy data and RWE prior to launch in rheumatoid arthritis

Using RWE to inform relative effectiveness estimates and trial design in metastatic melanoma

Propensity weighting and extrapolation in non-small-cell
lung cancer

Adjusting for confounding bias in a heterogeneous control arm in a pragmatic trial

Comparison of methods for competing risks in pragmatic trials

Controlling for confounding in a pragmatic study with time-to-event outcomes

Adjusting for confounding in early postlaunch settings: going beyond logistic regression models