The lack of generalisability associated with randomised controlled trials (RCT), as well as their increasing complexity, restrictive enrolment criteria, overly controlled study environments and escalating costs, mean that regulators are sometimes forced to accept immature RCT evidence due to high levels of unmet medical need.
As a result, health technology assessment (HTA) bodies have to make uncertain decisions based on imperfect data. Real world evidence (RWE), any data used for decision-making that are not collected in conventional RCTs, which was once thought of as inferior, has been mooted as capable of bridging widening evidentiary voids.
Currently, use of RWE tends to be restricted to areas where RCTs are impractical, such as long term safety data, and hierarchies of evidence still reflect the overarching opinion that RCTs are the gold standard, reliable above all other sources of evidence. Such a focus on this pervasive culture has affected the acceptance of RWE in HTA.
Despite their importance in evidence based medicine, trials can produce an artificial picture of a pharmaceutical product’s value resulting in an ‘efficacy-effectiveness’ gap. Populations used in RCTs do not tend to reflect a typical patient. They also adhere to strict protocols allowing isolation of treatment effects and rely on certain centres of expertise where better outcomes are likely to be achieved. This is in contrast to routine practice where clinicians use their best judgment to make treatment decisions. Furthermore patients are likely to be unwilling to accept randomized treatment if they can secure access to a therapy already in routine practice.
Recently renewed interest in RWE has been fuelled by advances in observational design and standards, increasing pressure on healthcare costs, increasing sophistication of HTA and the increased number of factors required for the evaluation of both clinical and financial value. Observational data is now thought to be able to respond faster and at less of a cost in terms of uncertainties resulting from RCT and provides a novel tool for both hypothesis testing and investigating the usability of treatments in daily practice.
As a result of renewed interest in RWE in the summer of 2016, Roche developed a series of Ad Boards with the explicit aim of seeking expert insight into understanding the European RWE environment. Work from initial Ad Boards showed that experts from Germany, Poland, France, Italy and the United Kingdom thought that RWE would potentially contribute significantly to confirmation of benefit/risk. They also outlined a number of challenges regarding RWE’s acceptance including its generally poor quality, the absence of methodological standards and a lack of transparency.
Whilst the Ad Boards produced a wealth of useful information and knowledge the fact remains that these opinions were gathered from only a small representation of stakeholders in the countries in question. This proposal therefore aims to use the Roche RWE community developed as a result of these Ad Boards to leverage links with key decision makers, beyond the expert groups involved in the Ad Boards, to gain additional insight into the wider use of RWE in 11 European countries.
The overarching objective of this proposal is to improve understanding of the use of RWE in a number of countries in Europe. In general we would like to understand how decision makers in Europe think, beyond the small sample of proxy decision makers involved in the current Ad Boards. The countries of interest include England, Scotland, Germany, France, Italy, Spain, Poland, Portugal, Hungary, Czech Republic and Sweden.
Outputs
November 2017
Authors: Jennifer Gill, Panos Kanavos, Joan Albanell, Bernard Avouac, Magdolna Dank, Robert Duncombe, Antje Fink-Wagner, John Hutton, Karina Jahnz-Różyk, Ingrid Kossler, Katerina Podrazilova, Wolfgang Schramm, Federico Spandonaro, António Vaz Carneiro & Markus Wartenberg
Interest in Real World Evidence (RWE), data not collected via traditional randomised controlled trials (RCT) used in different contexts, is increasing for market-access and reimbursement decision-makers. A global survey was undertaken to understand the use of RWE in these contexts.
View the poster presentation for ISPOR.
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The use of Real World Evidence – Opportunities for Europe
November 2016 - January 2018