PCP3 - Navigating the use of Real-World Evidence in Effectiveness Research and Patient Access Decisions

Abstract

Reimbursement and patient access decisions for new medicines should be guided by relevant and good-quality evidence. Randomised controlled trial (RCT) data are used to support these decisions, however real-world evidence (RWE) could be considered too, especially in cases of accelerated regulatory access where RCT data is less mature, in orphan disease areas where randomised trials are difficult to conduct, or where RCTs do not reflect the patients or clinical practice outside of the trial setting. Stakeholders have different views of the acceptability of using RWE, therefore the GetReal Initiative’s (www.imi-getreal.eu) key aims are to educate and stimulate discussion on the appropriate use of RWE in medicines decision making. The Initiative has developed the "RWE Navigator" (rwe-navigator.eu) which explains key evidence challenges in relative effectiveness research and indicates potential uses for non-randomised evidence, accompanied by overviews of data sources, study designs, quality assessment and bias adjustment of RWE. The navigator also provides links to case studies that demonstrate the potential application of RWE to support regulatory and patient access decisions. It will continue to serve as a gateway to the ongoing work of the GetReal Initiative. The work of the GetReal Initiative indicates that further dialogue is needed to develop policies and perspectives on RWE. The RWE Navigator has been developed to stimulate and inform the debate on the use of RWE in effectiveness research, and it has created a platform for a dialogue between industry and decision-makers. It is a unique source of information on alternative study designs and analytical methods, informed by key decision makers’ views on the acceptability and usefulness of RWE, to inform health technology assessments andpatient access decisions in Asia and elsewhere.