No (if not please add comments): Not applicable No: major changes needed, please comment (re-review required) Executive Summary The IMI GetReal consortium has drawn up definitions of key terms, both for the purpose of GetReal, and also with the aim of providing clarity to external stakeholders around these terms. In order to explore the origin and context of some of these terms, the glossary is preceded by an overview of real world data and the origin of the key concepts associated with the term. In addition, this document explores the definitions of both and and attempts to define how ‘efficacy’ ‘effectiveness’ efficacy studies can be distinguished from effectiveness (pragmatic) studies. Deliverable content Defining real world data, and where it comes from Healthcare funding decisions increasingly rely on evidence beyond that which is collected in randomised controlled trials (RCTs), which are required by regulatory authorities for marketing authorisation. There are many reasons for this, including the need to understand health outcomes in routine clinical practice, which it is recognised may differ from those observed under the idealised conditions of a RCTs (for more on this, refer to the section versus efficacy’). This has ‘Effectiveness long been referred to as the collection of real world data, without consensus on the definition of real world data, or what constitutes a real world study. The evolution of this terminology is probably due to the continual and rapid development of IT infrastructures and capabilities; increasing availability
No (if not please add comments): Not applicable No: major changes needed, please comment (re-review required) Executive Summary The IMI GetReal consortium has drawn up definitions of key terms, both for the purpose of GetReal, and also with the aim of providing clarity to external stakeholders around these terms. In order to explore the origin and context of some of these terms, the glossary is preceded by an overview of real world data and the origin of the key concepts associated with the term. In addition, this document explores the definitions of both ‘efficacy’ and ‘effectiveness’ and attempts to define how efficacy studies can be distinguished from effectiveness (pragmatic) studies. Deliverable content Defining real world data, and where it comes from Healthcare funding decisions increasingly rely on evidence beyond that which is collected in randomised controlled trials (RCTs), which are required by regulatory authorities for marketing authorisation. There are many reasons for this, including the need to understand health outcomes in routine clinical practice, which it is recognised may differ from those observed under the idealised conditions of a RCTs (for more on this, refer to the section ‘Effectiveness versus efficacy’). This has long been referred to as the collection of real world data, without consensus on the definition of real world data, or what constitutes a real world study. The evolution of this terminology is probably due to the continual and rapid development of IT infrastructures and capabilities; increasing availability
D1.3%20GetReal%20Glossary.docx