A practical toolkit for the identification, selection, and measurement of outcomes including in real-world settings

The overall aim of the Big Data for Better Outcomes (BD4BO) programme is to facilitate the use of ‘big data’ in the development of a more value-based and outcomes-focused healthcare systems Europe.

One of the ways the programme is supporting this objective is through the standardisation of outcomes in different disease areas. Collecting the same outcomes across a range of sources has many advantages including enabling the pooling of outcome data across a wider population. Individual disease-specific projects are developing minimum sets of outcomes (often referred to as core outcome sets (COS)). When agreeing a COS, incorporating a wide range perspectives enables outcomes important to a number of important stakeholders to be collected.

This toolkit has been developed by the Coordination and Support Action of the BD4BO programme, DO>IT, as a practical guide for the individual disease-specific BD4BO projects to support the projects in the identification, selection and measurement of a COS in their disease area.

The toolkit proposes six main stages to develop a COS, from scoping to dissemination, with a focus on stakeholder input across all stages to ensure a wide range of perspectives are taken into account. Whilst the toolkit highlights any existing best practice to developing COS, it importantly also presents a range of methodological options which BD4BO projects can consider depending on the scope of the work and resources available. Each stage includes decision-making flowcharts, summaries of key considerations and presents case studies to highlight the key factors and considerations when developing COS. These typically reflect aspects that are of importance to BD4BO projects around the use of data from a range of sources from ‘real world settings in addition to clinical trials.

The following appendices to the toolkit include original work and case studies informing the toolkit. Some appendices are available at the links below. The work for other appendices are being prepared for publication in peer-reviewed journals; links to these will be provided, as and when they are ready.