Summary

Institutions across Europe house rich databases with detailed personal and biological information. If linked, this ‘big data’ has the potential to deliver insights that will allow healthcare systems to ensure that the patient is truly at the centre of decision-making, with health outcomes that matter and resources focused on treatments that really make a difference. IMI’s Big Data for Better Outcomes (BD4BO) programme is generating knowledge, data and methodologies needed to support the transition towards more outcomes-focused, sustainable healthcare systems in Europe. Through its projects, the programme focuses on a number of key therapeutic areas, namely Alzheimer’s disease, heart disease, and certain cancers.

Big data and outcomes: opportunities and challenges

Currently, decisions on treatments and pricing are based largely on data from clinical trials. This data is inevitably limited in scope and does not fully reflect the situation of all patients with a given disease. Yet immense amounts of data are generated daily by researchers as well as clinicians and patients themselves. If we could harness this ‘big data’, it could revolutionise research and healthcare and help us move towards more sustainable healthcare systems focused on outcomes for patients. However, delivering ‘big data for better outcomes’ is far from easy. On the big data side, bringing diverse data sources and formats together, linking them up and analysing them is far from easy due to technical, legal and ethical issues. On the outcomes side, identifying and defining outcomes for patients that are meaningful, measurable and relevant for all stakeholders in healthcare is also a challenge.

Introducing BD4BO

IMI’s Big Data for Better Outcomes (BD4BO) programme aims to address these challenges. It brings together all stakeholders, including patients, academic researchers, healthcare policy makers, regulators, healthcare providers, payers, and the pharmaceutical industry. Overall coordination of the programme is the responsibility of the DO>IT project, a coordination and support action set up for this task.

Leading the work on data integration is the European Health Data Network, a project which is under development and is scheduled for launch in the second half of 2018.

Meanwhile, a series of projects focused on specific therapeutic areas will put the concept of ‘big data for better outcomes’ into practice in the fields of cardiovascular disease, haematological malignancies (blood cancers), and Alzheimer’s disease. A fourth therapeutic area project, on prostate cancer, is under development and should start in the first half of 2018.

Expected impacts

By making it easier to tap into ‘big data’, BD4BO will make research more efficient, as researchers will be able to re-use existing data instead of having to generate new data. The sheer volume of data involved will make it easier for researchers to uncover new insights into diseases and treatments, and accelerate the development of innovative, more effective medicines.

For healthcare systems, the tools and resources created by BD4BO, as well as the networks of experts in the projects, will make it easier to identify of which treatments work best (and which do not) for different groups of patients.

For patients, BD4BO will help to ensure that treatments are designed and selected on the basis of outcomes that matter to patients. It will also increase the likelihood of patients receiving a treatment that works for them.

For the pharmaceutical industry, BD4BO will supply greater knowledge of how patients experience their condition, and which outcomes really matter, allowing them to better target potential treatments and demonstrate efficacy in real-life conditions.

Ultimately, BD4BO will contribute to the evolution of healthcare systems, making them more sustainable and crucially, more focused on outcomes for patients.

Projects

DO>IT

The DO>IT project was launched to provide a coordination platform for the BD4BO programme, exploiting synergies across the projects and maximising its impact on healthcare systems. Specially, DO>IT will aggregate learnings and disseminate findings from the projects; develop minimum data privacy standards; engage with key stakeholders; and recommend areas for future collaborative research.

ROADMAP

Alzheimer’s disease is on the rise in our ageing population, and new, effective treatments are urgently needed. ROADMAP aims to deliver methods and tools that will allow the scalable, transferable integration of data on patient outcomes in the real world. The tools will be developed and tested through pilot projects and will lay the foundations for a Europe-wide platform on real world evidence in Alzheimer’s disease. The project will also deliver tools for patient engagement and address the ethical, legal and social implications of adopting a real world evidence approach to Alzheimer’s disease.

HARMONY

Blood cancers, or haematologic cancers (e.g. leukaemia, lymphoma and myeloma), affect the production and function of blood cells and account for about one third of cancer cases in children and about one third of cancer deaths. As many blood cancers are rare, and healthcare practice varies across EU, a lack of data on relevant outcomes represents a challenge for clinicians, researchers, and decision-makers alike. HARMONY aims to use ‘big data’ to deliver information that will help to improve the care of patients with these diseases. More broadly, the project will result in a pan-European network of stakeholders with expertise in this disease area.

BigData@Heart

Cardiovascular disease (CVD) is a major killer in Europe, accounting for 45% of all deaths. BigData@Heart focuses on three types of CVD. It aims to develop new definitions of diseases and outcomes; informatics platforms that link, visualise and harmonise different data sources; data science techniques; and guidelines on the cross-border use of big data resources. In the long term, the project expects to have an impact on our understanding of heart disease, the discovery of new targets for treatments, and progress towards personalised treatments for CVD.

PIONEER

Prostate cancer accounts for 9 % of all cancer deaths in men. Currently, it is hard to predict which patients will respond best to different treatments, and which patients can be managed safely without undergoing treatment. PIONEER is using big data to address key knowledge gaps related to the screening, diagnosis and treatment of prostate cancer patients. To do this, they will standardise and integrate existing ‘big data’ from sources such as clinical trials and electronic health records into a single, innovative data platform. The project will draw on this database to identify ways to improve prostate cancer outcomes and health system efficiency. Ultimately, the project results should feed back into clinical centres so that patients can benefit from the best possible care.

EHDEN

Healthcare data has the potential to transform our understanding of health, disease and outcomes, yet it is currently scattered across multiple institutions and countries, stored in different formats, and subject to different rules. This makes it very difficult to fully utilise this data to benefit patients.  The goal of EHDEN is to make the large-scale analysis of health data in Europe a reality. The project aims to do this by building a federated data network of allowing access to the data of 100 million EU citizens standardised to a common data model. At the heart of the project will be a group of trained, certified small and medium-sized enterprises (SMEs) responsible for transforming the data owned by hospitals to the common data model. The data will remain under complete control of the original data owner, thereby ensuring ethical and local data privacy rules are respected.

Achievements & News

EHDEN looking for SMEs to take on health data challenge
April 2019

IMI’s EHDEN project is looking for small and medium-sized enterprises (SMEs) to get involved in the project’s efforts to map and convert health data from diverse sources to a common model. The project has launched a pilot call for SMEs who want to get training and certification in this exciting new field. ###EHDEN’s ambitious goal is to standardise 100 million patient records across Europe covering different data source types, including hospitals, registries, and population databases. Transforming this data to the Observational Medical Outcomes Partnership (OMOP) common data model (CDM) is an immense task that EHDEN hopes will be carried out by a community of SMEs. SMEs selected through this pilot call will receive training in this and a certification that will highlight their expertise in this area. ‘We expect that those who receive training and certification through EHDEN will actually go on to perform mappings to help build the network,’ the project writes. ‘We also hope that they will become active members of the community, sharing ideas and possibly helping to develop or improve data mappings and analysis tools.’ According to the project, SMEs will also benefit from access to a larger market, the opportunity to build expertise in and contribute to a relatively new field, and the support of the wider EHDEN community. Finally, certified SMEs will receive invitations to additional training sessions as well as hackathons to improve the open source tools and other events.

  • Details of how to apply can be found on the EHDEN website.
  • Deadline for applications: 1 May 2019

First blood cancer data transferred to HARMONY big data platform
April 2019

Partners in blood cancer alliance HARMONY have uploaded the first datasets into the HARMONY Big Data Platform. This is a major milestone for the large alliance that aims to improve the treatment of haematologic malignancies. This first batch of data focuses on acute myeloid leukaemia (AML), a form of blood cancer that is most common in the over 75s. ###There are effective treatments for AML, but many elderly and weaker patients are not strong enough to tolerate the intensive chemotherapy regimens. Improved insight into the molecular basis of the disease is essential to develop better treatments for these patients. This requires data from thousands of patients. Therefore, HARMONY is bringing together datasets from all over Europe. The datasets that were recently uploaded into the Big Data Platform are from the German Austrian AML Study Group (AMLSG); the Haemato Oncology Foundation for Adults in the Netherlands (HOVON); and Novartis’s RATIFY trial. More HARMONY Partners are ready to transfer their data to the platform as well. ‘With such positive efforts in sharing aggregated data of hundreds of patients, the hope is that other data custodians across industry and academia will also be inspired to join the HARMONY Alliance’, said HARMONY project lead Mirko Vukcevic from Novartis. ‘In the future, the aspiration is that the HARMONY Alliance model will allow us to create a blueprint that can be applied to future projects in other disease areas, ultimately benefiting many more patients in Europe and well beyond.’ HARMONY is a unique network of more than 80 public-private organizations. It is part of IMI’s Big Data for Better Outcomes programme.

BD4BO releases toolkit to assist big data projects on outcomes work
July 2018

DO>IT, the coordination project of the Big Data for Better Outcomes (BD4BO) programme, has delivered a toolkit to support the other BD4BO projects in the identification, selection and measurement of outcomes. The BD4BO programme currently has projects focusing on Alzheimer’s disease, blood cancers, prostate cancer, and heart disease. ###The new toolkit represents a practical guide which will help the projects to adopt a standardised approach when developing core outcome sets (COS) in their disease areas. The toolkit proposes six main stages for developing 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 for developing COS, it 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 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.

Participants

Details of all project participants can be found on the individual project factsheets.

CONTACT

Contact details for the projects can be found in the individual project factsheets.