[bg_collapse view=”link” color=”#006BB9″ icon=”arrow” expand_text=”Organisations, networks and taskforces working in the areas of RWD and RWE” collapse_text=”Show Less” inline_css=”font-size: 18px” ]
|Australian and New Zealand Real-World Data Network (RADiANT)
|RADiANT is a researcher-led scientific network established to promote policy that support good practice in the access to and use of real-world data for research. A related initiative is the secondary use of Australia’s electronic health record system. The data in this EHR can be used to provide insight into treatment effectiveness, in order to improve health outcomes and ensure patients are receiving evidence-based care.
|Clinical Trials Transformation Initiative CTTI
|CTTI is a broad collaboration of over 80 organisations (primarily in US) whose mission is to develop and drive adoption of practices that will increase the quality and efficiency of clinical trials. CTTI has a workstream promoting greater awareness and appropriate use of RWD sources in clinical trials planning
|Core Outcome Measures in Effectiveness Trials (COMET) Initiative
|The COMET Initiative brings together people interested in the development and application of agreed standardised sets of outcomes, known as ‘core outcome sets’ (COS). COMET works to develop and apply agreed standardised sets of outcomes that should be measured and reported in all clinical trials on a specific condition. They have developed a database of all studies relevant to the development of core outcome sets for use in clinical trials.
|The Duke-Margolis Center for Health Policy has an ongoing research initiative in ‘Improving the Development and Use of Real-World Data and Evidence’. This includes collaborative projects on enhancing regulatory acceptability of RWE, improving methods of data collection and use and strengthened the shared infrastructure for evaluating medical products.
|EMA Patient Registries Initiative
|The European Medicines Agency (EMA) has set up an initiative to make better use of existing registries and facilitate the establishment of high-quality new registries if none provide an adequate source of post-authorisation data for regulatory decision-making.
|European Federation of Pharmaceutical Industries and Associations (EFPIA)
|EFPIA represent the interests of research-based biopharmaceutical companies and trade associations throughout Europe. The Integrated Evidence Generation & Use Working Group coordinates engagement on RWE-related issues impacting the full span of the product lifecycle.
|European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP)
|ENCePP is an EMA-coordinated network of public institutions and contract research organisations that develops and maintains methodological standards and governance principles for pharmacovigilance and pharmacoepidemiology (e.g., Guide on Methodological Standards in Pharmacoepidemiology, Checklist for Study Protocols, Code of Conduct for promoting transparency and scientific independence in the research process) and maintains the EU Register of Post-Authorisation Studies (EU PAS).
|European Network of HTAs (EUnetHTA) – Joint Action 3
|EUnetHTA Joint Action 3 (running from 2016 to 2020) aims to implement a sustainable model for scientific and scientific cooperation on HTA in Europe. Work package 5 will consider the generation of evidence along the life cycle and develop tools that support collection and use of high quality data.
|Haute Autorite de Sante (HAS)
|HAS is an HTA agency based in France. HAS are involved in EUnetHTA, as well as undertaking research as part of the GetReal project relating to reimbursement decisions based on real-world evidence review.
|Health Technology Assessment International (HTAi)
|HTAi’s mission is to support and promote the development, communication, understanding and use of HTA around the world as a scientifically-based and multidisciplinary means of informing decision-making on the use of effective technologies and the efficient use of resources in health care. HTAi’s Global policy forum in 2018 focused on the use of RWE in HTA.
|HMA Clinical Trial Facilitation and Coordination Group (CTFG)
|The CTFG is a forum for discussion and agreement by the Heads of Medicines Agencies (HMA) on common principles and processes for clinical trials in Europe, including pragmatic clinical trials. It co-ordinates the implementation of the EU Clinical Trials Directive 2001/20 EC across member states.
|HMA/EMA EU-Innovation Network
|The HMA/EMA EU-Innovation network is a regulatory forum for the support of uptake of innovative methods in medicines development through case study discussion, sharing of best practices, horizon scanning, and identification of assessor training needs.
|HMA/EMA Joint Task Force on Big Data
|Task force of EMA and national competent authority representatives exploring challenges associated with the use of big data in the assessment of medicines and developing a roadmap and recommendations for solutions.
|IMI GetReal Initiative
|The GetReal Initiative, launched in 2018, brings together partners from the original IMI GetReal project, with the ultimate goal to drive the adoption of tools, methodologies and best practices from GetReal, and increase the quality of real-world evidence generation in medicines development and regulatory/HTA processes across Europe.
The Initiative has an established ThinkTank, consisting of senior leaders in the generation and use of real-world evidence, that consult on the activities and vision of the Initiative.
Also, the GetReal Initiative has two task forces that drive the focused development of tangible solutions to the key challenges identified in IMI GetReal and the Think Tank. These task forces have facilitated the development of the ADDIS tool and the GetReal Initiative Trial Tool.
|Institute for Clinical and Economic Review (ICER)
|ICER is an independent and non-partisan research organisation that objectively evaluates the clinical and economic value of prescription drugs, medical tests and other health care and health care delivery innovations within the US. They are involved in research surrounding how real-world evidence can be incorporated in value assessments and insurer coverage decisions.
|International Consortium for Health Outcome Measurement (ICHOM)
|ICHOM develops and promotes adoption of disease-specific core outcome measure sets for use in clinical practice and quality improvement activities.
|International Network of Agencies for Health Technology Assessment (INAHTA)
|INAHTA is a network of 51 HTA agencies that support health system decision making. These agencies co-operate and share information about producing and disseminating HTA reports for evidence-based decision making. They have collaborated with EUnetHTA, and have been involved with task groups related to real world data as part of HTAi.
|International Society for Pharmacoeconomics and Outcomes Research (ISPOR)
|ISPOR is a professional society for health economics and outcomes research. They develop influential guidelines for methodological best practices. They have collaborated with ISPE on some RWE issues such as transparency of database studies. An ISPOR-convened multi-stakeholder work group is currently developing recommendations for improving the transparency of observational research through, e.g., broader public registration of studies and posting of results.
|International Society for Pharmacoepidemiology (ISPE)
|ISPE is a professional society for pharmacoepidemiology. They develop influential methodology and governance guidelines (e.g., Guidelines for Good Pharmacoepidemiology Practices). They have collaborated with ISPOR on some RWE issues such as transparency of database studies.
|Joint ISPOR/ISPE Special Task Force
|The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and International Society for Pharmacoepidemiology (ISPE) have collaborated to form a workforce dedicated to improving standards and practice for the collection and analysis of real-world data.
|National Health Care Institute, the Netherlands (ZIN)
|ZIN is an HTA agency based in the Netherlands. They have contributed to projects related to RWD through their involvement in the GetReal Initiative, EUnetHTA and HTx. Specifically, a review of their conditional reimbursement scheme was undertaken as part of the GetReal project.
|NEW Drug Development ParadIGmS (NEWDIGS)
|A network of 31 institutions led by Massachusetts Institute of Technology (MIT) aimed at enhancing the capacity of the global biomedical innovation system to reliably and sustainably deliver new, better, affordable therapeutics to the right patients faster. NEWDIGS members recently co-authored a study setting out ways of enabling patients to access innovative medicines faster (Baird et al., 2014).
MIT also hosts other departments with an interest in Adaptive Licensing including the Center for Biomedical Innovation and the Department of Political Science and Engineering Systems Division with relevant studies being published across these. See also the Janus Initiative here.
|Observational Health Data Sciences and Informatics (OHDSI)
|OHDSI promote adoption of the Observation Medical Outcomes Partnership (OMOP) common data model. A European chapter of ODHSI was established in 2018.
|Patient-Centered Outcomes Research Institute (PCORI)
|PCORI is a non-profit, non-governmental organisation in the US with a mandate to improve the quality and relevance of evidence available to support informed health decisions.
|PCORnet is a US-based national patient-centred clinical research network ‘to make health research more efficient and powerful, and less expensive, by collaboratively harnessing the power of large amounts of health data’; this network includes both clinical research and patient-powered research networks.
|Pharmaceutical Research and Manufacturers of America (PhRMA)
|PhRMA represent the interests of major research-based pharmaceutical companies in the US. Their RWE Working Group focuses on barriers to the use of RWE to support regulatory decision-making in the US.
|Real-World Evidence Transparency Initiative Partnership
|The Real-World Evidence Transparency Initiative Partnership is a collaboration between ISPOR, the International Society for Pharmacoepidemiology, the Duke-Margolis Center for Health Policy, and the National Pharmaceutical Council. The objective of this initiative is to establish a culture of transparency for study analysis and reporting of hypothesis evaluating real-world evidence studies on treatment effects.
|The Swedish Dental and Pharmaceutical Benefits Agency (TLV)
|TLV is an HTA agency based in Sweden. They are involved in multiple projects and networks related in the use of RWD, including EUnetHTA, HTx and HTAi.
|TransCelerate BioPharma Inc.
|TransCelerate BioPharma Inc. is a non-profit association of biopharmaceutical companies that develops pragmatic solutions, such as templates, guidance, and model frameworks and systems, to address barriers that constrain the development of new medicines, particularly in the clinical development phase. Current activities include work to improve how patients experience the clinical trial process, improved data sharing and harmonisation, and drug safety initiatives.
|U.S. Food and Drug Administration (FDA)
|The FDA has a number of initiatives relevant to RWE: Patient-Focused Drug Development Initiative, the Patient Representative Program, and the Patient Preferences Initiative.
|UK National Institute of Health and Care Excellence (NICE)
|NICE is an HTA agency based in the UK. NICE is involved in a variety of research projects relating to the use of real-world data in the HTA process. NICE published a statement of intent to use different types of evidence in its guidance development, including RWD, (Technical Support Document DSU17), in order to improve the quality and transparency of its future assessments.
|US National Evaluation System for health Technology Coordinating Center (NESTcc)
|NESTcc, an initiative of the Medical Device Innovation Consortium, seeks to support the sustainable generation and use of timely, reliable and cost-effective real-world evidence through the medical device lifecycle, using real-world data that meets robust methodological standards, currently undergoing development by its data quality and methods subcommittees.
|Sentinel is a medical product monitoring system in the US, launched by the US FDA in 2008. Based on a distributed database model, it has largely been used by the US FDA’s Center for Drug Evaluation and Research (CDER) to assess the safety of prescription drugs. A current strategic aim is to ‘Accelerate access to and broader use of real-world data to evaluate effectiveness’ in partnership with the FDA-Catalyst program.
Additionally, a number of international projects are either ongoing or have been completed in this area. We list these below. (last updated February 2020).
[bg_collapse view=”link” color=”#006BB9″ icon=”arrow” expand_text=”Ongoing projects” collapse_text=”Show Less” inline_css=”font-size: 18px” ]
|Childhood and adolescent obesity is a major global and European public health problem. BigO aims to develop a platform through which real-world data related to obesity can be collected and utilised in the design and implementation of public health policy. Duration 4 years (December 2016 to November 2020).
|The overarching objective of the COMED project is to push the boundaries of existing methods for cost and outcome analysis of healthcare technologies, both within the Health Technology Assessment (HTA) and Health System Performance (HSP) framework. COMED’s aim is to develop new methods for assessing patient reported outcomes for mHealth technologies, while their ambition is to develop scientifically rigorous recommendations on what, when and how real world evidence sources can and should be used for assessment of medical devices. Duration 3 years (January 2018 to December 2020).
|Sudden cardiac arrest causes ~20% of all deaths in Europe. ESCAPE-NET focuses on exploiting all available data around sudden cardiac arrest to improve SCA management. They aim to build a unique and growing database of >100,000 DNA samples, including >20,000 SCA patient samples, by combining already existing databases and infrastructures. By using this, risk factors and treatment strategies will be better identified, and will be translated into changes to clinical practice. Duration 5 years (January 2017 to December 2023).
|H2020 HTx (Next Generation Health Technology Assessment)
|HTx aims to create a framework for the Next Generation Health Technology Assessment (HTA) to support patient-centred, societally oriented, real-time decision making on access to and reimbursement for health technologies throughout Europe. This project includes a work package centred around real world evidence methods development. Duration 5 years (January 2019 to December 2023).
|IASIS aims to integrate clinical data from multiple sources, including genomics, electronic health records and academic literature to create a global health knowledge base. This knowledge base could then be used to inform policy decision making and provide precise, personalised care to patients. Duration 3 years (April 2017 to March 2020).
|The MIDAS consortium is a partnership involving health authorities in five EU countries and the U.S. and technical big data experts from research institutions, MNCs and SMEs. Managing big data for ‘health in all’ is a monumental challenge for policy makers. MIDAS is addressing this challenge by developing and delivering an integrated solution which will liberate knowledge from data silos and unify heterogeneous big data sources to provide evidence-based actionable information and transform the way care is provided. Duration 3.3 years (November 2016 to February 2020).
|H2020 IMPACT HTA (Improved Methods and Actionable Tools for Enhancing HTA)
| IMPACT HTA is a research project looking at new and improved methods across ten thematic areas aiming at:
Duration 3 years (January 2018 to December 2020).
|IMI Big Data for Better Outcomes (BD4BO)
|The Big Data for Better Outcomes programme aim is to act as a catalyst, supporting the evolution of European healthcare systems to becoming more outcomes-focused and sustainable by capitalising on the opportunities of using big and deep data sources to generate evidence to inform policy debates.
IMI BD4BO projects are:
with co-ordinating projects
|IMI BigData@Heart (part of BD4BO)
|BigData@Heart aims to develop new definitions of cardiovascular 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. Duration 5 years (March 2017 to February 2022).
|The ultimate goal of ConcePTION is to create a trusted biomedical ecosystem capable of providing evidence-based information on the safety of medications during pregnancy and breastfeeding in an efficient, systematic and ethically responsible way. Duration 5 years (April 2019 to March 2024).
(part of BD4BO)
|The aim of EHDEN is to enable large-scale analysis of health data by building a federated data network, allowing access to the data of 100 million EU citizens standardised to a common data model. Duration 5 years (November 2018 to April 2024).
(part of BD4BO)
|HARMONY aims to use ‘big data’ to deliver information that will help to improve the care of patients with haematologic cancers. Duration 5 years (January 2017 to December 2021).
|The aim of MOBILISE-D is to produce validated and accepted digital mobility outcomes to monitor daily life gait of people with different mobility problems, with the goal to improve follow-up and personalized care and improve outcome assessment in clinical trials. The objective of Work Package 2 of this project is to create a database of existing real-world and laboratory data and algorithms for the quantification of digital mobility outcomes. Duration 5 years (April 2019 to March 2024).
|Patients Active in Research and Dialogues for an Improved Generation of Medicines (PARADIGM) aims to develop a framework, processes, and tools for patient engagement in research priority settings, clinical trial design, and early scientific dialogues. Duration 2.5 years (March 2018 – October 2020).
(part of BD4BO)
|The aim of PIONEER aim is to use big data to address key knowledge gaps related to the screening, diagnosis and treatment of prostate cancer patients. Duration 5 years (May 2018 to April 2023).
|The aim of PREFER is to develop recommendations for guidelines for industry, regulators, and HTA organisations on how and when to incorporate patient preferences/perspectives on benefits and risks of medicines Duration 5 years (October 2016 to September 2021).
|RADAR-AD aims to develop a digital platform that draws on smartphone, wearable and home-based digital technologies to track subtle changes in the cognitive and functional abilities of people with Alzheimer’s disease. The project is adapting the existing RADAR-CNS project’s platform for this purpose. Duration 3 years (April 2019 to June 2022).
|RADAR-CNS aims to develop new ways of monitoring major depressive disorder, epilepsy, and multiple sclerosis using wearable devices and smartphone technology. The key goal of the project is to improve patients’ symptoms and quality of life and to change how these and other chronic disorders are treated. Duration 5 years (May 2016 to April 2021).
|Trials@Home aims to explore the potential of digital technologies for use in ‘remote decentralised clinical trials’ (RDCTs). They will develop and test methods to streamline data collection as well as patient recruitment and retention. Duration 5 years (September 2019 – August 2024).
|IMI WEB-RADR 2
|WEB-RADR 2 aims to further develop take the WEB-RADR app by making its functionalities available through application programming interfaces (APIs). Duration 2 years (September 2018 to March 2020).
|Observational Patient Evidence for Regulatory Approval and uNderstanding Disease (OPERAND)
|Convened by Optum Labs and Harvard Medical School, a privately-funded, multi-stakeholder initiative that seeks to use observational (EHR/EMR and claims) data to validate the results of published RCTs
|An FDA-funded demonstration project by Harvard researchers that seeks to use observational (claims) data to validate the results of ~30 published RCTs. The intent is to understand for what types of clinical questions RWD analyses can be conducted with confidence and with which study designs and analytic methods. It is similar to OPERAND in its overall goal but differs in approach in several respects.
|A privately funded, Harvard-based project that is evaluating the transparency and reproducibility of published RWD (claims) analyses of medication effectiveness and safety. Researchers will attempt to reproduce the results of 150 published studies based on publicly reported information.
[bg_collapse view=”link” color=”#006BB9″ icon=”arrow” expand_text=”Completed projects” collapse_text=”Show Less” inline_css=”font-size: 18px” ]
|EMA Adaptive pathways to patients
|EMA ran adaptive pathway pilots, followed by exploration of the concept further in the context of parallel scientific advice with HTA bodies. Duration 2.5 years (March 2014 – August 2016)
|Cancers of the Head and Neck Region (HNC) are the 6th most deadly cancers worldwide. BD2Decide aimed to produce a decision support system that provides clinicians with population specific epidemiological data, patient-specific genomic, clinical and imaging data and big data multiscale prognostic models to improve patient care. Duration 3.75 years (January 2016 to September 2019)
|The current management strategy of hearing loss is to provide hearing aids, although their use is often problematic and costly, with little overall clinical benefit. EVOTION aimed to utilise large heterogeneous datasets provided by hearing loss patients (physiological, cognitive, behavioural, lifestyle, occupational and environmental data) combined with clinical repositories to develop an integrated platform that support public health policy decision making. Duration 3.25 years (November 2016 to January 2020).
|IMI ADAPT SMART
|ADAPT-SMART was an IMI project enabling a platform for coordinating Medicines Adaptive Pathways to Patients (MAPPs) activities which looked to ‘foster access to beneficial treatments for the right patient groups at the earliest appropriate time in the product life-span in a sustainable fashion’. (July 2015 – April 2018)
|IMI DO>IT (part of BD4BO)
|The DO->IT project was the overall coordinator of the BD4BO (Big Data for Better Outcomes) programme activities within IMI and established the mechanisms for a sustainable focus on outcomes-based healthcare delivery (February 2017 – January 2019)
|IMI Electronic Health Record systems for Clinical Research (EHR4CR)
|EHR4CR aimed to provide adaptable, reusable and scalable solutions (tools and services) for reusing data from electronic health record (EHR) systems for clinical research. (March 2011 – February 2016)
|IMI European Medical Information Framework (EMIF)
|The IMI EMIF project was launched to develop common technical and governance solutions and improve access and use of health data. A common Information Framework (EMIF-Platform) will link up and facilitate access to diverse medical and research data sources. (January 2013 – June 2018)
|IMI ROADMAP (part of BD4BO)
|The IMI ROADMAP project aimed to deliver a series of methods and tools that will allow the scalable, transferable integration of data on patient outcomes in Alzheimer’s Disease in the real world. (November 2016 – October 2018)
|WEB-RADR developed a mobile application which allows patients to directly report potential medicine side effects and receive reliable information on their drugs. (September 2014 – December 2017)
In addition to the initiatives listed above, there are additional initiatives listed on individual pages within this site (for example, on specific RWD sources).
Emily Leckenby, NICE
Dalia Dawoud, NICE
Michael Chambers, MC Healthcare Evaluation