Incorporating real-life clinical data into drug development


Start Date
End Date
IMI1 - Call 7
Grant agreement number

IMI Funding
8 000 000
EFPIA in kind
6 910 397
1 367 103
Total Cost16 277 500


Incorporating data from ‘real life’ clinical settings into drug development and associated decision-making represents a serious challenge for pharmaceutical companies, regulators, and health authorities alike. By bringing together all key stakeholder groups (namely industry, academia, regulatory agencies, reimbursement agencies, healthcare budget holders, and patient groups) to share their insights and know-how, GETREAL will develop new approaches for incorporating real life data into drug development, and pave the way for a greater consensus on this issue.

Bridging the data gap

Once a new drug has been developed, it must be reviewed by both the regulators (for the decision on marketing authorisation) and health technology assessment (HTA) bodies (for the decision on access to patients). The regulators draw on data, mostly from clinical trials, to determine if a drug is safe and works well enough to be authorised for use in patients. For their part, HTA organisations assess more broadly the value of a new drug for a given healthcare system. For this, they need data to support the assessment of ‘relative effectiveness’: that is the extent to which a treatment does more good than harm when compared to one or more alternative treatments when provided under normal healthcare circumstances.

However, while there are clear, detailed guidelines on how to carry out clinical trials, there is little guidance on how to generate real world data and integrate this into drug development before launch. This is a serious issue in drug development – even if a drug is approved as safe and effective by the regulators, insufficient evidence supporting relative effectiveness at the time of launch may delay, or restrict (partially or entirely) patient access to new treatments. The challenge, therefore, is to incorporate relative effectiveness research into the earlier stages of drug research and development, and that is where the GETREAL project comes in.

Injecting real life data into drug development

The goal of GETREAL is to develop new ways of incorporating real-life clinical data into drug development. The benefits of this are manifold – not only will it help pharmaceutical companies take better decisions during drug development, it will aid healthcare decision makers when deciding how best to grant patients access to a new treatment.

Among other things, the project will analyse existing processes and methodologies for HTA. It will also generate a decision-making framework to help pharmaceutical companies design drug development strategies. The framework would include ideas for the design of trials and studies capable of providing information on the real world effectiveness of medicines, including relative effectiveness.

The project also aims to develop tools that will allow different stakeholders in drug development and approval to test different mathematical models in their decision making and assess their impact.

A significant part of the project is devoted to organising training activities for researchers, healthcare decision makers and other stakeholders on relative effectiveness concepts and how they can be applied.

Finally, the project will create a network of regulators, HTA organisations, companies, academics, healthcare professionals, patients and other societal stakeholders. This is important because the challenges faced by the pharmaceutical industry, regulators and other healthcare decision makers are linked.

By bringing together all key stakeholder groups (namely industry, academia, regulatory agencies, HTA bodies, reimbursement agencies, healthcare budget holders, and patient groups) to share their insights and know-how, GETREAL will help to generate a consensus on best practice in the timing, performance and use of real life clinical studies in regulatory and reimbursement decision-making. It will also help to create a strong platform for the communication of results and for future discussions in this important area.

Achievements & News

GetReal issues recommendations on real world evidence in drug development
November 2017

Launched in October 2013, GetReal was set up to explore how how robust new methods of real-world evidence (RWE) collection and synthesis could be adopted earlier in pharmaceutical R&D and the healthcare decision making process. The project, which brought together experts from diverse backgrounds, has delivered a range of tools and resources to help in this, including tools on the use of RWE and pragmatic clinical trials, as well as an online course on RWE. ### Now, the project’s Policy Expert Group has set out seven key themes that it believes require further attention to advance the use of RWE and real world data (RWD) in drug development.

1. Integrity, quality, access and privacy protection of RWD sources

2. Guidance on RWE study design, evidence synthesis and interpretation in decision making

3. Standards for decision makers’ use of RWE in decision making

4. RWE training and education

5. Broader involvement of stakeholders, especially patients and healthcare professionals, in RWE generation and use of RWD

6. Emphasis on a joint (regulatory/HTA/payer) scientific advice process

7. Construction of a standing forum and linking with ongoing initiatives

GetReal launches tool to assist pragmatic trial design
July 2017

IMI’s GetReal project has launched an innovative tool to aid in pragmatic clinical trial design. Pragmatic trials aim to capture the true effect of a treatment strategy in the real world. As such, they provide a great opportunity to generate real-world evidence in the early stages of drug development. However, pragmatic trials are still in their early days and designing them is far from easy.### GetReal designed the PragMagic tool in collaboration with gaming company IJsfontein. ‘In a user-friendly (and dare we say fun) way, PragMagic gives insight into the possible consequences of more pragmatic trial design choices and operational challenges on generalisability, risk of bias, precision, stakeholder & ethical acceptability, cost and duration,’ explained Mira Zuidgeest of the University Medical Center Utrecht. The project hopes that the new tool will increase the value and feasibility of pragmatic trials by informing stakeholders when designing and assessing pragmatic clinical trials. ‘The tool helps to balance the aim to be pragmatic with the feasibility of the trial and other implications of design choices such as validity of the trial results,’ said Dr Zuidgeest. For more information on the tool, visit the PragMagic website.

Sign up for the GetReal course on real-world evidence in medicine development
June 2017

IMI’s GetReal project will run the first edition of an interactive online course entitled ‘Real-world evidence in medicine development’ from 2nd October to 12 November 2017. Under the supervision of leading academics from the project, the course will provide students with an introduction to the topics covered by GetReal, and present the current techniques, opportunities and challenges for the use of real-world evidence in medicine development. Participants are expected to come from pharmaceutical companies, regulatory authorities, health technology assessment (HTA) bodies, patients’ organisations, consultancy companies and academia. As such, the course will foster discussion, interaction and mutual learning across all professionals involved in medicine development.###

The course will focus on the following topics:

  • overview of the medicine development landscape
  • real-world evidence generation
  • real-world evidence synthesis
  • decision-making and weighing of evidence
  • demonstrating relative effectiveness to decision-makers

Participants who successfully complete the assignments will be awarded a certificate.

GetReal launches Real World Evidence Navigator
March 2017

In March, the GetReal project launched the Real-World Evidence (RWE) Navigator. This is an online resource designed to help users from a broad range of research and healthcare backgrounds get to grips with RWE. RWE refers to evidence of relative effectiveness - how well medicines work in the real world, as opposed to the controlled experimental settings of clinical trials.### Data to generate RWE can come from a range of sources, including patient registries, medical records, pragmatic trials, and other observational studies. The overall aim of IMI’s GetReal project was to explore how new, better ways of generating and using RWE can be incorporated earlier on in drug development and healthcare decision-making. The RWE Navigator is an educational resource to help users find out more about potential issues in demonstrating the relative effectiveness of new medicines (referred to as ‘effectiveness issues’). It also guides users to specific types of analyses or study designs that use RWE to support the development of medicines. Finally, it is a comprehensive directory of resources that support the use of RWE in medicines development. The RWE Navigator has been designed for a wide variety of users. For example, pharmaceutical companies may find it useful to increase awareness about the potential use of RWE among their medicine development teams, or patients may use it to understand concepts related to RWE and the challenges faced when of using or generating RWE.

GetReal to host a closing meeting in Brussels
October 2016

Before it draws to a close at the end of this year, IMI’s GetReal project will hold a closing event to showcase its results and discuss its future legacy. The event, titled ‘Delivering tools for real-world evidence development’, will take place on 24 November 2016 at the Royal Flemish Academy of Belgium for Science and Arts in Brussels.### Registrations are on a first-come, first-served basis. This event comes five months after a big stakeholder conference in London which aimed to gather perspectives on the potential value of GetReal in decision-making within industry, regulatory authorities, and patient groups. The report from that event is now available online. Launched in October 2013, GetReal is a three-year project which aims to show how robust new methods for real-world evidence collection and synthesis could be adopted earlier in pharmaceutical research and the healthcare decision-making process. It brings together 29 organisations representing pharmaceutical companies, academia, regulators, patient organisations and SMEs.

GetReal and ADAPT-SMART launch glossaries
June 2016

Information on drug development is often filled with jargon. Now IMI’s GetReal and ADAPT SMART projects  have released new/updated glossaries to facilitate discussions in their respective subject areas.###

GetReal is investigating how to best incorporate ‘real world evidence’ into drug development. Its updated glossary, which was the subject of a consultation, also includes a more detailed discussion on questions such as the difference between ‘efficacy’ and ‘effectiveness’.

ADAPT SMART was set up (among other things) to coordinate IMI activities in the area of MAPPs (medicines adaptive pathways to patients). Many of the items in its glossary are drawn from the GetReal glossary. The project welcomes feedback on the glossary.

Interested in real world evidence in drug development? Sign up for GetReal’s webinars!
March 2016

IMI’s GetReal project will hold a number of events this year on the themes of relative effectiveness and how it can best be integrated into drug development. Registration is now open for the following events.###

18 April: Introduction to the concept of drivers of effectiveness - The webinar will introduce the concept of drivers of effectiveness and will provide an overview of the methods which can be used to identify them early during the drug development plan.

10 May: Synthesis and integration of real-world evidence in network meta-analyses and outcome prediction - The focus of this webinar will be on the use of network meta-analysis for evidence synthesis and on the extension of its conclusions in real-world settings, using predictive modelling.

Further webinars are planned for later in the year. Visit the GetReal website for the latest updates.

GetReal workshops promote discussion on use of real-world evidence
September 2015

IMI’s GetReal consortium has run the first five workshops bringing together stakeholders to discuss the use of real-world evidence for demonstrating the effectiveness of new drugs. Workshop participants have included so far: patients, clinicians, academic specialists, clinical trialists, pharmaceutical company representatives, regulators, health technology assessment (HTA) bodies and payers.### Collaboration between these diverse stakeholders is paramount for the success of this project and this is the first time many of the participants have worked together in a collaborative environment. By demonstrating that participation is both safe and beneficial, GetReal has successfully created a new platform for interlocutors to engage and share new ideas.

Each workshop includes up to 40 participants and the outputs will inform the delivery of a framework which will outline the potential uses of real-world evidence at different stages of medicine development. The workshops are part of case studies in the following disease areas:

  • Multiple sclerosis
  • Non-small cell lung cancer
  • Rheumatoid arthritis
  • Chronic obstructive pulmonary disease
  • Metastatic melanoma

Launched in October 2013, GetReal is a three-year project which aims to show how robust new methods for real-world evidence collection and synthesis could be adopted earlier in pharmaceutical research and the healthcare decision-making process.

Participants Show participants on map

EFPIA companies
  • Amgen NV, Brussels, Belgium
  • AstraZeneca AB, Södertälje, Sweden
  • Bayer AG, Berlin, Germany
  • Boehringer Ingelheim International GmbH, Ingelheim, Germany
  • Bristol-Myers Squibb Company , Princeton, NJ, United States
  • Eli Lilly and Company Ltd, Basingstoke, United Kingdom
  • F. Hoffmann-La Roche Ltd, Basel, Switzerland
  • Glaxosmithkline Research And Development LTD, Brentford, Middlesex, United Kingdom
  • Janssen Pharmaceutica NV, Beerse, Belgium
  • Merck KGaA, Darmstadt, Germany
  • Merck Sharp & Dohme Corp., Whitehouse Station, New Jersey, United States
  • Novartis Pharma AG, Basel, Switzerland
  • Novo Nordisk A/S, Bagsvaerd, Denmark
  • Sanofi-Aventis Research and Development, Chilly Mazarin, France
  • Takeda Development Centre Europe Ltd., London, United Kingdom
Universities, research organisations, public bodies, non-profit groups
  • Academisch Ziekenhuis Groningen, Groningen, Netherlands
  • European Organisation for Research and Treatment of Cancer, Brussels, Belgium
  • Haute Autorité de Santé, Saint-Denis la Plaine , France
  • International Alliance of Patients' Organizations, London, United Kingdom
  • National Institute for Health and Care Excellence, Manchester, United Kingdom
  • Panepistimio Ioanninon, Ioannina, Greece
  • The University of Manchester, Manchester, United Kingdom
  • Universitair Medisch Centrum Utrecht , Utrecht, Netherlands
  • University of Leicester, Leicester, United Kingdom
  • Universität Bern, Bern, Switzerland
  • Zorginstituut Nederland, Diemen, Netherlands
Small and medium-sized enterprises (SMEs)
  • LA Sante Epidemiologie Evaluation Recherche sarl (LA-SER) , Paris, France
Non EFPIA companies
  • European Medicines Agency, Canary Wharf, London, United Kingdom


Project coordinator
Managing entity
Diederick Grobbee
Universitair Medisch Centrum Utrecht