Start Date
End Date
IMI2 - Call 5
Grant agreement number

Type of Action: 
RIA (Research and Innovation Action)

IMI Funding
6 000 000
EFPIA in kind
6 000 000
15 548
Total Cost
12 015 548


Patient perspective is important in all medical research and particularly in drug development. The PREFER project aims to assess when and how patient preferences on benefits and risks should be incorporated into decisions on medicinal products. While industry, regulatory authorities, health technology assessment bodies, reimbursement agencies and patient organisations all agree that patient preferences are very valuable, there is little guidance on conducting and using such studies. The goal of PREFER will be to provide a set of systematic methodologies and recommendations to assess, engage and include patient perspectives during the development, approval, and post-approval of new therapies. PREFER brings together experts from academic research institutions, pharmaceutical companies, patient organisations, a health technology assessment body, and small and medium-sized enterprises. In addition the consortium has set up stakeholder advisory groups to work closely with patients, regulators, health technology assessment (HTA) bodies and payers, to ensure that recommendations are evidence based, relevant and useful.

Achievements & News

Regulators tentatively endorse PREFER’s patient preference approach
November 2021

A major regulatory milestone has been reached for researchers working on the IMI-funded project PREFER, who have been investigating and testing the best ways to include patients’ voice in medicine development and decision-making.### A positive draft opinion from the European Medicines Agency (EMA) on the consortium’s proposed framework for patient preference studies, as well as a document outlining points to consider when selecting methods for structured patient input to medical product decision-making, recently went through a public consultation.

The IMI-funded PREFER project is currently finalising a set of recommendations that industry, regulatory authorities and HTA bodies will be able to use to develop guidelines on how and when to include patient perspectives on the benefits and risks of medicines.

In parallel, the consortium worked out a research framework for patient preference studies, as well as a document with points to consider when selecting the methods for carrying out a patient preference study.

The project submitted an application for a qualification opinion to the EMA-EUNETHA (the first joint regulator an health technology assessment bodies joint procedure), and the EMA released a draft opinion that endorses both the framework and the points to consider document, ‘as a comprehensive reference document for planning and conducting patient preference studies’.

The draft opinion underwent a one-month consultation period so that researchers, patients, pharma companies, regulators, HTA bodies or anyone else could contribute their comments before EMA releases its final opinion.

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PREFER probes patients’ preferences for gene therapy in haemophilia
September 2021

For innovative treatments and treatments for rare diseases, finding a way to include the patient perspective in decision-making can be crucial. With answers from 117 people with haemophilia, PREFER researchers present their results from their clinical case study about haemophilia patients’ preferences for gene therapy.### They found that patient preferences vary greatly. And that informing patients about gene therapy can facilitate acceptance.

The results from this clinical case study showed the importance of patient education on gene therapy, as the researchers included an educational tool on gene therapy at the start of their survey and the time that patients spent on this tool was found to have an impact on the results. The patients who took more time to take in the educational materials offered, tended to be more accepting of gene therapies. For innovative treatments, educational tools may be especially useful to collect the informed views of patients. This to make sure they have the information they need to make informed trade-offs between the different benefits and risks of current treatment options and gene therapy.

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IMI projects cited in new orphan drug development guidebook
September 2020

Three IMI projects – c4c, EUPATI and PREFER – are cited in a new Orphan Drug Development Guidebook released by the International Rare Diseases Research Consortium (IRDiRC). ###Developing ‘orphan drugs’ (i.e. drugs for rare diseases) is highly challenging, and just 5 % of rare diseases have an approved treatment. The IRDiRC describes the new guidebook as ‘a patient focused guidebook that describes the available tools, incentives, resources and practices for developing traditional and innovative drugs/therapies for rare diseases and how to best use them.’ It lists a range of resources from organisations around the world, including IMI.

c4c aims to generate a sustainable infrastructure that optimises the delivery of clinical trials in children, and the guidebook suggests this could be useful for those developing medicines for rare paediatric diseases.

On EUPATI, the guidebook describes its patient education resources as ‘very relevant to rare disease medicines development and highly useful and appreciated by the rare disease patients that finished the academy and use the toolbox’.

On PREFER, the guide highlights how patient preference studies can be useful during regulatory benefit-risk assessment for certain drugs in several major ways.

The guidebook is presented in an article in Nature Reviews Drug Discovery. ‘By enhancing the use of available tools, delays in development timelines can be avoided, risks and costs reduced, and patient and regulatory acceptability improved,’ the authors conclude.

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PREFER sets out 15 ways to listen to the patient voice
September 2020

There is now broad recognition that patients can and should be involved in all stages of medical research and drug development, as understanding patients’ preferences can improve decision-making. However, putting ‘patient-centricity’ into practice is not always easy. IMI’s PREFER project has now identified 15 critical decision points in industry, regulatory and health technology assessment (HTA) decision-making where input on patient preferences can support the process.###

Their findings are published in the journal Health Policy. ‘Currently, PP [patient preference] information is not considered as obligatory information to submit for any of the MPLC [medical product lifecycle] decision-points,’ the scientists write. ‘However, PP information is considered an important component by most stakeholders to inform future decision-making across the MPLC. The integration of PP information into 15 identified decision-points needs continued discussion and collaboration between stakeholders.’

First author Chiara Whichello of Erasmus University Rotterdam says: ‘We do see regulators and HTA having to take more steps to integrate patient preference information into their decision-making processes, compared to industry. Simply because they have to also take into account how much weight should be given to patients’ preferences compared to other required information, such as safety and cost-effectiveness.’

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IMI patient engagement projects sign memorandum of understanding
November 2018

Two IMI projects focusing on patient engagement, PARADIGM and PREFER, have signed a memorandum of understanding (MoU) to enhance collaboration between the projects and to maximise results. The MoU outlines how the projects will work together and share ideas. ###While PARADIGM is broadly focused on patient engagement at three points in the research and development process, PREFER looks at how and when it is best to perform and include patient preferences in decision making during the medical product life cycle. Through the MoU, the two projects hope to identify areas of mutual interest; identify any gaps that are hindering progress; establish collaborative activities to address these gaps; and share knowledge and data. The projects will also mutually participate in project events and use each other’s communications channels to promote news and results. In a joint statement, the projects write: ‘There is an ample opportunity to leverage the work of these projects, to avoid duplicate efforts as well as maximise results.’

Participants Show participants on map

EFPIA companies
  • AbbVie Ltd, Maidenhead, United Kingdom
  • Actelion Pharmaceuticals LTD, Allschwil, Switzerland
  • Amgen Limited, Cambridge, United Kingdom
  • Astellas Pharma Europe BV, Leiden, Netherlands
  • Astrazeneca AB, Södertälje, Sweden
  • Bayer Aktiengesellschaft, Leverkusen, Germany
  • Csl Behring GMBH, Marburg, Germany
  • Eli Lilly And Company LTD, Basingstoke, United Kingdom
  • F. Hoffmann-La Roche AG, Basel, Switzerland
  • Janssen Pharmaceutica Nv, Beerse, Belgium
  • Merck Kommanditgesellschaft Auf Aktien, Darmstadt, Germany
  • Merck Sharp & Dohme Corp, Whitehouse Station, New Jersey, United States
  • Novartis Pharma AG, Basel, Switzerland
  • Pfizer Limited, Sandwich, Kent , United Kingdom
  • Sanofi-Aventis Recherche & Developpement, Chilly Mazarin, France
  • Takeda Pharmaceuticals International AG, Glattpark-Opfikon (Zurich), Switzerland
Universities, research organisations, public bodies, non-profit groups
  • Centre Federal D'Expertise Des Soins De Sante, Brussels, Belgium
  • Erasmus Universiteit Rotterdam, Rotterdam, Netherlands
  • Forum Des Patients Europeens, 1040, Belgium
  • Katholieke Universiteit Leuven, Leuven, Belgium
  • The University Of Birmingham, Birmingham, United Kingdom
  • Universitair Medisch Centrum Utrecht, Utrecht, Netherlands
  • Universitatsklinikum Erlangen, Erlangen, Germany
  • University Of Newcastle Upon Tyne, Newcastle upon Tyne, United Kingdom
  • Uppsala Universitet, Uppsala, Sweden
Small and medium-sized enterprises (SMEs) and mid-sized companies (<€500 m turnover)
  • Istituto Europeo Di Oncologia SRL, Milan, Italy
  • Mindbytes, Merksplas, Belgium
  • Steinbeisser Project Management Ug (Haftungsbeschrankt), Munich, Germany
Patient organisations
  • European Cancer Patient Coalition, Brussels, Belgium
  • International Alliance Of Patient'Organizations, London, United Kingdom
  • Muscular Dystrophy Group Of Great Britain And Northern Ireland, London, United Kingdom
Third parties
  • International Strategic Marketing Services (Isms), Merksplas, Belgium
Project leader
Conny Berlin
Novartis Pharma AG
General questions
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