What’s needed versus what’s out there – surveys and gap analysis
The project partners first worked to gather the perspectives, needs and expectations of all stakeholders involved in medicines development by carrying out surveys and focus group consultations. The results of this research were then used to develop a set of criteria for assessing whether patient engagement (PE) needs were being met. These criteria were developed using a process called the DELPHI technique which consists of multiple rounds of questions involving expert panels. Following this step, the partners carried out a gap analysis whereby they measured the extent to which those needs, expectations and preferences for effective PE were being met in reality.
In total, 70 PE initiatives from existing international databases were gathered and consolidated, and 16 specific gaps across nine of 14 overarching themes were identified. This allowed them to create metrics and tools that can increase the evidence demonstrating the impact of patient engagement practices.
The work done in identifying stakeholder needs and expectations, the creation of criteria to assess patient engagement practices, and the gap analysis resulted in a specific set of tools. These tools are freely available online in the PARADIMG PR Toolbox for others to use. They are categorised into three sections: planning PE, conducting PE and reporting and evaluation of PE activities. Some of the tools address needs at one of the three specific decision-making points and others have broader applicability. The toolbox centralises PARADIGM’s recommendations, tools and relevant background information in one place in order to effectively facilitate their dissemination by the consortium partners and their availability through external partners and supporters.
For example, the gap analysis revealed the need for a standardised set of rules for PE that covers practical guidelines, common values, ethical principles and rules for the collaborating partners. The PE Toolbox contains the Code of Conduct that was drawn up as a result of this gap, and is intended as an easy-to-follow guide that answers to this need, describing the essentials for meaningful collaboration of all stakeholders involved in PE activities in medicines development.
The Toolbox also includes guidelines on managing competing interests in a multi-stakeholder environment, covering conflicts of interest and risk mitigation strategies, and an updated version of the guidelines for industry that were originally drawn up by IMI patient education project EUPATI. It also includes guidelines on the contents of contracts signed by both parties (covering consultancy, collaborations, speaking engagements, advisory boards etc.) and how to find the right match when choosing the right patient for the right PE activity.
The Patient Engagement Monitoring and Evaluation Framework
Capturing the ‘return on engagement’ is complex, given the many factors that influence the impact of patient engagement. This Patient Engagement Monitoring and Evaluation Framework, with metrics, was created to help partnerships between patients and/or patient organisations, bio-pharmaceutical companies, regulators and health technology assessment (HTA) bodies to self-evaluate the progress and impacts of PE in the medicines development lifecycle for all stakeholders involved.
The objective of this tool is to support learning to facilitate meaningful PE, helping users to understand the pathway to impact of PE and demonstrate better decision-making in medicines development.
During the lifetime of the IMI PARADIGM project, three Patient Engagement Open Fora (PEOF) were organised jointly with EUPATI and Patient Focused Medicines Development (PFMD). The events were an opportunity to collaborate and discuss tools and recommendations for effective PE, methods for monitoring and evaluation of impact and outcomes in patient engagement activities and fair market compensation for patient input. The events included interactive sessions on assessing good practices in PE. The PEOF continues, after PARADIGM ended, co-powered by the European Patients’ Forum, EUPATI and PFMD, supported by corporate partners.
PARADIGM International Liaison Group
The PARADIGM International Liaison Group (PILG) was created as an exchange and co-creation hub to connect PARADIGM with like-minded initiatives. The PILG allowed for collaboration that helps reduce fragmentation and duplication, and provided external guidance based on the work of the project.
The IMI-PARADIGM consortium also developed a roadmap for others to support optimal PE in key decision-making points across medicines development process, and to ensure long-term use of the resources developed during the project. The roadmap is based on three dimensions that make PE sustainable, i.e. culture, processes and resources. Anyone involved in the lifecycle of medicines, i.e. patients and patient organisations, medicines developers, academia, regulatory authorities, health technology assessment bodies, payers, policy makers and public research funders can use the roadmap from the perspective of their own stakeholder group and each action is adapted to the perspective of one, more than one, or all stakeholder groups
Key publications and outcomes
- Evaluating the "return on patient engagement initiatives" in medicines research and development: A literature review
- Evaluation of patient engagement in medicine development: A multi-stakeholder framework with metrics
- Understanding multi-stakeholder needs, preferences and expectations to define effective practices and processes of patient engagement in medicine development: A mixed-methods study
- Sustaining Meaningful Patient Engagement Across the Lifecycle of Medicines: A Roadmap for Action
Achievements & News
IMI project PARADIGM has launched its Patient Engagement Toolbox that brings together in one place all the project’s recommendations, tools and relevant background information to make patient engagement in medicines development easier for all. ###The toolbox is the result of a huge co-creation effort involving extensive research, surveys, focus groups, case studies, workshops, and multiple rounds of reviews.
The tools are divided into three groups: planning patient engagement; conducting patient engagement; and reporting and evaluation. Each tool comes with information detailing how the tool was produced and who contributed to its creation.
‘To make patient engagement easier for all in medicines development, several stakeholders need to work together, to come up with the most suitable solutions for all parties involved,’ says PARADIGM coordinator Mathieu Boudes of the European Patients’ Forum (EPF). ‘At PARADIGM, we’ve been extremely grateful to be able to develop the Patient Engagement Toolbox with the funding scheme of the IMI, as a true public-private partnership. To me, without the IMI, this would not have been possible’
PARADIGM is drawing to a close, but the Patient Engagement Toolbox will be maintained and accessible via the EUPATI and PFMD (Patient Focused Medicines Development) platforms.
Find out more
The Patient Engagement Open Forum is organised jointly by IMI’s PARADIGM and EUPATI projects as well as Patient Focused Medicines Development (PFMD). ###The aim of the event is to provide a holistic perspective of patient engagement, the landscape and actors, and foster collaboration and co-creation while breaking down fragmentation and silos that are often present in patient engagement work.
This year, the event is going virtual, and will be a series of short (under two hours) online events running from June to November. Events will be recorded and published online.
Topics range from tools and recommendations for effective patient engagement, methods for monitoring and evaluation of impact and outcomes in patient engagement activities, and fair market compensation for patient input, to interactive sessions on assessing good practices in patient engagement and more.
Find out more
- Visit the event website
There is now growing recognition that patients and their representatives can and should be involved in all stages of medicines development (R&D). But how do you do this in practice, and is it worth it? IMI’s PARADIGM project was set up in 2018 to create a framework for structured, effective, meaningful and ethical patient engagement, and to demonstrate the ‘return on engagement’ for all stakeholders.###
The project focuses on patient involvement in three key points in the medicines R&D process: research and priority setting; clinical trial design; and early dialogues with regulators and health technology assessment (HTA) bodies. So what have they delivered so far? With just months to go before the end of the project in August this year, the IMI Programme Office caught up with project coordinator Mathieu Boudes of the European Patients’ Forum (EPF).
‘PARADIGM is an atypical project – very short and most of the tangible achievements are due at the end of the project,’ explains Boudes. ‘We have robustly identified what is still very much needed by all actors involved in the development of medicines and that will make the most impact in terms of patient engagement. The consortium is currently developing a monitoring and evaluation framework for stakeholders who are doing patient engagement to help them measure the impact of their initiatives. It will be a game changer to bring patient engagement to the next level.’
On 18-19 September, IMI projects PARADIGM and EUPATI are joining forces with Patient Focused Medicines Development (PFMD) to organise the Patient Engagement Open Forum in Brussels, Belgium. ###The aim of the event is to provide a holistic perspective of patient engagement, the landscape and actors, and foster collaboration and co-creation while breaking down fragmentation and silos that are often present in patient engagement work. The agenda offers a deep dive into some ongoing patient engagement work done by many collaborative initiatives. Topics range from tools and recommendations for effective patient engagement, methods for monitoring and evaluation of impact and outcomes in patient engagement activities, and fair market compensation for patient input to interactive sessions on assessing good practices in patient engagement and more. Registration is free but obligatory.
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.’
ParticipantsShow participants on map
- Alexion Services Europe SPRL, Brussels, Belgium
- Amgen Limited, Cambridge, United Kingdom
- Bayer Aktiengesellschaft, Leverkusen, Germany
- Eli Lilly And Company LTD, Basingstoke, United Kingdom
- F. Hoffmann-La Roche AG, Basel, Switzerland
- Federation Europeenne D'Associations Et D'Industries Pharmaceutiques, Brussels, Belgium
- Glaxosmithkline Research And Development LTD., Brentford, Middlesex, United Kingdom
- Grunenthal GMBH, Aachen, Germany
- H. Lundbeck As, Valby, Denmark
- Institut De Recherches Internationales Servier Iris, Suresnes, France
- Janssen Pharmaceutica Nv, Beerse, Belgium
- Labcorp Early Development Laboratories Limited, Harrogate, United Kingdom
- Merck Kommanditgesellschaft Auf Aktien, Darmstadt, Germany
- Merck Sharp & Dohme Corp, Whitehouse Station, New Jersey, United States
- Novartis Pharma AG, Basel, Switzerland
- Novo Nordisk A/S, Bagsvaerd, Denmark
- Pfizer Limited, Sandwich, Kent , United Kingdom
- Sanofi-Aventis Recherche & Developpement, Chilly Mazarin, France
- The Association Of The British Pharmaceutical Industry, London, United Kingdom
- UCB Biopharma, Brussels, Belgium
- Verband Forschender Arzneimittelhersteller Ev, Berlin, Germany
Universities, research organisations, public bodies, non-profit groups
- Agenzia Italiana Del Farmaco, Rome, Italy
- European Forum For Good Clinical Practice, Brussels, Belgium
- Forum Des Patients Europeens, 1040, Belgium
- Fundacio Privada Per A La Recerca I La Docencia Sant Joan De Deu, Esplugues de Llobregat, Spain
- Instituto Aragones De Ciencias De La Salud, Zaragoza, Spain
- Stichting Vu, Amsterdam, Netherlands
- University of Oxford, Oxford, United Kingdom
Small and medium-sized enterprises (SMEs) and mid-sized companies (<€500 m turnover)
- Health Technology Assessment International Society, Edmonton, Canada
- Team - It Research SL, Barcelona, Spain
- The Synergist, Bruxelles, Belgium
- Alzheimer Europe, Luxembourg, Luxembourg
- European Aids Treatment Group Ev, Duesseldorf, Germany
- Eurordis - Rare Diseases Europe, Paris, France
- Synergist Services, Bruxelles, Belgium
|Name||EU funding in €|
|Agenzia Italiana Del Farmaco||50 000|
|Alzheimer Europe||248 500|
|European Aids Treatment Group Ev||381 750|
|European Forum For Good Clinical Practice||198 000|
|Eurordis - Rare Diseases Europe||463 598|
|Forum Des Patients Europeens||702 516|
|Fundacio Privada Per A La Recerca I La Docencia Sant Joan De Deu||176 125|
|Instituto Aragones De Ciencias De La Salud||181 086|
|Stichting Vu||458 438|
|Synapse Research Management Partners SL (left the project)||399 595|
|Team - It Research SL||73 903|
|The Synergist||125 374|
|University of Oxford||621 593|
|Name||Funding in €|
|Synergist Services||418 455|
|Total Cost||4 498 933|