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