Patient involvement in medicines R&D – no longer why, but when, where, why and how?

Jan Geissler, Coordinator of the EUPATI Futures Team and PFMD Management Group member
Despite growing and widespread agreement that patient involvement is essential in medicines R&D if health interventions are to meet patients’ needs and priorities, practical guidance on when, where, why and how to integrate patient experts in a meaningful way is still rare. To tackle this, I have been deeply involved in two key initiatives – the European Patients’ Academy (EUPATI) as a patient-driven, bottom-up educational movement to increase the knowledge and expertise of the patient community in medicines R&D, and PFMD as a systematic, top-down collaboration that is co-creating a systematic meta-framework for patient engagement and facilitating best-practice sharing. Both work hand-in-hand to drive patient engagement and its implementation in medicines development.
The European Patients Academy (EUPATI) was set up in 2012 as a IMI-funded, patient-led public private partnership to increase the capacity and knowledge of European patient advocates about the whole medicines development process. It has developed and conducted two Patient Expert Training Courses which has already trained 96 patient experts– and these “EUPATI Fellows” are today creating impact in more than 50 disease areas for example as speakers at conferences, as advisors in investigator meetings or as members of regulatory committees. 60 more trainees are about to start with the third EUPATI course in summer 2017. EUPATI has also deployed a 9-lingual Toolbox which explains the drug development lifecycle from pre-clinical research over clinical trials, pharmacovigilance, risk/benefit assessment, regulatory affairs and health technology assessment. More than 160,000 individuals have so far used the EUPATI Toolbox since its launch in January 2016, demonstrating the huge demand of the patient community to receive education and training. EUPATI has actually grown the key resource needed for true patient engagement: educated patient advocates who, over and above their disease experience, have the technical skills to contribute and create impact in R&D.
Initiated by a global roundtable of patient advocates and industry experts in medical R&D, PFMD was established in 2015 to meet the need to co-create a meta-framework with associated tools and resources for integrating the patient perspective across all phases of medicines development. This is not about re-inventing the wheel – rather it’s about working in multi-stakeholder groups and drawing on experience and good practices that are out there and taking a collaborative approach to fill in the gaps. One of the challenges for patient involvement is that although there are many valuable workshops and outputs, little is formally documented and shared, hampering our ability to learn from these efforts. This is what PFMD is determined to change.
The recent publication of “Improving Patient Involvement in Medicines Research and Development: A Practical Roadmap”, authored by me and Bettina Ryll as experienced patient advocates as well as Susanna Leto di Priolo and Mary Uhlenhopp with huge industry experience, is now connecting the dots: the key phases of the drug development life-cycle and its regulatory steps, and practical elements where and how patients can be meaningfully involved in those. The Practical Roadmap is the result of a series of multi-stakeholder meetings like EPALCO, EUPATI workshops and workgroup discussions during 2013-2016 and crystallizes some of the key themes from these rich discussions on how to capture and integrate the patient voice across the medicines R&D pathway, complemented by practical experience where tangible patient involvement became reality in R&D projects or regulatory processes.
The rational approach to development of the Roadmap is very much aligned with PFMD’s approach to meta-framework co-creation. The authors reviewed existing patient involvement material to identify key stages in R&D, specific opportunities for patient involvement at each stage and existing patient involvement guidance and strategies at each opportunity. Workshops and meetings helped to collect experience in guidance/strategies and we pulled all of these together into a roadmap across the R&D lifecycle. The publication of the Roadmap also provides concrete examples of specific patient involvement activities aligned to different stages of medicines R&D, giving practical ‘how to’ illustrations.
Patient involvement in medicines R&D is a complex and ambitious undertaking – but has been reality in many disease specific communities, specifically in HIV or rare disease, already for a long time. However, it is not yet implemented in a systematic way across diseases, stakeholders and regions – but that is changing. The building blocks for a new, patient-centric research reality are coming together nicely now: the meta-framework and implementation resources being developed by PFMD will help to synergise and shape the system. Tools such as the Roadmap and EUPATI’s Guidance documents help to guide practical implementation of patient involvement in the medicines development lifecycle and regulatory affairs. PFMD’s SYNaPse global mapping and networking tool enables systematic sharing and measurement of best practice examples. And EUPATI’s Toolbox and Patient Expert Training Course empower patient advocates that are educated to be engaged in a meaningful way.
Regulators, academic groups, patient organisations and industry researchers are using those tools to add value in their efforts towards consistent and meaningful patient involvement in medicines development and lifecycle. For example, the Roadmap has been used widely across the EUPATI Toolbox material to illustrate patient involvement at different stages of medicines research and development. It has been presented and used at various conferences, training meetings and workshops of both industry, academia and the patient community. It is also a key element of EUPATI’s Guidance documents on the interaction with industry, ethics committees, regulators and HTA bodies.
As a founding member of both PFMD and EUPATI, seeing how disparate but complementary efforts and resources are now coming together as we connect the patient engagement landscape is hugely rewarding.