‘Patient engagement in drug development: moving beyond tools and standardization?’ Overview of a multi-stakeholder dialogue

Claudia Egher

InPart Postdoctoral Researcher

Dec 12, 2022

On November 18, Dr. Claudia Egher together with the InPart team organized the multi-stakeholder hybrid workshop “Patient engagement in drug development: moving beyond tools and standardization?” at the Maastricht University Campus Brussels. The goal of the event was to stimulate dialogue on patient representation in drug development, on the performative effects of the tools and materials that are currently available in this area, and on how to ensure their broad and responsible dissemination. The participants included patients, patient advocates, patient education providers, members of the pharmaceutical industry, of multi-stakeholder groups active in the field of patient engagement, researchers, and students.

The workshop included different types of activities: academic presentations, vignette-based discussions, and a group-based mapping of the current and ideal drug development landscapes through the use of objects with different sensorial qualities. This post focuses on the content shared during the presentations and during the ensuing dialogue among the participants. During the first part of the workshop, I presented my findings based on the thematic analysis of three different types of data: (1) guidances and materials on patient engagement in drug development put forward by the Patient Focused Medicines Development (PFMD) multi-stakeholder group, the U.S. Food and Drug Administration Agency (FDA) and the European Medicines Agency (EMA); (2) 39 semi-structured interviews with patient advocates, patient experts, regulators, members of public-private groups active in the field of patient engagement, and pharmaceutical industry professionals; (3) online participant observations of the Patient Engagement Open Forum (PEOF) sessions held between 2020-2022. The presentation of the findings was structured in two parts.

In the first part, I argued that the materials described above under (1) and (3) have performative effects, that they actively construct the future of patient engagement in drug development. The materials analyzed seek to ensure that patient engagement in drug development becomes a widespread reality by arguing that the needs and preferences of patients should be the starting point for the development of any new medicine. To achieve this, these materials put forward new roles for the main actors in this field. More specifically, they envision patients as representatives and research consultants, they envisage drug development teams as hospitable hosts to patients, and patient organizations as highly influential mediators.

In the second part of her presentation, based on the analysis of all three types of data, I revealed that numerous epistemic requirements are placed upon patients who want to represent broader groups in drug development. Thus, patients are expected to be endowed with three types of knowledge - autobiographic knowledge, community knowledge, drug development knowledge- and with adaptive skills, to perform different types of representation work. The findings presented indicated that variations in the level of any of these types of knowledge and in one’s adaptive skills are linked to differences in the complexity of the representation work patients are thought to be able to perform. Thus, patients endowed with higher levels of knowledge are expected to perform more substantial and challenging representation work in drug development.

In the ensuing dialogue, the participants agreed that care needs to be taken to avoid the development of a situation, where patient representatives bare most of the collective efforts required for patient engagement in drug development, where they undergo the more substantial transformations, whereas minimal changes are brought to the practices of other stakeholders. The participants further voiced the concern that these expectations may constitute attempts by the pharmaceutical industry to shift some of its responsibilities onto patient representatives. Overall, three main risks were mentioned in regard to patient engagement in drug development: (1) that long established inequalities and forms of exclusion will be perpetuated; (2) that cultural and geopolitical differences will continue to be disregarded; (3) that patients will be ‘locked’ in roles which are largely pre-defined by others, with only minimal recognition and reimbursement for the work they do.

Some of these risks were developed further by Dr. Katherine Deane, an academic and patient advocate, in her presentation on key accessibility issues in patient engagement in drug development. Dr. Deane emphasized the fact that whereas physical and mental ill-health conditions are widespread, the spaces where patients are invited to share their views and insights are largely designed for healthy, able-bodied individuals. She also drew attention to the importance of intersectionality, as various identity aspects, such as a patient’s sex, gender, ethnicity, along with the social and cultural understanding of the condition with which s/he is diagnosed may further the exclusion and stigmatization s/he experiences. Even though research and drug development environments are often designed in ways that are hostile to patients, Dr. Deane reminded the participants that access, engagement in drug development and other healthcare activities, is a right.

The final presentation of the workshop was given by Dr. Olga Zvonareva, the Principal Investigator in our project, and it focused on the invisible work necessary to make tools and standards work across different settings. Dr. Zvonareva showed that patient engagement activities are unequally spread across the globe. Whereas such initiatives are starting to be more frequently encountered in the U.S. and in Western European countries, they are largely absent from South America and Africa. In Dr. Zvonareva’s view, this uneven distribution is not necessarily worrying for the time being, as innovations take time to spread. She warned, however, that it is important to find the right balance between standardization and flexibility, between the need for systematic approaches and local adaptations, to ensure that innovations such as patient engagement in drug development, are broadly adopted.

At the end of the workshop, the participants shared their views on what still needs to happen to ensure that patient engagement in drug development becomes a widespread reality. Thus, they highlighted the urgent need for quantitative and qualitative studies on the impact of patient engagement in drug development. They also stressed the importance of conducting robust research into how the currently available tools and guidances on patient engagement are implemented across different settings, and argued that best practices should be frequently shared and updated.

The workshop ended with an open invitation for further and new collaborations, for enlarging the circle of people who seek to ensure that the patients’ needs and preferences become part of the drug development process from the very early stages. Whereas the stakeholders who have already played a considerable role in furthering patient engagement in drug development need to continue their efforts, stakeholders who unti l now have only marginally been involved, such as policy-makers, are warmly invited to delve deeper into this fascinating and hope-inducing set of practices.