Is measuring the impact of patient engagement activities in medicines R&D, a way to sustain the cultural change for more systematic and meaningful patient engagement? We asked this question during a session at the DIA Global Annual Meeting 2019 in San Diego earlier this summer

Author: Mathieu Boudes, European Patients’ Forum and PARADIGM Coordinator

There is an increasing belief that measuring the impact of engagement is crucial to supporting and sustaining patient engagement in medicines R&D. Despite a growing interest in assessing the impact of patient engagement, little is known about ‘what’ to evaluate and ‘how’ to best evaluate patient engagement. At the Global DIA meeting, several speakers provided insights on this topic, as the panel teased out how evaluating patient engagement initiatives can be the key to unlocking their sustainability. 

There is no universally agreed definition of what sustainability means, indeed, there are many different views on what it is and how it can be achieved. The idea of sustainability stems from the concept of sustainable development which became common language at the World’s first Earth Summit in Rio in 1992. The original definition of sustainable development is usually considered to be:

“development that meets the needs of the present without compromising the ability of future generations to meet their own needs,” according to the Bruntland Report for the World Commission on Environment and Development in 1992. Since then, there have been many variations and extensions on this basic definition. It is commonly accepted that sustainability is the process of people maintaining change in a balanced environment, in which the exploitation of resources, the direction of investments, the orientation of technological development and institutional change are all in harmony and enhance both current and future potential to meet human needs and aspirations. For many in the field, sustainability is defined through the following interconnected domains or pillars: environment, economic and social.

In his presentation, Nicholas Brooke, PFMD Chief Executive, stated that when it comes to patient engagement in medicines R&D, it appears clear from many surveys or focus groups conducted by several initiatives (such as PFMD) across stakeholders, diseases and countries that measuring the impact of an activity in its ecosystem is of the utmost importance. 

For example, the DIA conducted a two-phase study with Tuft University which showed that being able to measure the impact of a patient-centric initiative impacts its implementation within biopharmaceutical companies, Debra Michaels from DIA explained.  

Furthermore, Jaye Bea Smalley, from Celgene and the Clinical Trials Transformation Initiative, explained that while there is growing evidence that patient engagement provides a substantial financial return on investment to research sponsors, both sponsors and patient groups think much more broadly about the true value of engagement. ‘Whether’ to engage should no longer be a question; what’s most important to ask, given the limited resources, is which engagement opportunities will provide all stakeholders with the greatest value. This is a question best answered through collaboration and discussion, she stated. Determining this is now possible with the Prioritization Tool for Sponsors and Patient Groups developed by CTTI and presented at the DIA Global Meeting.

Researcher and Lecturer Meaningful and Sustainable Patient Engagement at the Vrije Universiteit in Amsterdam Lidewij Vat added that while standardised metrics may be appealing, the complex and contextually-dependent areas of patient engagement should not be overlooked and therefore she recommended discussing relevant and feasible metrics per setting with all stakeholders involved. Due to the before-mentioned complexity, the value of patient engagement initiatives can best be measured by using a coherent set of metrics; taking inputs, processes, learnings and changes, impact and the context into account.

The panel discussion was followed by an interactive audience Q&A session. One audience member highlighted how the purpose of evaluation may differ per stakeholder group. For example, for researchers and industry partners, it might be about evidence that patient engagement improves the quality and efficiency of research, while for patients it might be more about influencing the R&D agenda to develop medicines for unmet needs. Showing these benefits may be an effective motivator for patients and companies and thereby help to sustain the cultural change. Some argue that evidence is needed to justify the ‘business case’ for engagement. This could help to establish a financial model to support engagement, addressing the economic feasibility aspect of the sustainability pillars.

Altogether, it appeared clear that the development of co-designed metrics to measure the return on engagement is one tool within a large toolbox that collectively will underline the cultural change needed to make the engagement of patients in medicines lifecycle a ‘no-brainer’ and a business decision as fundamental and as normal as others. 

PARADIGM is at work producing a monitoring and evaluation framework taking into account the needs of all stakeholders so that it can be widely used in a sustainable fashion.