Patient engagement in medicines R&D: The need to measure “Return on engagement”


“Patient engagement is currently within the realm of early adopters and innovators; it’s time for it to go mainstream.”

Mathieu Boudes, coordinator of the PARADIGM public-private partnership at European Patients’ Forum (EPF), discusses the consortium’s efforts to create a structured framework for patient engagement in medicines R&D. He discusses why development of agreed patient engagement metrics will help quantify the benefits of patient engagement to facilitate progress from episodic to mainstream implementation.

Patient engagement at different points of the medicines lifecycle is undisputedly critical to fostering patient access to innovative therapeutic solutions, and delivering better health outcomes for patients. However, the industry still lacks a common approach to engaging patient communities, which makes it especially challenging to determine how patient-centric clinical development really is.


“It’s not always happening systematically across and even within companies, but can be sporadic across regions, often reliant on personal initiatives to drive it forward,” says Mathieu Boudes, coordinator of the PARADIGM consortium (Patients Active in Research and Dialogue for an Improved Generation of Medicines). Boudes, who will be speaking at MAPS 2018 EMEA Annual Meeting taking place in October, adds: “The mission of the consortium, which is led by EPF and European Federation of Pharmaceutical Industries and Associations (EFPIA) and 34 partners public and private partners is to provide a set of practical tools and practices in this regard."

Advancing meaningful patient involvement

The overarching goal of PARADIGM, which is financed within the framework of the Innovative Medicines Initiative (IMI), is to participate in the co-creation of a sustainable framework that will allow ethical, meaningful and systematic patient engagement in medicine R&D. “We want to provide the tools, processes, templates and methodologies that are needed for the community involved to operationalize patient engagement during the lifecycle of medicine by the end of the 30-month lifespan of the project. PARADIGM will focus on three key decision-making points in relation to development of medicinal products – the research priority setting, the design of clinical trials, and early dialogue with regulators and HTA bodies – and also on vulnerable populations (young persons and older people with dementia) and adult patients. The first step is to better understand via a survey the patient engagement expectations and needs from stakeholders involved in the medicines lifecycle. Progress toward a framework for patient involvement requires a collaborative and international approach by all stakeholders,” says Boudes.

Return on meaningful engagement

The value and benefits of patient involvement – better prioritization of early research, improved resource allocation, improved trial protocol designs that better reflect patient needs, often linked to enhanced recruitment and retention – need to be clear and will provide the impetus for improvement. However, universal measures of success have yet to be agreed as an industry and, more importantly, as a community.


“Patient engagement is currently within the realm of early adopters and innovators; it’s time for it to go mainstream. We believe one of the key ways to have widespread adoption is through performance measurement to demonstrate the concrete value of engagement. It’s notoriously difficult to measure – you can tell you recruited a trial faster but this doesn’t tell you the outcome and we’re not sure if it’s only due to patient engagement.”


For Boudes, measurement will lead to wider adoption, as the value will be understood to be measurable. “We will find acceptance and belief in the engagement process and then slowly, we can change the mindset. It will cascade from the top, and the value chain of the development will embed the patient perspective when relevant. But this can’t be done alone and the industry will benefit by working in a pre-competitive space on these tools. It’s what you do with the tools afterwards that may become competitive advantage.”


To achieve these ambitious goals, PARADIGM is joining forces with other patient engagement initiatives, and organized the 1st Open Forum on Patient Engagement with EUPATI (European Patients’ Academy on Therapeutic Innovation) and PFMD (Patient Focused Medicines Development) last April. Metrics that are relevant to each of the stakeholders, who come to the process with often unique needs, is not an easy task. Co-creation is the pathway to sustainability, and finding synergies, while resisting competition is fundamental to success. PARADIGM is yet another stepping-stone on the pathway to evolution of the ecosystem to embed patient engagement as the norm, rather than the exception.

Medical: custodians of the patient voice

According to Boudes, Medical Affairs as the interface between the internal pharma organization and the patient community has a key role to play here. “Medical Affairs has to translate the input from the patient representative within the internal process and vice versa. The dialogue between the internal organization and the patient needs to be efficient and there needs to be alignment in this regard. The main improvement required is early engagement, as it is never too early."


“While both the pharmaceutical industry and regulators have achieved some progress in incorporating patient perspectives into their activities, the lack of a standardized framework and metrics has made it challenging to achieve consistency and measure success in patient engagement. We need to change the mindset and prove engagement works, otherwise we’re going to miss out on this opportunity that was created through decades of advocacy work by the patient groups. Working with patients creates value for the whole healthcare ecosystem and that’s in everyone’s interests, and this is embedded in EPF’s six strategic goals.”