EMA patient experience workshop: let’s use existing multi-stakeholder platforms to accelerate action

The European Medicines Agency is heading in the right direction on patient engagement and patient experience data. And we can progress faster if we make the most of existing multi-stakeholder collaborations and build on the groundwork that has already been laid.?

Let me explain.?

Good news! The last multi-stakeholder workshop held by the EMA before the pandemic was on patient engagement. The first one the Agency hosted when face-to-face meetings returned after the pandemic was focused on patient experience data .

This, to me, is a strong signal that the EMA is taking patient experience data and patient engagement seriously. It sees the value ? and, I might say, necessity ? of moving forward in this field. More than that, the regulator brought together a strong group of contributors to drive the agenda and offered insights on its own work in this area.?

I was pleased to hear these diverse perspectives, including from patient groups, rare disease advocates and industry, alongside Emer Cooke , Executive Director of the EMA, and Harald Enzmann, chair of the EMA’s key medicines committee (CHMP). [Enzmann’s comments on how working with patients can be a mutually enriching experience was a highlight for me]

As well as highlighting the many ways in which EMA is moving patients to the centre of its future work, Emer Cooke also noted that patient engagement is not yet systematic ? nor is PE embedded across the lifecycle of medicines development.?

To me, this acknowledgement is as important as any of the welcome initiatives that EMA is already taking. Why? Because it acknowledges that there is still a long way to go before we get to where we all agree we need to be.?

The question then must be: what are we going to do about it??

Notwithstanding its current efforts, the EMA has not yet issued guidance on what exactly is expected from medicines developers regarding patient engagement as part of marketing authorisation. The idea of a guidance was mentioned several times by EMA experts. Still, it's not easy to prescribe how patient engagement should be done and what patient experience data should be generated. [The FDA, in contrast, has taken the lead by issuing guidance already, with a focus on data and clinical outcomes assessment (COA).]

The workshop was also an opportunity to iron out some of the conceptual issues holding back progress. For example, the stated goals of the meeting were to achieve a common understanding on what constitutes ‘patient experience data, including patient engagement, patient preferences and patient-reported outcomes’.?

To me, this frames the issue incorrectly.?

Patient engagement (PE) is not a branch of patient experience data (PED): the first is an activity that, if done properly, leads to more meaningful and better representative data for decision making. (This was tested out at a recent ISPOR webinar which aimed to define, and distinguish between, patient preferences, patient-reported outcomes, and patient engagement.) We cannot have impactful, meaningful PED without PE from the start. This represents a more general challenge: the need to integrate patient input from the start and throughout the process ? not just at the end.

This point was made by Kaisa Immonen (EPF) and Yann Le Cam (EURORDIS), two of the most knowledgeable patient advocates you could find. Patients need to be included early and often. Indeed, the system needs patient input from the outset. Otherwise, how are we selecting which PED to prioritise? What experiences matter? Which preferences count? It was proposed to evolve from patient experience data to patient experience evidence. Their presentations advocated for qualitative engagement together with data design and generation, as well as the processes and resources to deliver it, for patients and for all stakeholders.

It was also repeated that this approach should include all health stakeholders from early researchers to clinical practices and the potential key role for EMA to lead beyond regulatory. HTA and payers were mentioned numerous times.

Many of us have, for years now, taken part in meetings on patient engagement which conclude with a call for early multi-stakeholder alignment WITH patients to define the right data to collect and the best methods for collecting it.

Of course, nobody could disagree ? we are all for it.?

It’s 2022 and I see this meeting as a post-Covid reboot establishing a solid base to bring patient engagement to the next level ? this is not the beginning of the process. It’s the middle.

We must join forces to avoid a fragmented approach that can only generate fragmented results.?

Speakers and participants mentioned numerous times the need for multi-stakeholder platforms to take us to the next level. These platforms currently exist and I am looking forward to bridging EMA and other regulatory initiatives with those. These patient-centered initiatives beyond one stakeholder group, have already started to answer questions raised during the workshop.

For example, there are 37 stakeholders from both sides of the Atlantic representing patients, industry, regulators and others, working on a PFMD initiative to ‘Harmonize and synergize PE and PED to deliver improved patient-centred impact ’.

Together, this group is mapping the work of regulators and HTA bodies on PE and PED. It is co-creating a global navigator, developing a roadmap for the future, and developing a Call to Action designed to move us forward.?

This is not the only multi-stakeholder effort co-writing the following chapters in this story.?

Several key players have come together recently to draft the Patient Engagement Action Plan ? a 7-step proposal that digs into the details of how to improve the use of meaningful PED in drug development and decision-making.?

I would also recommend reading Jan Geissler’s excellent reflections on the event and the examples he mentions.

In short, we are past describing the problem and need to continue taking action ? building on the work that currently exists. If I may use a medical analogy, we are beyond diagnosis and have started intervention and together we will provoke more impactful lasting change for patients and health systems.

I was really energised by the EMA Amsterdam meeting ? not just by seeing familiar faces ? but by a well-organised event that highlighted our shared desire to make PE a priority. I feel a renewed sense of commitment and energy amongst stakeholders that was reignited by the EMA workshop.

Yes, there are still challenges. The good news is, we already have the basis for how to tackle them together.

Next milestone, the Patient Engagement Open Forum organized by PFMD .org , EUPATI and the European Patients' Forum . PE champions are gathering to collaborate, share & co-create solutions for better #PatientEngagement practices