Shake it off and shake it up

I’ve been working as a Patient Engagement Professional since 1996. This has included working for industry, for healthcare agencies, for universities and – less frequently – for Patient Advocacy Groups (PAGs). At this point, I’d like to differentiate between a disease area summit and a patient summit … the latter always being led by patient groups. At the risk of sounding dismissive, I’ve genuinely lost count of the number of disease area ‘Summits’ I’ve attended; this includes sessions I’ve even presented at or have chaired. But one thing that is a common link in all of these is that the agenda and content has always been set by the industry ‘host’.

Until now.

Specifically, until the PBC Summit that I was invited to ‘observe’ in Edinburgh last week.

This was the first time I’ve been to a therapy area summit where 22 different PAGs attended, 8 pharma companies took a step-back to let the patients set the agenda; and HCPs joined in so that all three stakeholders could adjust the agenda as the need emerged. In reality, such flexibility could only be achieved as a result of being led by the PAGs. Of course, there was a comprehensive content backbone (this was a very well-organised meeting; key discussion points are listed below) – but the atmosphere and the culture of co-creation and no-hierarchy was spectacular. As an ‘observer’ – this was my biggest take-home; when we let PAGs lead then listening, and learning, is guaranteed.

To observe or not to observe; that is the question!

Anyone who knows me will understand how difficult it is for me to ‘observe’ anything; such is my personality and professional experience of always trying to amplify the patient voice which usually involves raising my voice a little too much! Indeed, just an hour into the three-day summit and the Chair (the indomitable Robert Mitchell-Thain) had to send me a message asking if we needed to discuss what ‘observer’ meant! This was meant with kindness and humour of course, but it highlighted the escalating excitement that was generated in every session, in every room, in every collaboration as the summit progressed. It was excruciating as an expert in patient engagement to see authentic co-creation and patient-led objective setting in progress and not join in! Obviously, I managed to ‘assert’ (blurt!) about my pet subjects (evidence-based advocacy, psychotherapeutic support and the power of patient organisations and patient-generated data) when I was supposed to be just listening! So it was legitimate to be asked to possibly pipe-down a little. Completely fair.

In any case; anyone who knows of the dynamic between myself and Robert knows full well that we have always had the confidence to be able to question each other and our behaviours in the parallel roles we have to highlight unmet patient needs – whatever the consequences or criticism of our communications methods (‘outspoken’) from the organisations we work with or for. In short – I’ll always take a challenge from someone so committed to making the right changes in our legacy healthcare systems. Afterall, it is the role of a CEO and a CPO to always question antiquated thinking and one another. (And to suggest that other voices in the room need to be heard!).

This is especially important when it comes to discussing the business objectives of industry, the regulatory hoops industry has to jump through and the role of PAGs to speak up when these objectives collide to the detriment of unmet patient need – an issue the PBC PAGs are tackling directly with their call to remove the need for follow-on placebo-controlled trials for marketing authorisation. These are the kind of big issues that, like this Summit, also have to be led by the PAGs.

The new guardrails for industry-PAG partnerships

Determining the exact number of summits led by patient organisations globally is challenging due to the vast and diverse nature of such events. However, numerous examples illustrate the significant role these organisations play in convening summits focused on patient advocacy, safety, and empowerment.

Notable Patient Organisation-led summits include:

European Cancer Patient Coalition Summit
This summit, held in Brussels, brings together patient organisations to address disparities in cancer care across Europe, focusing on sharing best practices and influencing research priorities.
Make 2nds Count Summit
Organised by the UK-based charity Make 2nds Count, this summit in Liverpool is dedicated to individuals living with secondary (metastatic) breast cancer. It offers tailored sessions with medical experts and resources to support patients navigating complex medical information.
The Flow Summit '24
A collaborative event held in London, combining the Patient Centred Flow Summit and the Patient and Carer Congress. It brought together patients, clinicians, and NHS providers to discuss patient-centred care and system improvements.
North East London Mental Health Summit
This summit was organised by mental health service users with support from NHS trusts, focusing on transforming mental health services by prioritising the voices of those with lived experience.
14th Annual Patient Safety Summit
Hosted by the Royal Society of Medicine, this UK-based summit featured contributions from patient safety advocates, clinicians, and students, highlighting the importance of patient safety and clinician well-being.

The PBC Summit built on this and added icing on the cake was creating a ‘Lion’s Den’ set of sessions for participants to question industry directly. However – in a cautionary nod to the ABPI code which prohibits industry ‘promoting to the public’ I did not attend these sessions. But my industry peers and PAG colleagues confirmed that they found these to be highly insightful parts of the event – people talking directly to other people about how to create enduring patient solutions. Imagine that! This is truly progressive patient engagement in practice. We can be certain that this is a great step to removing the guardrails that no longer serve purpose and building new relationships with PAGs – that are based on trust, integrity and patient-relevant outcomes.

Conclusion

While an exact global count of patient-led summits is not readily available, these examples demonstrate that patient organisations are instrumental in leading and organising summits that address various aspects of patient care, safety, and empowerment. Their efforts are crucial in fostering collaboration and advocating for patient-centred healthcare improvements.

Which is why this Summit was so provocative, productive and progressive. It was led by the PBC Foundation which is led by Robert. He is the beating heart of the Foundation and the Foundation is a global conscience for all professionals (pharma, physicians and patients) determined to bring a new way of collaborating to be the normal.

I’m already eager to attend next year’s PBC Summit – although I’m not sure they’ll have me back as an ‘observer’! One way or another, I’m sure the CEO will let me know.

PBC Summit 2025: Key Themes & Messages

Global Collaboration & Localism

· First truly patient-led international PBC summit; a milestone for patient leadership in rare diseases.

· Creation of a positive agenda for PBC, learning from international best practices to inform national and regional approaches.

· Localism emphasised — adapting global insights to local realities, including:

A) Developing local tools to guarantee treatment access.

B) Recognizing PBC as an autoimmune condition and actively screening for comorbidities.

Evidence & Policy Change

· Insurance and reimbursement systems still prioritize cost over individualized care.

· Strong call for evidence-based advocacy beyond pharmaceutical therapies, especially around symptoms like fatigue.

· Regulators (FDA/EMA) urged to modernize frameworks to include real-world evidence (RWE), patient-reported outcomes (PROs), and long-term observational data.

· Notable proposal: 5-year follow-up observational study comparing outcomes in placebo vs. drug arms post-trial.

Patient-Centered Innovations

· Project 90:90 -- Grounded in 90% early diagnosis and 90% appropriate treatment.

· One-stop-shop for PBC services.

· LFT (liver function test) automation and reflex testing integration into EMRs.

· Use of digital symptom trackers linked with clinician tools.

· Engagement of LIMS vendors and HCPs for implementation.

Fatigue & Underappreciated Symptoms

· Fatigue in PBC is prevalent and often misunderstood—on par with end-stage cancer in severity.

· Call for:

A) Standardised fatigue assessment tools in autoimmune diseases.

B) Use of the Clinician Global Impression Scale (CGI) to capture physical, cognitive, and emotional impact.

Mental Health & Psychological Harm

· High burden of psychological harm due to uncertainty and lack of specialist support.

· Proposal to develop a self-care toolkit and culturally appropriate wellness programs (e.g., the Canadian model).

· Emphasis on working with clinical psychologists and embedding mental health in care pathways.

PBC Patient University

· Education and empowerment program for patients to become scientific, regulatory, and communications advocates.

· Skills training in writing, presentation, and stakeholder influence.

· Seen as a pathway into broader regulatory advocacy (e.g., lobbying FDA/EMA).

Research, Data & Real-World Evidence

· Develop centralised, interoperable repositories (e.g., 90/90 registry, digital repositories).

· Harmonize endpoints, encourage prospective data collection.

· Need for endorsement by professional societies and inclusion in regulatory frameworks.

Stakeholder Engagement & Communications

· Importance of unified and coherent communication between patient groups.

· Regular forums to share global best practices, with:

A) Clear agendas.

B) Rotating leadership.

C) Shared resource libraries.

D) Aligned press releases and public statements.

The Summit of Summits: Removing the ‘Guardrails’ Between Industry and PAGs