Patient engagement professionals need to have a powerful mix of compelling business cases and personal passion – both of which featured strongly at this jam-packed conference. Read on to learn what you need to know from Day One...

Keynote panel discussion: case studies

The conference kicked off with sharing case studies through a keynote panel discussion hosted by Emma and featuring Heidi Müller from Galapagos, Steve Clark from Strive for Five, Diana Heimberg from The Bloc, and Rebecca Lisle from Jazz Pharma.

Heidi demonstrated how patients have been integrated into the Galapagos sustainability framework, for example via a commitment to reporting the percentage of Phase 2/3 clinical trials for which patient engagement has taken place, and also capturing internally the percentage of recommendations received from patients which are implemented.

“Under CSRD (Corporate Sustainability Reporting Directive) in Europe, you are required to publicly share your sustainability objectives – that now includes patient-relevant objectives – and report on relevant KPIs.”

— Heidi Müller, Head of Patient Advocacy, Galapagos

Steve then followed with a presentation on the impact of his work with Medscape on how integrating patient voices into medical education improves communication between healthcare professionals and patients. A white paper published through this collaboration won a publication excellence award and resulted in a fundamental refocus in Medscape’s strategy and activities, evidenced by the embedding of patient integration into their key pillar of ‘effective communication’ and the recent appointment of Victoria Harvey-Jones as Worldwide Patient Integration Lead. Watch this space for an ISPEP Voice to Value case study from Medscape of the impact of their new patient-integrated medical education!

“Communication from doctors to patients is one of the biggest gaps I see in healthcare.”

— Steve Clark, Founder and Patient Advocate, Strive for Five

Diana then shared information on In.Pazienti – an online community from The Bloc involving collaboration with patient influencers to connect pharma, patients, and physicians. Through working with Nestle Health Sciences to spotlight real-world experiences of inflammatory bowel disease, almost 400,000 people were reached and extensive media coverage and boosted traffic to a relevant patient association website were observed.

Last but not least, Rebecca demonstrated the value of forming a long-term Patient Leadership Council, an initiative conducted by Jazz Pharma to help overcome challenges to engagement with the rare epilepsies community. These challenges include:

Patient advocacy groups in this area are often very small, as the community can find itself siloed into tiny sub-groups in accordance with specific diagnoses.
Advocates are often parents for severely disabled children, meaning it can be extremely challenging to juggle caregiving responsibilities with collaborating with industry.

By forming this Council, Jazz Pharma were able to contract these advocates for two years, reducing administrative burden and easing sustained engagement. Further, insights gained from the Council have informed the company’s strategic decision-making, including stopping plans going ahead which would not have delivered the intended/expected value to the rare epilepsies community. What’s more, the Council brought together advocates representing a range of different rare epilepsy diagnoses, easing sharing of information and formation of meaningful, productive relationships. Rebecca summarised by concluding that the Council has “brought value both for the advocates and for our company and really strengthened how we work together.”

Looking ahead: calls for change from ISPEP and Anthony Yanni

After these case studies, it was time to hear why patient engagement is not only hugely valuable, but also undeniably urgent. Anthony Yanni from Astellas revealed that a whopping 50% of drugs launched in the last 15 years underperformed analysts’ sales estimates by over 20% (source: LEK Consulting) – clearly, we need to do things differently, and we don’t have time to waste. As Anthony summarised, “What are we waiting for?”

And yet, despite such powerful evidence that the ‘old ways’ of pharma are failing to meet expectations, securing budget for patient-focused activities remains, in Emma’s words, a “Herculean effort” – even for a mere 3% of the Medical Affairs pot. Noting the unsustainability of this current level of funding, Emma also pointed to a white paper published by Russell Reynolds in 2024 which predicted “a shift in patient engagement professionalism across pharma companies” requiring “a radical cultural and strategic realignment”.

Such realignment will require a robust, systematic approach to patient-focused drug development – something that is fundamentally incompatible with traditional ways of working in pharma. Fortunately, there are pockets of fantastic work being done here, with Michaela Dinboeck, Head of Patient Engagement at Novartis sharing how her organisation has approached this vital shift.

Systematic patient engagement

Michaela began her presentation by outlining how due to factors including the 21st Century Cures Act, there has been an acceleration of demand for evidence on patient perspectives and therefore calls for robust patient experience data. In response to this demand, Novartis has developed a vision of being at the forefront of patient-focused drug development, supported by four key strategic pillars:

Development strategy informed by patients
Clinical trial design and execution reflecting patients’ perspectives
Dossiers incorporating patient experience
Contribution to the patient engagement ecosystem

Multiple examples of how this approach has had a positive impact on decision-making were shared and can be seen below.

What’s more, Novartis are prioritising two key aspects of effective patient engagement – accountability and sustainability. For accountability, the company publishes annual, public reports of their progress on the Novartis Commitment to Patients and Caregivers. For sustainability, the Novartis Patient Insight Navigator has been developed so that any Novartis employee can access the information they need, hugely reducing the chance of information wastage and burdening patient organisations with requests for the same insights every few years.

Fast facts, psychology in clinical trials, and patient-generated data

The afternoon sessions included presentations focused on research, starting with Rasmus Hjorth of James Lind Care (and self-declared “proud ISPEPer”!) who outlined multiple handy ‘fast facts’ demonstrating the need for better patient engagement in clinical trials:

80% of clinical trials are delayed mainly due to enrolment problems.
30% of clinical trial participants drop out.
500,000 USD is the estimated cost per day a clinical trial is delayed.
82% of phase 3 trials undergo at least one protocol amendment, which can result in 500,000 USD costs per amendment.
75% of patients said they would have considered enrolling in a clinical trial if they had known it was an option.

This was complemented nicely by a presentation from chartered health psychologist Dr Sumira Riaz, who outlined numerous psychological frameworks and case studies of their application in clinical trials.

Moving away from clinical trials into other forms of research, Robert-Mitchell Thain (CEO, PBC Foundation) presented how through their dedicated app, the PBC Foundation is “using the patient experience to improve the patient experience”. Through this app, the Foundation is able to conduct patient surveys, with the resulting data being used with regulators, academic bodies, and clinicians. These data are so powerful that they provided the impetus for an NHS audit of PBC services – and the results of that audit matched those of the survey. Overall, this is a fantastic example of how patient organisations are increasingly taking a lead in data generation, laying the foundations not only for evidence-based advocacy, but also for a world where patient data is owned by the communities who create it. This is a major step forward in the spirit of the fundamental adage “nothing about us without us”.

The power of personal purpose

Also championing the spirit of advocacy, we were privileged to hear from Melanie Dixon of Cure DHDDS, who shared her powerful story beginning with the diagnosis of two of her children with an ultra-rare genetic condition. This neurodevelopmental condition, resulting from a variant in the DHDDS gene, can not only result in devastating neurodegeneration but also has no approved treatments.

“In ultra-rare conditions, it’s often up to the parents to fund and drive research for treatment … It was a ticking time-bomb. To our knowledge, no one in the world was researching this condition that could steal our children’s futures.”

— Melanie Dixon, Trustee, Cure DHDDS

It was incredible to learn how Melanie and other parents have come together in just a couple of short years to establish a charity, build a strong, international scientific advisory board, and kick off projects including a patient registry, natural history study, and research into drug repurposing, gene therapy, and more. It was also humbling to be sat among industry representatives and hear of these incredible achievements earned (and the barriers faced along the way) amidst such life-altering personal circumstances. If we ever needed motivation to do more to overcome barriers to serving the rare disease community, this was it.

Summary

With case studies, clinical expertise, and calls to action, in just one day, this conference covered a huge range of topics and offered a rich range of perspectives. And we were only halfway through… so watch this space for highlights from Day Two!

What were your memorable moments? Who do you wish you could have heard from? Let us know in the comments!

Case studies, calls for change, and advocates leading the way: Day One highlights from the 2025 Patient Centricity & Collaboration World Congress