The panel took place last week. Project Mercury was not on that stage. The questions raised there are the ones our Country Working Groups have worked on since 2023.
What was discussed
The panel was titled From Patient Voice to HTA Evidence: Insights from the EU-funded PaLaDIn project. It was moderated by patient advocate Hicham Alaoui of AMIS FSH and FSHD Europe, and brought together Ria de Haas, PhD, CEO of FSHD Europe; David Allison, CEO of TREAT-NMD; and Tim Kanters of the institute for Medical Technology Assessment (iMTA) at Erasmus University Rotterdam.
Each panelist brought a different piece of the problem: patient advocacy, registry infrastructure, health economics. PaLaDIn was designed around that combination.
Four points the congress kept returning to
FSHD Europe reported back from the panel and from the sessions and workshops around it. Four themes ran through them.
- Patient engagement must mean real influence. Patients need to be involved early, with a meaningful voice in research priorities, trial design and decision-making. Consultation after the protocol is written is not engagement.
- Lived experience is valuable evidence. Patient-reported outcomes, real-world evidence and disease registries capture what clinical trial data alone misses.
- Collaboration is essential. Patient organisations, researchers, clinicians and industry have to work together to make trials more inclusive, accessible and relevant.
- Innovation must lead to access. Advances in rare disease treatment mean little if patients cannot reach them.
Most of this is familiar inside the FSHD community. The difference is the audience: payers, regulators and industry were in the room.
Where Project Mercury fits
The second point, lived experience as evidence, is the one Project Mercury has spent the most effort on, and it is the hardest to deliver. Evidence that decision-makers accept does not come from goodwill. It comes from infrastructure: registries that collect comparable data, outcome measures that mean the same thing in every country, and a shared understanding of what the disease actually does to people over time.
Since 2023, working through ten Country Working Groups, Project Mercury has:
- Grown enrolment in FSHD patient registries past 10,000 participants
- Launched or expanded registries in six countries
- More than doubled the number of qualified clinical trial sites, from 25 in 2023 to 53 in 2025
- Published a site onboarding and readiness toolkit
(Figures from the Project Mercury Interim Progress Report 2023–2025, published 11 May 2026.)
Alongside this, our first workstream has been working on a real-world data framework, on harmonized clinical monitoring through the 295th ENMC workshop, and on making the PaLaDIn registry data set fit for HTA and payer use. The panel in Europe and the work in our working groups are two ends of the same effort: one builds the evidence, the other argues for it where decisions are made.
Why a member at the podium matters
FSHD Europe represents 14 member organisations from 12 countries and takes what it learns back to them. Project Mercury works the same way across ten countries: a toolkit developed in one country is used in the next.
Project Mercury completes its work in 2027. The toolkits, frameworks and relationships built since 2023 are being prepared for transition into the World FSHD Alliance, so that they outlive the project that produced them. Across rare disease, effective tools are developed, validated, and then quietly lost for want of an institutional steward. We are trying not to repeat that.
Thank you
Our thanks go to FSHD Europe for taking the FSHD voice into this forum, to Hicham Alaoui for moderating, and to the panellists and organisers.