About
Stefan Bos
Independent patient expert, advisor and keynote speaker. I have lived inside a disease, a clinical trial and a reimbursement decision, and I am trained in how each of them is built.
How I got here
I was born with spinal muscular atrophy, a progressive neuromuscular disease. I was told early that treatment was too distant to count on. My parents raised me to look at what I could do rather than what I could not, which turned out to be sound advice and reasonable preparation for this work. More than fifty hospital admissions later, it still holds.
In 2021 a treatment reached me. My breathing became easier. My voice returned. My swallowing improved. The acute, unplanned admissions that had shaped much of my life nearly stopped.
On the formal outcome measures used in the study, I score zero. Nothing moved.
Both of those are accurate, and only one of them counts when a decision is made. That is the point at which this stopped being my private situation and became my professional subject.
I could have argued about it publicly. I decided it would be more useful to understand how the line gets drawn, and to be in the room while it is still moveable. So I trained with EUPATI, the European Patients' Academy on Therapeutic Innovation, and began working with the companies and institutions that design treatments and build the case for access.
I have SMA. I am not SMA.
Not a single-disease expert
SMA is where I learned this. It is not the boundary of it.
I work across neuromuscular and rare disease, including SMA and FSHD, because the problem is structural rather than diagnostic. Endpoints that miss meaningful change, populations excluded because they are difficult to measure, and access boundaries set by the edge of the evidence rather than the edge of the benefit. These recur, largely unchanged, across conditions.
Background
EUPATI Fellow, certified 2024. Independent patient expert, advisor and speaker, working with pharmaceutical and biotech companies, research institutes and healthcare organisations all over the world on trial design, outcome measures, patient engagement, access and reimbursement.
I also hold voluntary patient-representation roles at national and European level, and I am a member of the accessibility working group at Amsterdam Airport Schiphol. My consulting work is independent of those roles, and my advice is my own.