Patient expert. Keynote speaker.
I have SMA.
I am not SMA.
I advise pharmaceutical and biotech teams on clinical trial design, outcome measures and access, and I speak to the people who make those decisions. My work sits in one place, the distance between what a trial can measure and what a patient actually lives, and what that distance costs when a payer looks at the file.
A patient perspective that comes with the technical detail attached
Patient input is easy to obtain and hard to use. Most teams get it too late, in a form that arrives after the decisions it should have informed.
What I offer is different in one specific way. I have lived inside the disease, inside a clinical trial, and inside a reimbursement decision, and I am trained in how all three are constructed. So I can tell you not only that an endpoint misses something, but what it misses, why the payer will notice, and what a workable alternative looks like given your timeline and your regulator.
That is the value. Not the story. The story is only the reason I am able to see it.
Where the perspective comes from
I have spinal muscular atrophy. Before treatment, more than fifty hospital admissions.
In 2021 a treatment reached me. My breathing, my voice and my swallowing improved, and my acute admissions nearly stopped.
On the formal motor outcome measures used in the study, I score zero. Nothing moved.
Both things are true. That is the gap in a single case, and it is not unusual. It is the ordinary consequence of measuring what is convenient rather than what changes a life. When it shows up in a reimbursement file, it stops being an academic point and starts deciding who receives a medicine.
I am EUPATI-trained, and I currently sit inside a Dutch conditional reimbursement study, which means I have watched an access decision being assembled from the inside rather than reading about it afterwards.
How I work with organisations
Advisory and the access-gap audit
A structured review of a trial design, before the protocol locks, identifying where the evidence will fall short of real patients and where it will meet resistance from strict payers. Alongside that, endpoint and outcome measure input, document review, and advisory boards.
Speaking and keynotes
Conferences, symposia and internal company sessions. Direct, practical, and aimed at the decisions the audience actually controls. This is often how a working relationship starts.
I work with pharmaceutical and biotech companies, research institutes and healthcare organisations around the world.
My background is in spinal muscular atrophy, and my work is not confined to it. I work across neuromuscular and rare diseases. The mechanics of the access gap are structural rather than diagnostic, and they behave similarly across conditions.
Get in touch
If you have a study in design, an endpoint you are uneasy about, or an event to fill, I am glad to hear from you.