In late 2025, I sat down with Katja Rüdell, one of the most experienced voices in patient-reported outcomes (PROs) and clinical outcome assessments (COAs).
We covered a lot of ground — from the nuts and bolts of PRO development to regulation, HTA, and where the field is heading. But what stuck with me most wasn’t a technical detail.
It was this simple idea:
No matter how sophisticated our models, endpoints, or submissions become — if we don’t truly understand what patients experience, we risk building evidence that looks good on paper but misses the point.
PROs are Everywhere — But Not Always Used Well
There are thousands of PRO and COA instruments available today. On paper, that sounds like a solved problem.
In reality, it isn’t.
Katja made a key distinction that often gets overlooked:
Generic instruments (like EQ-5D) are essential for comparability and HTA decisions
Disease-specific instruments are essential for understanding whether a treatment actually helps patients in ways that matter to them
The problem?
HTA systems naturally gravitate towards generic measures — while disease-specific insights often get side-lined, even though they are far more meaningful for clinicians and patients.
Both are needed. We just don’t always treat them that way.
Patients Have a Voice — Our Job is to Make it Heard
One of the most powerful moments in the conversation came when Katja shared how her personal experience as a patient and caregiver shaped her career.
After a serious accident in her late teens and a long rehabilitation period, she made a conscious decision to work in a field where she could improve patients’ lives at scale. Later, a university lecturer said something that stuck with her:
Patients have a voice — and we should make that voice heard.
That mindset runs through everything she does in PRO research.
It also explains why she’s so critical of box-ticking PROs — instruments included because regulators expect them, not because anyone plans to learn from them.
Why good PROs still don’t make it into trials
If PROs are so valuable, why are they still underused?
Katja was very candid here:
PROs add cost, complexity, and operational burden
Smaller biotech firms often include only the minimum required endpoints
Electronic PROs promise a lot — but missing data and usability issues remain a real challenge
The result is a familiar pattern:
Treatments get approved
Patients receive them
And only after launch do we start asking whether they truly fit into people’s lives
That’s a missed opportunity — ethically, scientifically, and economically.
A Simple Test for “Good” PRO Evidence
One practical takeaway I really liked:
A good PRO doesn’t need to be perfect.
But it must show change in the right direction — in a way that patients can feel and recognise.
If symptoms improve, functioning improves, or daily life becomes easier — the instrument should capture that clearly and credibly.
If it doesn’t, the problem may not be the treatment… but the measurement.
Where the Field is Heading
Katja is optimistic — cautiously so.
We’re seeing:
More patient involvement in research and conferences
Greater attention to productivity, functioning, and daily life
Growing recognition that “convenience” (oral vs infusion, dosing frequency, burden) actually matters
But progress won’t come from better instruments alone.
It comes from taking patient experience seriously early — not as an afterthought.
