There is a temptation, when scoping a rare disease KOL mapping project, to treat it as a smaller version of something you have done before. Fewer patients, fewer specialists, fewer publications. Simple, right?
Not quite.
Rare disease KOL mapping is not oncology with the volume turned down. The scarcity is real, but the complexity is not scaled back to match. In many ways, the challenges are amplified precisely because the landscape is so thin. When every expert counts, a single blind spot is not a minor inconvenience. It is a strategic gap that can quietly derail an entire launch.
Here is where those blind spots tend to live, and how we find them.
The Data Desert Problem
In a busy therapy area like Type 2 diabetes or rheumatoid arthritis, the published literature is deep enough to swim in. Bots can scan PubMed, pull citation counts, cross-reference congress presentations and clinical trial registries, and produce a reasonably credible starting point.
In rare disease, the pool is shallow. And in many markets, it is not just shallow. It is written in a language your database has never been trained to read.
Brazil, Korea, Egypt, Saudi Arabia, Thailand: these are markets with active, respected rare disease communities and legitimate regional expertise. But a significant portion of their scientific output is published in local language journals, presented at domestic congresses that never make it onto the global radar, and shared through professional networks that exist entirely outside of English language medical databases.
A purely AI or database driven approach to KOL identification in these markets will consistently surface the same small group of internationally published specialists. That group may be excellent. But it is not the full picture. The clinicians who actually shape treatment decisions for most patients in those countries are often invisible to any tool that relies on Western indexed data alone.
This is where our in-country experts change everything. We work with people who are physically present in these markets. They know which regional congresses matter. They understand local medical hierarchies. They can tell you whether the person at the top of the hospital organogram is actually the one who influences prescribing, or whether that influence sits two levels below with someone who has never published a single English-language paper.
When One Person Is Half the Landscape
In a large therapy area, the influence of any single KOL is meaningful but rarely singular. There are enough voices in the room that no single departure, shift in opinion or change of institutional affiliation sends shockwaves through the whole field.
In rare disease, you regularly encounter situations where one person accounts for an extraordinary proportion of the intellectual and clinical weight in a given market. The global thought leader who not only runs the only specialist centre in the country but also chairs the guidelines committee, trains every fellow who will go on to manage this condition, and sits on the international advisory board that shapes clinical practice globally.
This concentration of influence creates two problems that often go unacknowledged in a standard mapping exercise.
The first is strategic over-reliance. If your launch plan is built around a single KOL and that person becomes unavailable, changes their view, or simply has too many competing commitments to give your programme meaningful time, you have a problem.
The second is landscape distortion. A pure quantitative analysis will correctly identify this person as highly influential. What it will not tell you is that their influence is so dominant that other potential voices in the field have been shaped or constrained by proximity to them. Understanding the true landscape means understanding those dynamics, not just counting publications.
This is exactly the kind of context our researchers surface through structured qualitative work alongside the data. We combine AI-powered analysis with human interviews and expert input to build a picture that is accurate, not just technically correct.
The Researcher, the Clinician and the Patient: Sometimes the Same Person
In mainstream therapy areas, the line between researcher, treating clinician and patient advocate is usually clear. In rare disease, it blurs constantly.
It is not unusual to encounter a leading KOL who was motivated to enter the field because a family member has the condition. Or a patient advocate organisation that is effectively co-led by a clinician who is also one of the foremost global experts. Or a parent who became a researcher, founded a patient registry and now sits on regulatory advisory panels.
These individuals carry enormous influence. They are trusted by patients and clinicians alike, they often have the ear of payers and regulators, and they move across professional categories in ways that standard KOL mapping frameworks are simply not built to capture.
If your mapping exercise only looks for people with academic or clinical affiliations, you will miss them entirely. And missing them is not just a data gap. It is a missed opportunity to engage with some of the most credible and impactful voices in the therapy area.
Our approach explicitly accounts for these hybrid profiles. We map patient advocacy organisations alongside clinical networks, and we surface individuals who sit across both worlds.
Getting It Right Actually Matters
We spend a lot of time in this industry talking about KOL mapping in the context of launch readiness, medical education strategy, and advisory board composition. All of that is legitimate and important.
But in rare disease, there is something else at stake.
These conditions are often severely underdiagnosed. Patients wait years, sometimes a decade or more, before anyone puts the right name to what is happening to them. The experts who are genuinely shaping diagnosis and treatment are often the direct reason some patients get there at all.
Getting the landscape right, finding the real experts rather than just the most visible ones, understanding where clinical leadership actually sits rather than where it appears to sit, is not just good launch strategy. For patients in rare disease, it is the difference between reaching the people who can help them and missing them entirely. That feels like a reason to get it right.
By Simon Rosenberg, Director of Research, Scout
simon.rosenberg@scoutdata.co.uk
