February 20, 2026
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Marketing in the rare disease space comes with unique challenges and meaningful opportunities. In this edition of 5 Questions With, Jen Loga, DeepIntent VP, Client Insights & Analytics, shares her perspective on how brands can navigate small patient populations, complex diagnostic journeys, and evolving measurement strategies to drive smarter engagement.
1. From an analytics standpoint, what makes rare disease marketing fundamentally different from more common conditions?
Aside from the smaller volume of patients, which makes both analysis and reach challenging, those with a rare disease are unique in their expertise. Someone with a rare condition has a PhD in their own condition. They often know more than their doctors because specialists are few and far between. These are patients who are up until 3am reading medical journals and connecting with others across the globe who share their diagnosis.
When marketing to this population, brands need messaging that is tight, clinically accurate, and data-driven.
2. Rare disease populations are small by definition. What tools can help brands reach the right audiences without wasted spend?
I wouldn't say it's the tools that work to a brand's advantage. It's the expertise. Healthcare experience is crucial for understanding the treatment and condition lifecycle.
For example, a recent study found that the typical rare disease patient saw six physicians before receiving the correct diagnosis, with an average of 30 appointments or hospitalizations. This isn’t a patient journey; it’s a rare disease odyssey, and it changes everything about where and when healthcare marketers should engage. Tools without expertise won’t be able to move the needle.
Many rare disease patients spend years undiagnosed, showing up in claims data with related symptoms or conditions long before they ever receive a formal diagnosis. Leveraging a robust clinical dataset is essential, both to identify and reach this niche population without wasted spend and to generate actionable insights that prove campaign value.
3. With small patient populations, how do you evaluate campaign success when traditional statistical significance is nearly impossible to achieve?
This is one of the biggest challenges in rare disease analytics and, honestly, it requires a mindset shift. You're not going to get the same statistical rigor you would with a diabetes or hypertension campaign, where you have millions of relevant consumers to work with.
When we don't have enough clinical data to support the primary metrics, we focus on directional trends and leading indicators. Are we seeing movement in the right direction? Are script volumes trending up, even if the numbers are small? Are we reaching the right HCPs, specifically the specialists who actually treat this condition?
We also look at efficiency metrics differently. Cost per script becomes critical because every single patient matters so much. The clinical data itself, and the recency of the dataset, is imperative to ensure proper tracking and insight over time.
4. How should brands think about the long diagnostic odyssey many rare disease patients experience when planning their media touchpoints?
The rare disease odyssey is real, and it's brutal. Research shows patients wait nearly five years on average to get a correct diagnosis. That's five years of uncertainty, misdiagnosis, and frustration.
From a media-planning perspective, this changes everything about where and when you reach people. For those with an accurate diagnosis, the media strategy isn’t tremendously different than any other condition. People with a rare disease are people, and their media consumption looks much like that of anyone else. The clinical signals of disease progression, however, are critical to engaging throughout the person’s odyssey.
However, you can't just focus on the point of diagnosis or post-diagnosis, because for many rare disease patients, that moment hasn't happened yet. Brands need to think about the undiagnosed and disease progressing population, people who are experiencing symptoms but don't yet have a name for what's wrong.
This means showing up in spaces where people are searching for answers. Symptom-related content, condition education, even caregiver forums. It also means your messaging needs to meet people where they are emotionally. Someone who has been dismissed by multiple doctors isn't looking for flashy brand messaging. They're looking for validation and information. Lead with empathy and clinical credibility, and you'll earn their attention.
5. How can brands balance precision targeting with scale when the patient population is inherently small?
This is the tension every rare disease marketer lives with. You want to be precise, but if you get too narrow, you end up with no scale and your campaign stalls. If you go too broad, you're wasting budget on consumers who aren’t relevant.
The unlock here is thinking beyond just the patient. Yes, the patient population is small, but the ecosystem around them isn't. You have caregivers, you have the specialists who diagnose and treat, you have the PCPs who might see early symptoms and need to know when to refer. Suddenly, your addressable audience gets a lot bigger without sacrificing relevance.
On the patient side, it's about being smart with your clinical signals. You may not be able to reach the diagnosed population at scale, but you can reach people showing symptoms or related conditions that often precede diagnosis. It requires a deep understanding of the condition and the patient journey, which is where healthcare expertise really matters. A generalist DSP isn't going to know that, for example, adults with undiagnosed Type 1 Gaucher often show up in claims data first with unexplained low platelet counts and an enlarged spleen. That context changes everything about how you build and refine your modeled audience.
Rare disease marketing demands a thoughtful balance of data, empathy, and strategic focus. When brands pair precise engagement with deep healthcare expertise, they can improve outcomes for their campaigns and for the patients and providers they aim to support.
Want to dive deeper into the possibilities of precision marketing in rare disease? Click here.
