Rare disease is complex by definition and requires complex solutions to connect patients with the treatments that could benefit their health.

More than 30 million Americans (~1 in 10) live with a rare disease. Yet, despite that scale, individual conditions affect relatively small populations, symptoms can vary widely, and diagnosis can take an average of 4-5 years, but often much longer, with frequent misdiagnoses and treatment failures along the way.

For patients and providers, that creates an extraordinarily difficult, frustrating, and sometimes even a frightening diagnostic journey. For pharma marketers, it creates another problem: How do you design an audience that accurately represents a patient population when the population itself is so difficult to define?

The answer requires sophisticated modeling, innovative software, and experts who understand the disease well enough to apply that intelligence.

Rare disease requires a more complex vocabulary

Think about a patient journey as a sentence constructed by every diagnostic, diagnosis, procedure, prescription, and HCP visit representing a word. For many common conditions, the vocabulary needed to understand that sentence can be relatively simple. There are more patients, more established patterns of disease and care, and often clearer signals to diagnose and treat them.

Rare disease is different. The meaning of the sentence may exist in a particular combination of events, their sequence and timing, and how those patterns relate to the broader population. Two patients with the same disease may reach the same diagnosis through very different journeys; different sentences, same outcome.

Now expand that across an entire patient population. Thousands of potential signals can occur in thousands of combinations. Some are highly informative. Others matter only alongside another signal or because of when they occur. And others still may be missing. A manually constructed rule might say: Diagnosis A + procedure B + specialist C. But the actual population may contain hundreds of meaningful variations.

Sophisticated modeling can evaluate those patterns at a scale that manually constructed audience definitions cannot. Instead of specifying every possible path in advance, models can learn which combinations of signals characterize the population and how much each contributes. The result is a richer, and more refined, representation of the disease and the population, and a better understanding of the vocabulary.

Sophisticated models need innovative software

An advanced model alone is only the first step to reaching the ideal audience. Rare disease experts need to explore what the model uncovers; understand tradeoffs between prevalence, reach, and channel; test hypotheses; and refine the population for activation.

Software makes that possible. The ideal audience is rarely hiding inside a single code or predefined rule. It must be modeled, explored, and designed by the people who understand it best.

How BranchLab approaches rare disease

BranchLab combines next-generation AI modeling, population exploration through world-class software, and putting experts directly in control within Pathwai™, our Semantic Activation Platform.

Our models learn the patterns that characterize a population, and Pathwai gives rare disease experts the software to explore that intelligence and optimize it toward peak performance. That’s why across rare disease campaigns, BranchLab audiences consistently outperform traditional audience approaches.

+70% Audience Quality | +65% Qualified Reach | +2x Script Lift | +10x Return on Investment
Mean performance independently verified by leading third-party measurement providers.

But sophisticated technology is most powerful in the hands of experts, which is why we work with partners like Underscore Marketing, the only media agency dedicated to supporting complex specialty therapeutics.

According to Jay De La Cruz, SVP of Media Activation at Underscore Marketing: “Rare disease commercialization is uniquely complex. Patients are harder to find, their journeys are harder to understand, and every opportunity to reach them matters. For more than 20 years, Underscore has helped biopharma manufacturers bring some of the most challenging therapies to market.

“We believe people living with rare diseases and their caregivers deserve every opportunity to learn about treatments that could make a meaningful difference in their lives. That's why we continually explore innovative technologies and collaborate with companies like BranchLab. Their AI-powered healthcare audience intelligence platform adds another dimension to our precision media capabilities, helping us identify and reach relevant audiences more efficiently and make smarter media investment decisions.”

Rare disease demands more from audience technology because the populations themselves are more complicated. It takes models capable of understanding a much more complex vocabulary, software that makes that intelligence usable, and experts with direct control over how it is applied.

That is what a Semantic Activation Platform makes possible.

References

1. Medical Science Educator. Zebras Among Us: Advocating for the 30 Million Americans Living with Rare Disease. 2023.

2. Orphanet Journal of Rare Diseases. Time to diagnosis for a rare disease: managing medical uncertainty. 2024.