SPEAKERS:
· Kim Moran, SVP & Head of U.S. Rare Disease, UCB
· James Smith, Director, Marketing ATTR, AstraZeneca
· Eric Peacock, Co-founder and President, MyHealthTeam; Chief Patient Officer, Swoop
KEY TAKEAWAYS:
· Patient panels reviewing campaign asset catch creative missteps no internal team will ever see
· Ultra-rare disease launches require building physician courage, not just physician awareness
· Virtual sales reps, dismissed five years ago, are now among the most effective tools for reaching long-tail rare disease physicians
When UCB launched in thymidine kinase 2 deficiency this year, the commercial team faced a stark arithmetic problem. According to the FDA, there are 120 patients living with the disease in the United States, although the condition may be underdiagnosed. Nearly half the patients from clinical trials are seen by a single physician in New York City. TK2d is often fatal, in patients with initial symptoms less than 12 years of age, 50% of patients face mortality within one year after symptom onset. "The primary endpoint is survival," said Kim Moran, SVP and Head of U.S. Rare Disease at UCB. "That ups the ante of what you're doing every day."
That launch, simultaneous with UCB's work in generalized myasthenia gravis, sits at the extreme end of a spectrum that every rare disease marketer navigates. The strategies that work at 120 patients are not the strategies that work at 10,000. And DTC strategies that work in mass-market pharma need tailoring to fit rare audiences.
Finding patients who don't know they're lost
The foundational challenge in rare disease isn't market share. It's diagnosis. Most patients cycle through multiple misdiagnoses for years before anyone names what they have. In myasthenia gravis, roughly 20% of patients present with bulbar symptoms; swallowing difficulty, muscle fatigue, that get misread as MS or other neuromuscular conditions. In ATTR amyloidosis, there are 145 to 150 genetic variants, each presenting differently, some leaning toward cardiomyopathy, others toward polyneuropathy. A cardiologist seeing an ATTR patient may never think to ask about GI symptoms. But those symptoms matter.
"If you're going to a cardiologist, you might still need to talk about constipation or diarrhea or GI issues," said James Smith, Director of Marketing for ATTR at AstraZeneca, "just so that physician has the full scope of what a patient's experiencing to help accelerate that identification."
Geography adds another layer. Certain ATTR variants cluster by ancestry. Portuguese variant patients concentrate in specific U.S. regions. Appalachian and Irish variants appear elsewhere. Smith's team uses that geographic specificity to give community physicians a more targeted picture of which patients are likely to walk through their doors - and what to look for when they do.
Moran's team has taken a different angle on the identification problem. UCB has been experimenting with symptom trackers and assessors that help patients articulate what they're experiencing before they see a physician. The goal is to sharpen the patient-physician dialogue. "We try to allow them to really be crisp on the symptoms so that when they are having the patient-physician dialogue, they are able to name it a bit more," Moran said. For a disease where the diagnosing physician may have had zero minutes of training on the condition, a patient who arrives with precise symptom language can make a difference in misdiagnosis.
The patient panel that killed a campaign
UCB's rare disease unit launched with four words as its operating principle: patient-first, digital-first. The team assembled a panel of 15 people living with generalized myasthenia gravis who reviewed everything - patient enrollment forms, patient services, HCP marketing, patient-facing campaigns.
One concept depicted patients using a battery-level metaphor, the kind of visual shorthand that tests well in internal creative reviews. The panel's response was unanimous and immediate. "Do not depict me as a battery," Moran recalled. "This is offensive. It's inappropriate."
The lesson wasn't just about that one creative execution. It was about the gap between what looks clever to a marketing team and what feels accurate to someone living with the disease every day. UCB now consults the panel at least monthly. One member is a young man who was diagnosed with MG at 15 and joined the panel at 18, after his mother had participated as a caregiver. His presence on the panel represents something a metaphor could never capture.
Eric Peacock, co-founder of MyHealthTeam and Chief Patient Officer at Swoop, sees the same dynamic across the 70 patient communities his organization runs, 20 of which are in rare diseases. Roughly 80% of community members are information seekers, not advocates. They're served by local neurologists or cardiologists who may have had 30 minutes - or zero minutes - of training on their condition. "They’re there because they want to learn," Peacock said.
Discover more on this topic at Pharma Customer Engagement USA 2026 (October 27-28, Philadelphia) - where commercial, marketing, medical, data and AI pioneers converge. Explore the agenda here.
Reaching physicians who have never seen a case
HCP strategy in rare disease splits into two problems that require different solutions. The first is the thought leader tier - the 50 or so specialists nationally who diagnose and treat the majority of cases. The second is the long tail: thousands of community physicians who may see one or two patients a year, if that.
For the thought leader tier, Smith's team has focused on smaller regional congresses alongside the major national meetings. "To build trust, we need to show up at those smaller, more regional congresses to engage physicians," he said. The team has also explored fellowship-style programs targeting younger physicians early in their training, before they develop the habit of missing a rare disease diagnosis. Expert-on-demand networks connect community physicians with regional specialists in real time, so a provider who suspects ATTR but lacks confidence can reach someone who can help - or can refer to an amyloid center of excellence with a warm handoff rather than a cold one.
For the long tail, Moran has landed on a combination that would have seemed counterintuitive a few years ago. "I don't think five years ago I would have been like, hey, we should maybe light up those phones again," she said of virtual sales reps. "Wow, it actually is hugely effective, especially when you're trying to find a needle in a haystack." Predictive analytics and suggestion engines help field teams anticipate which community physicians are likely to encounter a rare disease patient in the next few weeks. Doximity, CME programs, targeted digital content - the channel mix is bespoke by physician, not uniform across the list.
Connected TV has moved from experiment to standard. UCB is now running DTC connected TV in rare disease, reaching 50% of the patient population at scale. Smith's team uses NPI tracking to measure how many target physicians are being reached through the same devices. "People laugh when we're saying you're going to do TV for a rare disease," Smith said. "Like, we're not buying Jeopardy, but we're buying custom audiences that we know have high affinity and high audience quality." The reach numbers in rare, precisely because the target population is small, make it easier to demonstrate impact - not harder.
AI as infrastructure, not oracle
The panel's discussion of AI was notably unsentimental. A recent study found AI misdiagnosed conditions in 53% of cases. In rare disease, where the training data is thin and the presentations are heterogeneous, that number is almost certainly higher.
The more productive question isn't whether AI can diagnose rare disease patients. It's whether AI can help surface suspect patients for physician review - and how to scope that function so it doesn't create more noise than signal. Smith's team has learned to limit EHR algorithm runs to patients a provider will see in the next two weeks or month, rather than running against an entire system. "If you go to Penn and they run it against their entire EHR system, there's going to be patients that are deceased, patients that no longer go to that provider," he said. A list of 1,000 flagged patients paralyzes a physician. A list of 12 creates action.
On the patient side, Moran flagged a trust problem that precedes the accuracy problem. Roughly 60% of patients are worried that AI will override their physician's judgment. Deep fakes and misinformation have eroded confidence in digital health tools broadly. "This trust element is going to be a trend that we continue to see rise over the next couple of years," she said.
Peacock offered a counterintuitive data point from MyHealthTeam's own experience. When his team launched AI-assisted tools in patient communities, expecting a flood of questions, they got near-silence. Patients didn't know what to ask. What worked was showing the six most common questions - essentially prompting patients to recognize their own concerns in someone else's words. The same principle applies to how pharma should think about feeding large language models: patient quotes and lived-experience language are what LLMs are currently starving for, and organizations that include authentic patient voice in their published content are seeing referral traffic from AI overviews increase, not decrease.
The playbook that emerges from this panel isn't a set of tactics. It's a set of commitments: to find patients the healthcare system has lost, to build physician confidence where training never existed, and to treat access as a relationship that starts before diagnosis and continues long after the prescription is written. In a disease with 120 patients, every one of those commitments is visible. There is no hiding behind aggregate numbers.
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Discover more on this topic at Pharma Customer Engagement USA 2026 (October 27-28, Philadelphia) - where commercial, marketing, medical, data and AI pioneers converge. Explore the agenda here.