SPEAKERS:
· Dana Vigier, General Manager, Regional VP Rare Diseases, Alexion
· Katri Asikainen, President, HARSO - The Finnish Alliance of Rare Diseases and Disabilities Organizations
· Rie Krabbe, Hypophosphatasia (HPP) patient representative The XLH Patient Association, Denmark
KEY TAKEAWAYS:
• Nordic countries provide only 19-29% rare disease drug access despite high GDP
• Four distinct customer groups require aligned engagement strategies beyond HCP focus
• Patient education through advocacy organizations delivers superior clinical communication outcomes
• HTA black box systems exclude patient input from reimbursement decision processes
• Approved treatments reduce rare disease diagnostic time by factor of four
Dana Vigier, General Manager and Regional VP Rare Diseases at Alexion, opened with a stark reality check for the pharma industry's customer engagement assumptions. Drawing a parallel to smartphone purchases—where consumers freely choose, buy, and use products—she highlighted the profound difference for patients navigating treatment access. "We don't have one customer, we have four customers here," Vigier explained, identifying prescribers, payers representing society, pharma suppliers, and patients as distinct stakeholder groups with divergent priorities.
The Nordic Access Paradox
The rare disease access emergency in Nordic countries defies conventional assumptions about healthcare system performance and affordability. Vigier presented data revealing a striking disparity that challenges fundamental beliefs about European healthcare equity.
"Out of all the last five years of innovative rare disease drugs which have been approved in Europe, 85% are available to German patients, reimbursed, available to German patients. 74% are available to Italian patients. Pretty much the same for French patients," she noted. "Do you know the amount that is available in the Nordic countries? Anywhere between 19% actually and 29% of these innovations."
This gap persists despite Nordic countries ranking among Europe's highest GDP nations, fundamentally challenging the narrative that rare disease drug costs create insurmountable affordability barriers. Instead, the data points to broken processes and stakeholder dynamics that prevent effective decision-making.
Katri Asikainen, President of HARSO—the Finnish Alliance representing approximately 400,000 rare disease patients across 28 disease and disability organizations—described the reimbursement reality facing patient advocates. "The reimbursement system in Finland for rare disease patients is broken. It's a black box system. We have no access to it," Asikainen explained. "We don't know—the only information we get from either of those dual systems in Finland is the name of the drug and the date that is going into those HTA bodies."
For ultra-rare populations, the challenge intensifies further. Rie Krabbe, HPP Patient Representative for Denmark's XLH Patient Association, explained her motivation for founding a patient organization despite managing three rare diagnoses herself.
"I was told that you can never have it, it's too expensive. So that's why I started to gather people with HPP in Denmark to try to make some visibility, to try to give us a voice," Krabbe said. "And we're only like 50 people in Denmark at the most diagnosed with HPP."
Communication Breakdowns in Clinical Care
Beyond access barriers, fundamental breakdowns in patient-physician communication prevent optimal care even when treatments exist. Vigier illustrated this with a clinical assessment story where a myasthenia gravis patient repeatedly answered "no problems chewing" to standardized questionnaires. "The nurse asked the second question. She said, when was the last time you had a steak? And the patient said, oh, no, I never have steak. I can't chew them," Vigier recounted. The anecdote reveals how consultation quality and question framing directly affect clinical data capture and treatment decisions.
Krabbe described her own clinical encounters with similar frustration, highlighting how time constraints and narrow specialty focus create incomplete patient assessments. "My disease is much more than a bone disease. It's a systemic disease. It's also muscle weakness, it's pain, it's a lot of other things, fatigue and such, brain fog. We didn't talk about that at all," she explained.
The knowledge gap extends beyond consultation quality to fundamental disease awareness among treating physicians. "Sometimes I get doctors who cannot pronounce my disease or who don't know that there is a drug and who don't know about the disease. They just look it up on like Wikipedia sites or something," Krabbe said. This knowledge asymmetry places patients in the untenable position of educating their own physicians while simultaneously seeking treatment.
Asikainen's experience reveals an even more fundamental gap in care continuity for rare disease patients without available treatments. "None of my diseases get any kind of ongoing survey or checkups from doctors. The only doctor that I see regularly is a dentist," Asikainen said. "So that tells a lot about the way—if you don't have a treatment or a cure or any kind of medication for your rare disease, you won't even necessarily be seen by a doctor for it."
From Patients to Advocates: Navigating System Failures
Both patient advocates described their pathway into advocacy work not as voluntary engagement but as necessary response to systemic failures. Asikainen's motivation was deeply personal, rooted in her own experience navigating multiple rare disease diagnoses simultaneously.
"I have four rare diseases myself. I'm a rare disease and disabled person," she explained. "And that brought me into patient advocacy because we were mistreated by the system and I was put in a place, by the system, by the society, that I could not get out of myself."
Krabbe similarly described her two-decade healthcare journey using language that captured the isolation and resource scarcity rare disease patients encounter. "I've been walking through the healthcare system for 20 years. I have three rare diagnoses," she noted. "But I've been walking—it's been a desert walk, trying to get help, trying to find a bus stop to move on and get some help."
Asikainen's advocacy work extends beyond individual patient support to legislative reform addressing systemic barriers. "We also have problems with research laws in Finland and usage of patients' information. So I do a lot of legislative work to get that on the side of rare disease patients so that our lives can be easier via the laws that govern us," Asikainen explained.
The financial burden on rare disease patients compounds clinical challenges. "The financial burden for rare disease patients is disproportionate compared to any other patient group out there. We are taking more on our individual backs and we still have a legislative uphill battle on getting things like disability retirements and sick leave," Asikainen said.
Krabbe emphasized the need for coordinated care that addresses patients holistically rather than fragmenting treatment across disconnected specialists. "For my co-patients to experience that they actually get a doctor that they feel like they're getting help and the doctor is seeing the whole patient, not just a single toe or foot or something," Krabbe explained. The burden of coordination falls to patients least equipped to manage it.
Redefining Pharma's Customer Model
Vigier argued that the pharma industry must fundamentally rethink its commercial model to address these systemic challenges. "We can't continue to consider customer and focus on super sophisticating our customer engagement when we talk about HCPs only," she stated. "We need, we must—of course payers are not hurdles, they are our customers. Society is our customer and patients are our customers."
The rare disease context offers particular urgency and opportunity for this transformation. "If we get it right in rare diseases, we will get it right in all the rest," Vigier argued. The logic suggests that engagement models effective for ultra-rare populations will scale effectively to larger therapeutic areas.
Central to this reimagining is recognizing that stakeholder groups operate with fundamentally different value definitions. "The biggest conundrum we're facing as pharma, in a way, is the very different perceptions of value by the different groups of customers, decision makers," Vigier explained. The solution requires convening rather than convincing—facilitating dialogue where stakeholders collaboratively define value.
Data supports pharma's ecosystem impact potential when engagement extends beyond traditional HCP audiences. "Do you guys know that rare disease patients spend on average about five years going from doctor to doctor before they get the first diagnosis?" Vigier noted. "And one thing that I was impressed by was there is a study lately that showed that basically as soon as there is an approved EMA treatment or FDA treatment, an approved treatment in a rare disease, that time is diminished by a factor of four."
Patient Education as Strategic Imperative
When asked how pharma could best support patients, Asikainen emphasized capability-building over awareness campaigns. "I'd say the key number one is educate the patients and you do that via the patient organizations. When we educate the patients to talk about themselves in a more non-passionate way to the doctors, we get a better response from them," she explained. "But we also need to educate the patients about the whole field and the bureaucratic jungle out there."
Asikainen described the complexity patients must navigate as requiring professional-level expertise. "It's not that simple that you just get sick and then you get a treatment—it's not like the flu when you have a chronic rare disease. It's a whole different ball game. And I say that it's being professionally sick because you're advocating for yourself," she said.
Building Trust Across the Value Chain
The session concluded with a call for trust-building across all stakeholder groups as the foundation for systemic change. "The last thing that I really would love to see more and more is trust between the stakeholders in the rare disease field. It's not just about us patients trusting the doctors, but the doctors trusting us patients, but also the pharma companies, the payers, the systems," Asikainen said.
Vigier synthesized the discussion into two core imperatives for the pharma industry moving forward. "HCPs are a diminishing part of your customers. There is a rising and a very important need to sort of consider the same way we're considering our HCPs as customers to consider patients, support their education, support their understanding and the relationship," she stated. "And the second thing I would like us to leave with is trust. Let's build that, let's use that." These twin priorities—expanding customer definition and building trust—frame the transformation required for pharma to effectively serve rare disease communities.
To get you highlights of Pharma Customer Engagement EU faster, we are using generative AI technology to summarise the transcripts of the sessions. If you have any feedback about the summary, please contact lucy.fisher@thomsonreuters.com.
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