Bringing a rare disease drug to market is not for the easily discouraged. Small patient populations mean low disease awareness—not just among the public but also healthcare professionals and policymakers. That translates to delays in diagnosis, uncertainty in treatment approaches, and challenges in resource allocation.
To make matters even trickier, many rare disease treatments are Advanced Therapy Medicinal Products (ATMPs), like gene therapies, which don’t fit neatly into existing healthcare systems. And then there’s the pricing problem—these therapies come with hefty price tags, requiring sophisticated and innovative market access strategies.
For big pharma, rare disease drug launches bring internal headaches too. With pipeline overflow, companies must decide which drugs get the full push and which get sidelined. In smaller markets like the Nordics, some products never even launch—or if they do, they might get minimal support. That’s devastating for patients who need these treatments.
Rare disease clinical trials by phase, therapy areas 2023²
And how do you build a team when your entire customer base in a country is just a few specialists? Do you still hire a Key Account Manager, Medical Advisor, and Brand Lead for each country? Or do you take the “Nordic model”—one team covering multiple countries? If so, be careful: a fully Swedish team launching in Finland is never a good idea. And vice versa.
An increasing number of smaller pharma and biotech companies are taking commercialization into their own hands—but it’s no easy feat. In the Nordics, this often means building an entire organization from scratch, a tough sell when competing for top talent with more established players. And in rare diseases, only the best will do—healthcare professionals and other stakeholders expect deep expertise. On top of that, it’s a high-risk, high-cost game; when your company’s survival hinges on the success of just one or two rare disease treatments, there’s little room for error.
These challenges are forcing pharma and biotech companies to rethink their operating models. How do you stay lean and agile while making sure you have top-tier expertise in place? Pharma is a conservative beast but with rare disease drugs, the old ways won’t cut it. Companies that rethink their approach now will define the next era of pharma.
References:
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EMA. Human medicines in 2024 (Jan 16, 2025)
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IQVIA. From Orphan to Opportunity: Mastering Rare Disease Launch Excellence. 2024.
- Fermaglich LJ & Miller KL. Orphanet J Rare Dis. 2023;18(1):163.
