Guest Column | October 15, 2025

Bridging RNA Innovation: A CEO's Guide To European Expansion

By Siham Ceballos, Ph.D., BioArtemis GmbH

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RNA medicines are no longer theoretical. I still remember working in the lab with RNA silencing tools and wondering whether they would ever leave the bench. Years later, I had the privilege of helping launch the first siRNA therapy. What was once a scientist’s tool becoming a treatment for real patients — that moment confirmed to me: RNA had arrived.

But proving the science is only half the battle. The other half is demonstrating that these medicines can reach people at scale. And for U.S. RNA companies, Europe is the first considerable test.

Europe appears straightforward from the outside — one EMA, one approval process. In reality, it is a patchwork of regulators, payers, doctors, and patient communities. If you treat it like a single market, you will fail. If you treat it like an afterthought, you will lose years.

Here are the patterns I’ve seen CEOs wrestle with and the lessons I’ve drawn from building European medical organizations.

The First Trap: Waiting Too Long

I’ve watched too many companies postpone their European strategy until approval is already in sight. For RNA, that’s far too late.

Phase 2 is the moment to start talking to European physicians and setting up diagnostic capacity. By Phase 3, you should already be asking payers to look at your protocol and give feedback.

Once, I watched a company share its Phase 3 protocol with European HTA bodies. They took the advice seriously, amended endpoints, and created an open channel of communication. When the data came, reimbursement was far smoother. It wasn’t because the molecule was better, it was because they listened early.

The Second Trap: Mismanaging Early Access

Europe’s reimbursement timelines can stretch to two years or more. Patients with progressive diseases don’t have that kind of time. Early access programs exist to fill the gap, but they are fragile.

I’ve seen how early access, if managed without European expertise, can create significant risks. In one instance, a U.S. partner applied an American model to Europe, and physicians were led to believe the program resembled a clinical trial. Such misunderstandings can spread quickly, putting trial enrollment and trust at risk. The lesson is clear: early access in Europe must be run by people who understand the local frameworks. You can draw a great deal from the U.S. model, but replication without adaptation rarely brings the best results

The Third Trap: Underestimating Stakeholders

In Europe, there are two groups you cannot ignore: clinicians and patients. Too many companies focus on one and forget the other.

Clinicians, key opinion leaders, and local experts shape how protocols are run and how treatments are adopted. In rare diseases especially, a handful of centers can make or break a launch.

Patient groups, though less powerful than in the U.S., still matter tremendously. They can tell you where patients are, how they navigate the system, and what arguments resonate with payers. In some cases, there isn’t even a formal group yet, and the company must help one come together, sometimes across countries.

In RNA, where diagnosis is inconsistent, these groups often help identify patients in the first place. They are not “nice to have.” They are part of the infrastructure.

The CEO’s Dilemmas

CEOs face competing demands for scarce resources. This is where the real tension lies.

Some typical questions CEOs must ask themselves include:

  • Do I hire a full European medical team in Phase 2 or rely on advisors until I see pivotal data?
  • Do I run affiliates in every major market or partner with a local pharma for greater reach?
  • Do I put budget into diagnostics and patient groups early or wait until the product is closer to launch?

I don’t believe there is a single right answer. I’ve seen CEOs succeed with lean models that relied heavily on consultants, and I’ve seen others benefit from committing early to a strong in-house presence. What matters is clarity and agility: deciding which investments align with your program’s risk, indication and timeline.

What Makes RNA Different

Every biotech faces challenges in Europe. RNA biotechs face a few that are sharper. For example:

  • You need patients identified through genetic or biomarker testing, and testing access varies wildly between countries.
  • Your medicines may require subcutaneous injection, IV infusion, or even intrathecal delivery, which means infrastructure has to be in place.
  • Payers will look closely at durability and long-term safety; you won’t get a free pass for novelty.
  • Patient groups are often fragmented, but without them you won’t get traction with regulators or payers.
  • And today, Europe is pushing for its own RNA manufacturing sovereignty, an opportunity if you partner locally but a risk if you ignore it.

A Pragmatic Way Forward

When I look back on the companies I’ve worked with, the most successful ones didn’t treat European expansion as a checklist. They treated it as a journey, a concerto in three movements:

  1. Building trust: engaging physicians, listening to payers, supporting patients, all before approval.
  2. Securing a foothold: choosing a couple of markets as early launch points, running early access well, collecting real-world data.
  3. Scaling smartly: expanding country by country, aligning reimbursement, and investing in European roots like diagnostics and manufacturing.

This isn’t neat or linear. It’s messy, and it requires judgment. But that’s the point: the strategic calls belong at the CEO and board level, not just in operational teams.

Conclusion

For RNA biotechs, Europe is the test of scale. It is where science meets strategy, where CEOs must choose between speed and depth, building and partnering, global templates and local realities.

The difference between success and failure lies not only in the strength of the molecule but in the wisdom of the strategy. When to engage, where to invest, who to trust – these are the decisions that determine whether an RNA company thrives in Europe or waits years while patients go untreated.

The first RNA medicines proved the science. The next generation will prove the strategy. Europe will decide who wins.

Useful Links:

  1. European Patients’ Forum. https://www.eu-patient.eu/
  2. EURORDIS – Rare Diseases Europe. https://www.eurordis.org/
  3. MedTech Europe. Ethical Collaborations Guidelines. https://www.medtecheurope.org/medtech-views/policy-views/ethical-collaborations-new-guidelines-help-companies-partner-patient-organisations/
  4. European Pharmaceutical Review. The year ahead for RNA therapeutic development. https://www.europeanpharmaceuticalreview.com/article/240554/the-year-ahead-for-rna-therapeutic-development-and-manufacturing/
  5. Shahid S, et al. Advances in RNA therapeutics. ScienceDirect. https://www.sciencedirect.com/science/article/pii/S3050632825000162

About the Expert:

Siham Ceballos, Ph.D., is a biotech executive and scientist-entrepreneur with over two decades of experience spanning Pfizer, Novartis, Celgene, Biogen, and Alnylam. She has led early and late-stage development programs and launched therapies in rare diseases, oncology  CNS, and age-related diseases . As founder and general partner of BioArtemis GmbH, she partners with U.S. and Asian biotech companies expanding into Europe. An innovation expert for Innosuisse, she conducts due diligence of startups and connects science, strategy, and market access to advance global therapeutic innovation.