The Spreadsheet With Forty Countries

Most market entry discussions I join start with a spreadsheet. Forty-odd countries down the side, prevalence, price and population across the top, and a ranking at the bottom that tells you to launch where the numbers are biggest.

For a common disease, that can work. For a rare disease, and certainly for a gene therapy, it usually doesn't. The numbers that matter most aren't on the sheet: whether a hospital can actually give the therapy, whether the payer can carry a one-time price in the millions, how many patients are diagnosed rather than just estimated, and what it will cost you to make all of that work.

This piece doesn't cover every strategic factor in a launch. That would take a book. It focuses on two questions I'm asked again and again by biotech and pharma teams preparing an orphan launch: which markets should we enter, and should we go in alone or with a partner? Where something is my practitioner judgement rather than how the system works, I'll say so.

Part 1: Choosing Where to Go

When you assess a market, some of the factors are about the country. Others are about you. Both matter, and they often pull in opposite directions.

Unmet need. The instinct is to go where the need is highest and set up shop where the patients are. Often that's right. But a large unmet need can be outweighed by other factors, and a market full of patients you can't diagnose, treat or get reimbursed isn't a market yet.

Infrastructure, and the ability to pay. Can the healthcare system actually support the therapy? For a gene therapy that means qualified treatment centres, specialist teams, cold chain and, for ex vivo products, cell collection and handling. And can the system pay for it? If the answer to either is no, you have to decide whether the investment needed to make it work is worth it. That takes a proper country-level assessment, not a desk estimate.

The investment needed to drive uptake. What will you need to build, hire or install in each country to get patients treated? Field medical, access and patient-support roles, centre qualification, logistics, diagnostics support. Put that cost next to the realistic patient numbers and the cost-benefit picture often changes.

Your own vision and strategy. This one doesn't show up in market research, but it shapes the decision more than people admit. If you intend to be a long-term player in gene therapy, it can make sense to invest in Centres of Excellence, supply chains and strong local teams even where the short-term return is lower. You're building capability for the next product, not just this one. Some companies also choose to enter difficult regions, including parts of Africa, because it fits their ethos and because it teaches them things about orphan products that pay off when they expand elsewhere.

None of these factors settles the question on its own. Deciding to enter a market, or to put one ahead of another, means weighing considerations that contradict each other. That trade-off is a leadership decision, and it's better made explicitly than left to the spreadsheet.

Part 2: Three Market Archetypes

Not all markets are created equal. Patient numbers, infrastructure, ability to pay and access routes vary enormously from one country to the next. I find it useful to group markets into three archetypes. It isn't a perfect model, but it gives a team a shared language for prioritising, and for deciding how much to invest where.

High-potential markets

These are the usual suspects: the US, and in Europe the UK, Germany, France, Italy and Spain. They tend to have enough patients, good disease awareness, well-developed healthcare systems and payers who are, eventually, receptive to the kind of value case a gene therapy makes.

They also demand the most. To capture their full potential you'll usually need a full in-country model with a high-touch organisation: local medical, access and patient-support roles that help set up a treatment network, then support the Centres of Excellence that administer the therapy, every day after launch. Under-investing here is one of the more expensive mistakes I see. You don't get a second first impression with the handful of centres that will treat most patients.

Hybrid markets

As the name says, these need to be looked at case by case. Depending on the disease and its epidemiology, a market usually labelled "lower potential" can deserve much closer attention, and a market usually labelled "attractive" can deserve much less.

Take thalassemia and sickle cell disease. Both are far more common in Mediterranean countries such as Greece, Turkey and Egypt, which don't always combine the infrastructure and access readiness a gene therapy needs. But with relatively large patient populations and high unmet need, those markets can be commercially attractive and justify serious investment to unlock them.

Flip it around and look at the Nordics. Strong healthcare systems, high ability to pay, and very small thalassemia and sickle cell populations. There, a regional or out-of-country model often makes more sense: one Nordic country as a hub, a light-touch team, and centres that can take referrals from across the region.

My practitioner view: for many rare diseases, Benelux and Switzerland sit in this hybrid group too. Belgium and the Netherlands have strong expertise centres, often linked to European Reference Networks, and payers who will engage seriously with an orphan dossier. But patient numbers per indication are small, and in many diseases you can name the treating centres on one hand. That usually argues for a cluster model run from one affiliate, rather than a copy of the German or French set-up. Luxembourg patients are often treated at centres across the border, which supports the same logic. Switzerland is wealthy and well equipped, but it sits outside the EU system: Swissmedic, not the EMA, decides on authorisation, and reimbursement is negotiated nationally. Plan it as its own market, even if a regional team covers it.

Limited-potential markets

These are markets with underdeveloped healthcare systems, weak infrastructure and little ability to pay for an expensive gene therapy. Together, those factors can make a significant investment very hard to justify.

Staying with thalassemia and sickle cell disease, this group can include countries in Africa and South-East Asia where prevalence is very high. On paper, the potential is huge. In practice, these markets are very hard to reach. Low diagnosis rates mean much of the patient pool is invisible, and mobilising patients for treatment is a challenge on its own.

Ex vivo therapies make it harder still, because they depend on sophisticated infrastructure and a high ability to pay. For these markets the realistic routes are often humanistic access programmes, or waiting for in vivo or second-generation ex vivo approaches whose process improvements and lower cost make the investment sustainable for both the company and local governments and payers.

That isn't a reason to ignore these markets. It's a reason to be honest about what can be done now, and what has to wait for the next generation of technology.

Part 3: Go Alone or Bring in a Partner?

Once a market looks attractive, the next question is how to get in: build your own presence, or work with a local partner.

This choice has changed. As more gene therapies launch, local partners are getting more experienced. The good ones now have real orphan and cell and gene therapy track records, strong relationships with centres and payers, and teams who have done it before. In some markets they offer a better entry path than building a team and infrastructure from scratch.

Going alone gives you control: over how the business runs, how the product is positioned and promoted, and how the centres are supported. Given the complexity and cost of a gene therapy, that control is often worth paying for, especially in high-potential markets. Sometimes the decision is made for you, because no suitable partner exists.

Partnering makes most sense in lower-potential or unfamiliar markets, if a good partner can be found. It's particularly useful where you need local expertise to manage difficult infrastructure, or to negotiate in reimbursement systems that are complex, unusual or still developing.

There is also a third option: out-licensing the product for specific territories. For a small biotech with a single asset, that can be the right call for markets it would never staff properly on its own.

My practitioner view: the choice doesn't have to be the same everywhere. A mixed model, with direct presence in the big markets, a lean affiliate or interim leadership for a cluster like Benelux, and partners or licensees elsewhere, is often the most realistic plan. What matters is that the choice is deliberate for each market, and that you can explain why. If you're weighing the lean option for Benelux, I've written about what an interim Commercial Director for a rare disease launch in Benelux actually does.

Market Entry Is a Launch Decision, Not a Spreadsheet Exercise

Building a commercial strategy for any new therapy is complicated. For a rare disease, and especially a gene therapy, the complexity goes up sharply. You need a detailed understanding of each market and its infrastructure, the patient journey, the access environment, the supply chain and the cost drivers. And you need to be willing to make trade-offs between factors that compete with each other, market by market.

The market entry decision also shapes everything that follows. In May I wrote about why the standard launch playbook fails for rare disease drugs in Europe, and in July about the risks of early access programmes before an orphan launch. Both start with the same question: which countries, in what order, and with what model?

How RareGenetics Helps

Most of the teams I work with know their science and their data well. What they often don't have yet is someone who has taken an orphan product into Europe market by market, and knows where a full affiliate pays off and where a lighter model is smarter.

That's the work we do. We help with orphan drug launch planning across Europe, from market prioritisation and entry model to access and launch readiness, with particular depth in Benelux and Switzerland.

If you're deciding where and how to launch a rare disease or gene therapy asset, I'm happy to talk it through. You can reach me at tom.meuleman@raregenetics.be.