In 2021, Ukrainian father Vitalii Svichinskyi flew with his sick young son to the US for what he hoped would be a life-saving treatment.

His son had spinal muscular atrophy (SMA), a rare genetic disease of the central and peripheral nervous systems that causes progressive muscle weakness and wasting. Children with the most severe forms often have a very short life expectancy.

In 2019, the FDA approved onasemnogene abeparvovec (Zolgensma), the first gene therapy to treat children younger than 2 years with the most severe form of SMA. The European Medicines Agency (EMA) granted conditional marketing authorization in 2020 and full approval in 2022.

Zolgensma delivers a functional copy of the SMN1 gene through a one-time intravenous infusion, targeting the root cause of SMA. It is not the only treatment available for SMA, but it is the first and only gene therapy for the disease.

When it was first approved, Zolgensma carried a list price of $2.125 million, making it the world’s most expensive therapy at the time. In Ukraine, the drug was not registered or approved, and the state did not pay for it. Svichinskyi watched children in other countries receive the treatment and saw his own son, who could barely move, fall further behind.

“I asked the Ministry of Health how we could solve the situation,” he told Medscape News Europe. “They said they couldn’t help, that it costs a lot, and that the drug isn’t registered here. I realized the only way was to start a charitable campaign.”

Within 80 days, his family raised $2 million to buy the therapy from Novartis in the US. Five other Ukrainian families soon launched similar campaigns.

“The Ukrainian government has a responsibility to secure access,” he said. “But my personal feeling is that it’s not normal that a treatment can cost $2 million. Big pharma says they only have a small amount of SMA patients, and investing in this treatment costs a lot of money. When rich countries don’t want to support those children, how can the low-income countries?”

Zolgensma has become a powerful symbol of both the promise and problem of gene therapies. A new generation of advanced therapy medicinal products (ATMPs) offers hope — and sometimes cures — for previously untreatable genetic disorders, yet across Europe, whether a child receives them often depends less on science than on geography and budgets.

Despite EMA approval, access to Zolgensma remains highly uneven. Its patchwork rollout exposes the rules, prices, and political choices that now decide who receives transformative treatments and highlights the need for new regulatory and financing models if gene therapies are to be anything approaching equitable.

One Authorization, Many Markets

In 2007, the EMA adopted a centralized market authorization framework for ATMPs, including gene therapies, to streamline approvals across the EU and European Economic Area. The aim was to accelerate development and avoid a patchwork of national regulations.

But once EMA authorization is granted, it is still up to each pharmaceutical company to decide where to launch a product and up to each country to negotiate prices — often under confidentiality clauses — and set its own reimbursement rules. The result is substantial variation in when, how, and whether patients can access a new therapy.

Currently, Zolgensma has regulatory approval in 37 European countries, and 32 have some form of reimbursement pathway, according to a Novartis spokesperson.

Ellen ’t Hoen, LLM, PhD, a lawyer and public health advocate who has worked on pharmaceutical and intellectual property policies for more than 30 years, said these disparities arise from multiple factors.

“Companies tend to first market their product in a country that will pay the highest prices,” she said. “They have the benefit of entering a single market, and once they have authorization from the EMA, they can market anywhere they like in the EU but they’re not obliged. Then the introduction of a drug happens much later or not at all. Smaller or poorer countries then can’t afford the kind of prices that are being charged.”

Affordability vs Solidarity

Experts interviewed by Medscape News Europe agreed that Zolgensma’s price is prohibitively high — even for many high-income countries. For lower-income countries, the cost of such therapies can strain healthcare budgets and force governments into impossible choices.

“Gene therapies are very expensive, and companies want to maximize profits,” said Panos Kanavos, PhD, professor in practice in International Health Policy at the London School of Economics and Political Science (LSE), deputy director at LSE Health, and director of the Medical Technology Research Group.

“It’s a mix of regulatory challenges, affordability, and uncertainty as to why there are issues around equity and access across Europe,” he said. “It’s also an ethical dilemma. How much is a life worth? If you can save or extend the life expectancy of 100 people vs saving one life with the cost of a gene therapy, what do countries decide? We’re treating individuals like statistics.”

According to ’t Hoen, patent protections and market exclusivity — especially under the EU’s orphan drug regulation, which grants the first approved medicine for a rare disease 10 years of market protection — give companies wide latitude to set prices.

“This all exists because there’s a belief that if you do that, you incentivize innovation,” she said. “But that comes at a very high price. I wonder if we just give them the money directly, it would be a better way of incentivizing innovation.”

A Novartis spokesperson told Medscape News Europe that SMA places a significant burden on health systems across Europe, with estimated healthcare costs per child ranging from €2.5 million to €4 million in the first 10 years of life.

“Our approach is to introduce our medicines at prices that reflect the value they bring to patients, healthcare systems, and society, while striving to ensure that they remain accessible and sustainable within the unique healthcare systems of each market,” the spokesperson said.

“We are continuing to work to develop concepts for sustainable partnerships in countries, including some in Europe, that lack access and that have an interest and willingness to invest in co-creating solutions to broaden access to Zolgensma.”

Alternative Pathways, Limited Safety Net

Some countries reimburse Zolgensma through national health insurance. Others rely more heavily on special mechanisms.

“Zolgensma is reimbursed in some EU countries. But lower-income countries often don’t have the resources to cover such an expensive treatment,” said Margaux Reckelbus, a PhD researcher at the Interfaculty Centre for Biomedical Ethics and Law at KU Leuven in Leuven, Belgium. “It’s unfair that some patients can access a life-changing therapy that’s highly effective, while others cannot. Everyone should have a chance to benefit from it.”

Reckelbus has studied alternative access routes, including hospital exemption and compassionate use programs. These, she said, are not sufficient to guarantee equitable access because they are inconsistently and often inadequately implemented.

For Kanavos, the price of a new gene therapy like Zolgensma also introduces considerable risk and uncertainty.

“For Zolgensma, decision-makers were asked to pay $2 million, but they only had evidence from a single-armed trial of 22 patients,” he said. “In practice, the therapy is proven to be effective, but obviously you cannot know in advance all of the outcomes. It’s a lot of risk. It’s not like an immunization that has been around for decades.”

To mitigate such uncertainty, some countries have introduced risk-sharing agreements that spread payments over time and tie them to continued benefit.

“In some arrangements, a health system might pay 20% of the cost upfront and then pay the remainder only if benefits are sustained over several years,” Kanavos said. “When we don’t know if a therapy delivered today is going to be effective tomorrow and in the next year or two, this is one helpful tool to use. But many countries don’t have it. It’s a regulatory gap that requires legislation to allow health insurance to negotiate with manufacturers.”

Poland, Bulgaria, and the Real-World Price Gap

High-income countries are generally better positioned to adopt risk-sharing agreements, negotiate confidential discounts, and take advantage of early access schemes or traditional submissions to health technology assessment (HTA) bodies.

In 2022, after 2 years of fundraising by families of children with SMA, Poland made Zolgensma eligible for state reimbursement. The country adopted a risk-sharing agreement that limits the financial exposure of the National Health Fund, said Urszula Skarżyńska, MBA, from the Department of Organization and Settlement of Medical Services at the Institute of Mother and Child in Warsaw, Poland.

“The price of Zolgensma is approximately PLN 9 million per dose ($2.47 million; €2.13 million), which is a huge burden on the health budget,” she said. “The government has declared it will maintain the reimbursement, although the availability of the therapy depends on further budget decisions and an assessment of the long-term effectiveness of the treatment. The biggest problem for hospitals is the lack of liquidity in paying for the treatment for patients with SMA and other drug problems. My hospital currently has unpaid treatment for the first quarter of this year.”

Price differences across Europe are stark. Poland is paying about €2.13 million per dose, while in Germany, the drug costs about €1.4 million.

In Bulgaria, there is no specific legal or regulatory pathway dedicated to ATMPs. Such therapies are assessed under the standard national HTA and pricing procedures designed for conventional pharmaceuticals, said Kostadin Kostadinov, MD, PhD, senior assistant professor of social medicine and public health at the Medical University of Plovdiv in Plovdiv, Bulgaria.

“The limited number of accessible gene therapies in Bulgaria reflects both European-wide and national challenges,” he said. “At the national level, Bulgaria lacks an adapted framework for these products, and the administrative and procedural steps before and after HTA can be lengthy. Furthermore, the expectation of curative outcomes sometimes exceeds the observed clinical results, which also influences the cautious national approach.”

In practice, Zolgensma is only obtainable in Bulgaria through direct purchase from the manufacturer after negotiations with the state. An individual reimbursement scheme used exclusively for children allows the Ministry of Health to fund treatment after a case-by-case review. The process involves multiple steps and can be slow — time that young children often do not have. Although Zolgensma has undergone a standard HTA process for general reimbursement under the positive drug list, that decision is currently being contested in court.

“Most patients in Bulgaria rely on the individual reimbursement procedure, which is currently the only feasible mechanism for accessing such high-cost orphan or advanced therapies,” Kostadinov said. “This pathway roughly mirrors similar mechanisms available in countries like Germany or France, where dedicated funds for rare and innovative treatments exist. But in Bulgaria, it remains an exceptional option rather than a universally available right.”

“The unequal or delayed access has serious consequences for patients and families,” he added. “Because these are time-sensitive conditions, the need to go through ad hoc approvals or court rulings can lead to uncertainty and distress. Some families have even resorted to public fundraising campaigns or legal appeals to secure treatment within the therapeutic window.”

Because Zolgensma is only available through such individual schemes, Bulgaria has paid substantially more than many other European countries — about €2.85 million per treatment.

“In these cases, the total public expenditure on each treatment is fully disclosed,” Kostadinov noted, “while in countries like Greece, confidential discount agreements are systematically applied under reimbursement contracts, making the actual net prices significantly lower but undisclosed.”

The Politics of Price Discrimination

For ’t Hoen, Zolgensma’s price variation across Europe illustrates a broader structural problem.

“Companies have such a strong position in having a single market,” she said. “If we could negotiate prices at the EU level, it could drive down prices, but we’d need an EU health policy to do that. Countries are basically on their own and smaller or lower-income countries simply just don’t have the leverage.”

“This is the irony of the pharmaceutical system: On the one hand governments give strong monopoly rights but then must negotiate with companies to get what they want. It’s the foolish way. It’s a huge injustice and we shouldn’t underestimate the prices put on the healthcare system. You can only spend a Euro once. If you spend €3 million on a drug for one person, others will not survive. It undermines the basic solidarity principle on which EU health systems are built. That is a huge problem.”

New EU Tools, Uncertain Impact

Can Europe move toward a fairer system for pricing and access to gene therapies?

At the EU level, experts point to the new joint clinical assessment framework under the HTA regulation, which is being phased in from 2025, as a significant development. The regulation aims to harmonize HTA criteria by conducting centralized EU-level assessments of the clinical effectiveness of new health technologies, including ATMPs.

Unlike the EMA’s regulatory process, which focuses on safety, quality, and efficacy for market authorization, joint clinical assessments will provide a shared evidence base on added clinical benefit to inform national reimbursement decisions.

“This will help countries decide where to spend money,” ’t Hoen said. “What are the most effective treatments? What is cost-effective? It won’t necessarily lower prices, but it can lead to more rational spending of limited resources. How would you otherwise base your reimbursement decisions?”

The proposed “Pharma Package” is another major initiative. It aims to modernize the EU’s medicines framework, improve timely and fair access to medicines — particularly for orphan diseases — and support innovation and competitiveness.

’t Hoen said the package includes incentives for companies to bring drugs to market across all EU member states. “Whether that will survive will remain to be seen, but it’s on the table and in the late stages,” she said. “But it doesn’t say that you have to bring to the market a drug that is affordable. Encouraging companies to market drugs isn’t the same as providing access to it. Beyond this, of course, is the issue of how the pharmaceutical company can determine the price of a drug or therapy.”

Collective Negotiations and Cross-Border Care

Some groups of countries have already started to work together to improve their negotiating position.

Under the Beneluxa initiative, Belgium, Netherlands, and Ireland negotiated a confidential agreement with Novartis for access to Zolgensma, which will be reimbursed by the respective health insurance systems.

“Cross-border cooperation on data collection, risk sharing, and possibly pooled financing for ultraexpensive therapies would further improve equity and sustainability of access across member states,” Kostadinov said.

Such country groupings remain the exception rather than the rule. Still, Kanavos believes deeper integration is possible.

“It’s not out of the question that the EU could move to a single market for drug pricing in the next 15 or 20 years,” he said. “The problem is that countries have their own health policy and budget. It’s difficult to agree on a single price for a drug.”

For now, existing EU mechanisms such as the cross-border healthcare directive offer partial relief. The directive allows patients to receive treatment in any EU or EEA country and be reimbursed by their home country, though implementation varies widely.

“The EU is working on new measures to make this framework more robust so more countries can actually implement it in practice,” Reckelbus said. “I feel the EU does a lot and does take into account the problem [of unequal access to drugs], but they don’t have the power; they can’t do everything.”

A Worrying Future for High-Cost Therapies

Looking ahead, ’t Hoen is concerned about the trajectory of access to very high-cost medicines.

“What you’re going to see in the coming years, because of the drive to put more public financing toward the military and security, is more pressure on healthcare resources,” she said. “Healthcare costs are rising due to aging populations. Are all these things going to put pressure on drug prices, or will they further rationalize access?”

For families like the Svichinskyis, the stakes are immediate and intensely personal. For European policymakers, Zolgensma is a test case for whether health systems built on solidarity can adapt to the era of multimillion-dollar cures — and whether children’s chances at those cures will continue to depend on the country into which they are born.

Svichinskyi, ’t Hoen, Kanavos, Reckelbus, and Kostadinov reported having no relevant financial relationships.

Sophie Cousins, MIPH, is a global health journalist who has reported from more than 20 countries.