Three years ago, we started a project with Orchard Therapeutics and PTC Therapeutics to map the challenges patients with an ultrarare disease face when they need to travel abroad to get their treatment with cell or gene therapy (Advanced Therapy Medicinal Products, ATMPs).
We unraveled the complex European legislature and revealed practical challenges in collaboration with key stakeholders (payer, regulator, healthcare providers, patient advocacy groups, sick funds). We identified challenges related to
Since our report has been published, we have had different opportunities to present our analyses. It is striking that many people are not aware of the problem or do not even consider it a problem.
However, after explanation of the challenges most people are quickly convinced that a solution is needed. Several people also noticed that clinical trials face similar challenges. For example, the clinical costs in their home country - during follow-up or when complications occur – are not covered within the trial nor by their national healthcare system. This is one of the challenges EU-X-CT is looking into.
We were pleased to notice that the cross-border issue was one of the topics at the World Orphan Drug Congress 2025. Giorgia Crimi from Fondazione Telethon referred to our report and complemented our findings with insights from clinical practice.
To cover the cost of the gene therapy and the hospitalization costs, European Social Security Regulations (883/2004 and 987/2009) are in place to get reimbursement for ATMP treatments abroad. Within this Regulation the patient needs prior authorization to receive treatment outside the Member State of residence. Authorization by the competent institution should be given when following conditions are fulfilled:
Even when there is theoretically little room for discretionary decisions if these conditions are fulfilled, Giorgia Crimi showed that not all patients get approval from their national authorities or only with delays, or advocacy efforts. From the 24 patients referred to San Raffaele hospital from 24 different EU countries, only 10 patients got approval from their national authorities without major issues, 6 patients with delays or advocacy effort and 8 patients did not receive the treatment due to concerns or because they opted for an alternative treatment. Follow-up of these patients is essential, but national approval was denied or delayed in all 4 cases. This approval from national authorities is required to get the treatment and related costs reimbursed.
However, only treatment and hospitalization costs can be covered by Regulation. Patients also face travel costs when residing for a period of time abroad to receive the treatment, which are not covered by the Regulation, but are sometimes covered by national funds. Fondazione Telethon has set up the “Just Like Home” project to cover travel and lodging costs for patients to give them sufficient support during the treatment. This includes both medical support as well as administrative, cultural and psychological support. This is an example of how these patients should be taken care of during treatment abroad and what is often still missing.
The numbers presented by Fondazione Telethon made it clear that borders still prevent patients with (ultra)rare diseases to get access to gene therapies. Currently about 20 gene therapies are EMA approved and still on the market, and many more are expected to come to the market in the coming years. So even if an ad hoc solution might still work now, we are still strongly convinced that a more sustainable systemic solution is required to solve cross-border-related challenges in the context of ATMPs.
Do you want to know more? Read our report where we drafted a solution framework.
Do not hesitate to reach out for more information or if you would like to discuss and work towards concrete next steps.
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