Treatments for rare diseases, such as gene therapies, are complex, requiring a high level of clinical expertise and often administration in a specialist center. As these diseases are not common, treatments and specialist centers are not accessible in every country in Europe. Patients with rare diseases may therefore have to cross geographical borders to receive a potentially life-transforming – or even life-saving – treatment that is not accessible in their home country.

Pathways to accessing care abroad

In Europe, patients have the right to request care in another country outside their home country and to be reimbursed for all or part of its cost. The European framework and legal basis for cross-border healthcare is enshrined in two pieces of EU law:

  • Directive 2011/24/EU
  • Social Security Regulations 883/2004 and 987/2009.

These pathways allow patients in the European Union (EU) or European Economic Area (EEA, which is made up of Iceland, Lichtenstein and Norway)), including those with rare diseases, to access medical diagnosis, treatment and prescription in any EU/EEA country and Switzerland.

The practical option

The cross-border healthcare system works well for common treatments for accidents and short illnesses while on holiday, or for routine procedures, such as hip replacements.

If using Directive 2011/24/EU, patients must pay for treatment themselves and then request reimbursement from their home country. This pathway is not usually practical for patients with rare diseases, due to the cost of specialist treatment.

The more commonly used pathway for patients with rare diseases is Social Security Regulations 883/2004 and 987/2009, where costs are paid directly by the home country to the country where the treatment is taking place. However, this pathway is complex and less well known by patients, physicians and healthcare providers and accessing care using this pathway can be a challenging and lengthy process with no guaranteed outcome.

Hurdles and delays

A major hurdle with the Social Security Regulations pathway is that it requires prior authorization before treatment can take place. Patients must apply for authorization by completing a European S2 form. This process is complex and can take several months.

Time delays to receiving treatment will have significant – even potentially fatal – consequences for patients with rare diseases.

Ultimately, the national health service or health insurance in the home country may not grant authorization for medical treatment. This refusal may be because the treatment is not approved or reimbursed in the home country, despite being approved and reimbursed in the treatment country, or because pricing models do not consider costs for cross-border healthcare being borne by payers in the home country.

Working to improve cross-border healthcare

PTC is working with industry peers and patient organizations to ensure that all patients with rare diseases have access to reimbursed treatment, no matter where in the EU/EEA and Switzerland the treatment is available, or specialist center is located. We are encouraging the European Commission to review cross-border healthcare legislation with the rare disease community in mind, to make accessing treatment quicker and less complex. We support the need for European-wide guidelines to ensure harmonized access to treatment across borders.

We also believe there should be better, practical information provided to all stakeholders on how cross-border healthcare can be accessed and last year we produced a user guide to applying for cross-border healthcare in Europe.

We need to blaze a trail, to find a solution with payers and healthcare providers in all countries in Europe. A practical solution must be found to ensure patients with rare diseases have access to innovative treatment no matter where they live.

Listen to Thomas Bols’ EUCOPE podcast on cross-border healthcare