5.7 C
Friday, February 23, 2024
EuropeEU cross-border healthcare

EU cross-border healthcare

DISCLAIMER: Information and opinions reproduced in the articles are the ones of those stating them and it is their own responsibility. Publication in The European Times does not automatically means endorsement of the view, but the right to express it.

DISCLAIMER TRANSLATIONS: All articles in this site are published in English. The translated versions are done through an automated process known as neural translations. If in doubt, always refer to the original article. Thank you for understanding.

The European Times News aims to cover news that matter to increase the awareness of citizens all around geographical Europe.

One truth is certain for all rare diseases, but especially for severe genetic ones: the faster patients get the right treatment, the better their chances of living a full life or potentially even being cured.

Time can be a precious luxury to someone diagnosed with a rare genetic disease, so getting timely access to new innovative therapies is critical.

Rare Disease Day, an annual event that is marked each year on the last day in February, is a unique occasion to increase awareness of rare diseases that affect an estimated 30 million people in the EU. But it is also an occasion to shine a light on the transformative scientific achievements of gene and cell therapies that are bringing new hope to patients who previously had few, if any, treatment options.

However, although the EU’s approach to cross- border healthcare is a critical patient access pathway for patients with rare and ultra-rare genetic diseases, it is not fit for purpose, and this is becoming increasingly clear as more and more gene therapies come to market.

Cross-border healthcare – a vital path for rare and ultra-rare disease patients

The reality for many patients and their families is sobering. Gene therapies and other Advanced Therapy Medicinal Products (ATMPs) require specialist treatment processes. They cannot be administered in an average hospital setting. Instead, highly specialised, accredited treatment centres are needed, staffed by specialist doctors and nurses who need dedicated training.

Furthermore, the disease expertise and specialist skills that are required may not be widely available in every European country. This makes a functioning cross-border healthcare framework at the EU level essential to provide patients with access to life-saving therapies. The European Commission’s evaluation of the Directive provides an opportunity to examine the current and future needs of patients in cross- border healthcare with both pathways in mind.

A complex and little-known framework that fails to address the specificities of gene and cell therapies

Patients whose very survival depends on therapies that can only be delivered abroad must face a flurry of challenges to access the treatment that will hopefully change their lives forever. The current EU framework is composed of two pieces of legislation which each lay out a different, but equally complex, patient journey: The ‘Directive on Patients’ Rights in Cross-Border Healthcare’ and the ‘Regulation on the Coordination of Social Security Systems’.

In addition to the limited awareness among clinicians and patients of these two pathways, and of the Regulation especially, the journey from diagnosis to treatment is marked by a labyrinth of burdensome and time-consuming processes. These negatively affect patient mobility and could ultimately affect their outcomes.

Furthermore, both pathways are impacted by several challenges that systematically prevent patient access to treatment. The current design of the Directive requires upfront payment by the patient, an unrealistic expectation highlighted by the European Court of Auditors, which is exacerbated in the case of high value, one-time gene therapies.

In effect, this has made the so-called ‘S2 pathway’, provided by the Regulation, the only viable route for patients seeking access to gene and cell therapies in another EU member state.

However, the S2 pathway is not without flaws. Patients using it to seek planned treatment abroad will face a complex approval process with several hurdles to overcome. Moreover, approval timelines vary by country, ranging from a few weeks to several months, presenting an obstacle to accessing urgent, potentially life-saving treatment.

The approval process if the treatment is not directly covered in the patient’s home country is discretionary and extremely opaque. This leads to unequal patient access across the EU. In addition, innovative payment and risk-share models are challenging to implement in a cross- border healthcare context.

What can be done?

In the short- to medium-term, to increase awareness among patients and healthcare professionals, it is critical that a unified single source of reliable, user-friendly and accessible information is established, to navigate the cross- border healthcare framework for all stakeholders (ie, a dedicated EU website).

The current challenges patients face when seeking access to an approved gene therapy or other ATMP could be partially addressed by a
set of practical changes to improve the current framework. This includes removing the burden of upfront payment from patients by enabling direct billing between health institutions.

The development of Commission guidelines that set harmonised review and approval timelines that will expedite time-to-treatment in the EU
will help address, in part, the shortcomings of the S2 pathway. Furthermore, to make more rapid and transparent approval decisions around this, national health authorities could draw on existing joint Health Technology Assessment initiatives, such as EUnetHTA.

It’s time for action

Rare Disease Day gives us the opportunity to look at the achievements of science, but it’s also important to look at what still needs to be done to help patients get access to new and innovative treatments.

There’s a real opportunity for the European Union to act as a ‘one-stop shop’ to increase process transparency and help patients, clinicians and national health decision-makers navigate the current complexity of the cross-border healthcare system.

In the last few years, the promise of gene and cell therapies has finally become tangible for certain patients and their families with a severe genetic and rare disease. But this promise will only become a reality if patients can gain rapid access before their disease progresses to a point of no return.

The clock is ticking for all stakeholders, from patient associations and clinicians to policymakers and industry, to work together to overcome today’s hurdles of cross-border access for rare and ultra-rare diseases. Only then can we ensure that rare disease patients in Europe can benefit – regardless of where they live.

Longer term, the EU should consider new, more ambitious policies, such as the establishment of a pan-EU solidarity fund from which member states could draw to fund access to gene therapies for rare and ultra-rare disease patients. This would be in keeping with the vision of the European Commission to create a healthier EU as announced in the EU4Health plans in May 2020.

- Advertisement -

More from the author

- Advertisement -
- Advertisement -
- Advertisement -spot_img
- Advertisement -

Must read

Latest articles

- Advertisement -