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What are 'unproven' therapies?

Broadly speaking, an ‘unproven therapy’ is one which has not received full regulatory approval for a specific use. The term covers investigational therapies, which are being assessed for safety and efficacy, unregulated therapies, which have not gone through this process, and compassionate use, a rare case where a therapy is made available to specific patients before it has completed the approvals process.

Travelling to access medical care

Not all health services are equipped to deal with rare diseases, and not all therapies approved by the EMA are widely available in all EU countries. Patients may therefore consider travelling outside their country of residence for treatment.

European Reference Networks

A patient with a rare or complex disease may struggle to find a healthcare provider with relevant clinical expertise. To address this, the EU created European Reference Networks (ERN) in 2017. These are networks of institutions and healthcare providers. They are organised across borders, facilitate the sharing of knowledge, resources and expertise throughout the EU. The goal is to lead to improved care for people living with rare disorders.

Under a 2011 EU directive, EU citizens are entitled in certain cases to travel to other EU countries for medical care and reimbursement. ERN are encompassed by this directive. Your healthcare provider will be able to discuss with you whether travelling for treatment is an option in your case, and what it will mean logistically for your future care if you do. The decision will be informed by the expertise of the ERN, but ultimately will be made between you and your clinician.

Other travel for treatment

If you are travelling to take part in a clinical trial in another country, to receive an unapproved/unregulated therapy, or to receive a therapy which is approved in another jurisdiction – for example, travelling to the United States of America for a therapy approved by the FDA (Food and Drug Administration) but not by the European Medicines Agency – we recommend that you discuss this decision with your local consultant. It is important that you know what follow-up care you will be entitled to in your own country. It is also important for your healthcare that your consultant is informed of any other treatments you are receiving, so that they can give you the most appropriate care possible.

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