Don’t BREXIT on Rare Disease Patients

Primary immunodeficiencies (PID) are disorders in which part of the body’s immune system is missing or does not function normally. A forum entitled ‘Don’t Brexit on rare disease patients – the case of primary immunodeficiencies’ took place in the European Parliament on 24/01/17.  This forum was organised by International Patient Organisation for Primary Immunodeficiencies (IPOPI) and was hosted by MEPs Carlos Zorrinho and Seb Dance.  At the forum, contributions were invited from policy makers, MEPs (including Ireland’s Ms McGuinness and Ms Harkin), patient associations and medical experts.  The forum aimed to ensure PID care at European level does not suffer from the Brexit.

Why did the Irish Primary Immunodeficiency Association (IPIA) attend?

If Brexit results in the UK being removed from the E112 ‘’Treatment Abroad Scheme’, European patients may not be able to access treatments in the UK. The IPIA attended this event to outline how Irish primary immunodeficiency patients currently rely on the UK.

Ireland currently uses labs in the UK for rare tests including; functional T-cell studies, proliferations, special diagnostics and complement studies. Ireland also sends many paediatric patients to the UK for treatment every year.  Every year we send patients with severe primary immunodeficiencies for Haematopoietic stem cell transplantation (HSCT or “bone marrow transplantation”), Thymic transplantation for athymic diGeorge syndrome and Lentiviral gene therapy for ADA-SCID. Ireland also has strong links with the UK in terms of clinical trials, research and immunology training. The Irish PID situation is echoed by many countries throughout Europe who rely on the specialised centres of expertise that have been developed in the UK.

What would IPIA like to see happen in the future?

Ireland has capacity to develop bone marrow transplantation for severe primary immunodeficiencies. In fact, the first paediatric transplant happened here last year. We do however need further significant investment and support from the Irish government to achieve this.

Due to our population size, we will still need to send patients abroad for both thymic transplantation and gene therapy. The IPIA would like to see ongoing access to thymic transplantation and gene therapy in the UK if possible. If this is not possible, then an alternative centre in the European Union with facilities and measures to allow families to travel and patients to receive their care in comfort. We would also like to see ongoing collaboration with the Immunology community in the UK, given their expertise in research, both basic and clinical.

What will happen next?

IPOPI will use the information shared in the forum to create a set of recommendations to support the Brexit discussions. The IPIA will continue to seek support from other patient organisations and policy makers. We want to ensure that future PID patients can access the treatment they need and do not suffer because of the UK leaving the European Union.

For more information please go to.