Edition of 16 March 2022

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Editorial

EU support for Ukrainian rare disease patients

As the humanitarian situation in Ukraine continues to deteriorate following Russia’s military invasion of the country in late February, the European community is coming together to address the health needs of people fleeing the war and those who remained in the country. The European Commission issued a press release on 2 March 2022 announcing the activation of a Temporary Protection Directive to grant immediate protection to those fleeing the war in Ukraine, including granting access to medical care. Under this Temporary Protection Directive, Ukrainians and legal long-term residents of Ukraine, including asylum seekers and beneficiaries of international protections, will be provided residency rights, access to the labour market, housing, social welfare assistance, medical or other assistance, and means of subsistence. The Commission has also issued operational guidelines for EU Member States on border managements to facilitate access for rescue services and humanitarian assistance into the EU, including to provide medical assistance. The guidelines indicate that Member States should also set up special lanes at border crossing points to ensure access and return of organisations providing humanitarian assistance to people in Ukraine. The European Commission also added an information webpage as part of its webiste intended for people fleeing the war in Ukraine in order to inform them of their rights with regard to crossing the border into an EU country, their eligibility for temporary protection when applying for international protection, as well as the rights of travel inside the European Union. The webpage is available in English but also in the Ukrainian and Russian langagues.

On 8 March 2022, in another press release, the Commission has outlined the support it is making available to help people fleeing war in Ukraine and the UE countries receiving them. The assistance available amounts to 500 million euros, including 90 million euros in humanitarian aid, notably for healthcare. The Commission announced the activation of the EU Civil Protection Mechanism, allowing the delivery of medical kits to Ukraine and neighbouring countries. The Commission also adopted the "Cohesion's Action for Refugees in Europe” (CARE) legislative proposal, allowing flexibility for EU funds to finance measures supporting people fleeing Ukraine, including investments in health.
On 4 March 2022, the Commission’s Directorate-General for Health and Food Safety held an-information sharing webinar on how civil society and health professionals can mobilise to support medical and public health needs of Ukrainian and displaced persons. The webinar presented EU actions and emerging additional needs, and aimed to prepare for the arrival of refugees from Ukraine. During a visit of a refugee reception centre in Poland, the Commissioner for Health and Food Safety Stella Kyriakides has announced that the EU has set up a system to ensure the safe transfer of patients in need of specific medical care to hospitals across the EU and that 10 000 hospital beds have been made available by Member States so far.

In a solidarity effort, the European Reference Networks have published a statement to support people with Rare Diseases and Complex Conditions affected by the war in Ukraine. The statement informs that about 120 000 Ukrainian rare disease patients have already arrived amongst the 2.5 million refugees in the EU on Friday 11 March 2022 and that at least 6% of the Ukrainian population, including those currently migrating, are affected by a rare disease. The ERNs have launched a new website (accessible in most EU languages, in Ukrainian and Russian) with contact points for each ERN by speciality and a list of members in Ukraine Border countries and beyond as well as a useful links webpage to help find rare diseases European actors. The ERNs also inform that they will support all national and EU public health authorities, the European Commission (including EWRS), WHO, NGOs and any other organisation and healthcare professional, if information is needed for triage or transfer of rare disease patients, by providing lists of healthcare providers which can provide the expert care for specific rare disease patients and providing diagnostic and therapeutic advice if no information is available or if a complex situation occurs. ERKNet has published a list of medical centres available for Ukrainians with rare kidney disease in Poland, Lithuania, Romania and Hungary. A full list of the ERN care centres for rare diseases is available on Orphanet’s website. .

In the same vein, EURORDIS has called on the international community and the EU to protect people with specific medical needs in Ukraine, including the rare disease community. EURORDIS recalls that an estimated 2 million people in Ukraine live with rare disease, and that this population is particularly vulnerable in times of war. Rare disease patients need more frequent care, and the destruction of vital infrastructure, such as hospitals and care centres, has devasting and life-threatening consequences by disrupting essential health services. EURORDIS recalls that efforts from the international community are required to restore and strengthen health services, including access to medicines and medical equipment. EURORDIS announced it will facilitate a coordinated effort with European Rare Disease Federations and will support patient organisations in Ukraine to understand the challenges they are facing and to help policy makers and the international community understand the challenges people living with a rare disease face on the ground. EURORDIS also echoed this message in an article published in the European Medical Journal, where it stated that shortages of medications and supplies were already witnessed for several rare diseases and that a large number of rare disease patients are unable to leave the country. EURORDIS is therefore calling large aid agencies to facilitate moving vulnerable people with rare diseases safely and quickly out of Ukraine and to facilitate groups to legally get essential supplies into the country and Ukrainian authorities to recognise the specialised needs of this population, including the immediate need for exemption for men who have a son, daughter, or partner with a rare disease to leave the country. EURORDIS also created a resource webpage on its website for people living with a rare disease in Ukraine and those who sought refuge in a European country as well as a press kit on how to address the challenges of people living with a rare disease in Ukraine.

In solidarity with the rare disease community in Ukraine, you can join EURORDIS’ urgent call for volunteers to help people with a rare disease. The association Orphan Diseases of Ukraine has also issued a list of medical items needed to support hospitals and patients.

Orphanet has published, and will maintain, a list of rare disease resources useful for refugees and displaced persons.

Spotlight

Rare Disease Day 2022 Round up

To mark the fifteenth edition of Rare Disease Day on 28 February 2022, over 650 buildings were lit up and thousands of events took place in over 100 countries following the launch of EURORDIS and partners’ Light up for Rare Campaign. EURORDIS also launched the 30millionreasons for European action on rare diseases platform. EURORDIS asked European Federations, National Alliances for Rare Diseases and 984 Patient Organisation Members, as well as the wider rare disease community to share their personal reasons for European policy makers to take action on rare diseases. This platform follows on from the publication of the Rare 2030 recommendations on Rare Disease Day 2021 while supporting the 30 million people living with a rare disease in Europe and supports the call for a European Action Plan for Rare Diseases.

At the international level, the NGO Committee for Rare Diseases, the Ågrenska Foundation, Rare Diseases International (RDI), and EURORDIS-Rare Diseases Europe also held a 2022 global Rare Disease Day event entitled "Rare Diseases: A Global Priority for Equity" at the Dubai World Expo on 28 February. The event celebrated the adoption of the UN Resolution on “Addressing the challenges of Persons Living with a Rare Disease and their families” this year. A full report of this event is available in the International News section of this edition.

Another notable event to mark Rare Disease Day was the 2nd International Conference on Rare Diseases held in Athens and online on 1-2 March 2022. The conference, entitled "Rare diseases: the immediate priority of public health" was organised by Association "95", Hellenic Alliance for Rare Patients and Heath Daily, BOUSSIAS under the auspices of the European Parliament and EURORDIS. The conference gathered more than 450 participants from 30 countries, including Greek and European decision-makers. The conference highlighted the need to protect people living with a rare disease in Greece and in Europe, and the need to tackle inequality and discrimination faced by rare disease patients, as well as to achieve equal access to diagnosis and care across Member States. During his speech, Dimitris Papadimoulis, Vice President of the European Parliament, noted the need for: "Immediate strengthening of cooperation in the EU, through European programs and initiatives for research and development. As a European family, we cannot be indifferent to the 6,000 rare diseases with 300 million patients worldwide."

At the European level, Stella Kyriakides, EU Commissioner for Health and Food Safety issued a statement on Rare Disease Day calling for stronger EU cooperation to make sure that all patients across the EU living with a rare disease have access to the highest standards of diagnosis, treatment and care they need. The Commissioner recalled actions taken in the rare diseases field at the EU level, including the planned reform of the legislation on medicines for rare diseases to foster innovation and ensure faster developments and more timely and equal access to medicines for rare diseases. Stella Kyriakides also mentioned the integration of 620 new members to the existing European Reference Networks this year, and the establishment of EU-wide registries as a key element of the Networks' activities. To harness the potential of health data and to create further innovative opportunities for patients to access it, these registries will be integrated into the European Health Data Space that the Commission will present in the coming weeks. The Commissioner also announced the launch a Joint Action later in 2022 to expand the work on integrating the Networks into national healthcare systems. A factsheet recalling EU’s action in the field of rare diseases has also been published by the EU Commission.

Stella Kyriakides stated that she is working together with the EU Presidency towards a stronger EU response on rare diseases, as shown during the ministerial conference organised by the French Presidency of the Council of the European Union on Rare Disease Day. The conference, entitled Ministerial Conference on Innovation and Care Pathways: For a European policy on rare diseases, brought together health ministers of the European Union and the EU Commissioner in order to identify means to increase cooperation on rare diseases at the European level. The conference presented the progress made in the field of rare diseases, includingthe reinforced policy for research on rare diseases thanks to the European Joint Programme on Rare Diseases (EJP-RD), the launch of Orphanet, the steps taken toward establishing the European Health Data Space, and the development of biotechnology companies.

France’s will to act on rare diseases and work towards a European Action Plan on Rare Diseases was demonstrated through the symposium on Rare Diseases organised by the French Institute for Health and Medical Research (Inserm) in the framework of the French Presidency of the Council of the European and under the high patronage of the French Ministry of Higher Education, Research and Innovation. The conference was held on 28 February at the Ministry of Solidarity and Health, Paris, France, with the aim to review the current state of research and the means implemented for the diagnosis, care and treatment of rare diseases, and the expected prospects for increased collaboration between Member States of the European Union, public and private partners, research actors on a European scale. A statement issued by the French Platform for Rare Diseases, including Orphanet, welcomed France’s leadership on rare diseases at the European level, while calling for concrete and renewed-policy actions and goals, including at the national level.

Editorial

Spotlight

European Reference Networks

Orphanet News

Summary of the edition of 16 March 2022

Editorial

Political news

Scientific news

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Editorial

EU support for Ukrainian rare disease patients

Spotlight

Rare Disease Day 2022 Round up

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Summary of the edition of 16 March 2022

Editorial

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