• Call to action for policy makers: How to protect people living with a rare disease as confinement measures are being lifted (10 June 2020)

• Rare disease community appeals to EU & member states to move into a new era of collective decision making in health (14 May 2020)

• 9 in 10 people living with a rare disease experiencing interruption in care because of COVID 19 (4 May 2020)

• EURORDIS open letter to policy makers: Recommendations to protect people living with a rare disease during the COVID-19 pandemic (8 April 2020)

• Rare disease community raises alert over discrimination in critical care guidelines during COVID-19 pandemic, EURORDIS urges immediate action and proposes concrete solutions (31 March 2020)

• NORD/ EURORDIS-Rare Diseases Europe Joint Statement on COVID-19 and Orphan Drug Legislation (30 March 2020)

• EURORDIS urges EU not to follow in footsteps of US with orphan designation of COVID-19 treatments (25 March 2020)

• A message from EURORDIS regarding the COVID-19 pandemic (17 March 2020)

• Statement: European Conference on Rare Diseases & Orphan Products 2020 moves online (12 March 2020)

• Information sources on Kawasaki disease

EURORDIS:

Different vulnerability levels will probably guide the prioritisation of people to vaccinate.

Age is certainly a criteria that will help define the most at risk populations in whom the initial benefit/risks will be positive with no dispute.

The number of conditions also increases the risk of being hospitalised.

Therefore, all organisations representing rare diseases are questioning whether the people they represent should be defined as vulnerable groups who should be vaccinated soon, or very soon.

Even when not immediately described by one of these conditions, risks could be increased. For example, people with Osteogenesis imperfecta have restricted pulmonary functions (due to scoliosis and shape of the rib cage), so if infected with the coronavirus, the disease might be worse than a normal person.

EMA:

The EMA is not involved in decisions on prioritisation of groups for vaccination. For patients with certain conditions, the risk of developing serious complications with COVID-19 may be increased. Unfortunately, there may not be comprehensive knowledge of the course of COVID-19 in patients with rare diseases and we recommend following the recommendations provided by national authorities and healthcare professionals. It would be logical to apply a precautionary principle when taking clinical decisions, for example for very fragile or vulnerable patients the prescriber will need to consider the benefits and risks of vaccination versus the individual risk of contracting COVID-19 disease.

EURORDIS:

Vaccines should first be authorised for adults, then data will be available for adolescent from 16 to 18 years of age, and then for younger citizens. It would be important to provide families with estimated timelines on when results in younger age groups will be available to the EMA COVID-19 Task Force, and when the CHMP could extent the indication to these age groups. Some adults living with a rare disease might opt to defer the vaccination for some reasons explained below, but to protect them, it could be important to propose the vaccine to their children, when available.

EMA:

At present it is not known whether and when vaccines will be authorised for use in children. Some currently ongoing trials are enrolling some adolescents. In general, the use of COVID-19 vaccines in children will be investigated once there is sufficient information from studies in adults and in adolescents above 16 years of age. EMA’s Paediatric Committee (the PDCO) can grant deferrals of studies in paediatric investigation plans (PIPs) for some medicines. Deferrals allow a company to delay development of the medicine in children until, for instance, there is enough information to demonstrate its effectiveness and safety in adults. Even when studies are deferred, the PIP includes details of the paediatric studies and their timelines. The PIPs for each vaccine will be published and will include these timelines. Existing PIPs are linked below:

• The date of completion of the PIP for Pfizer-BioNTech BNT162b2 is July 2024

• The date of completion of the PIP for Moderna (mRNA-1273) is December 2024

EURORDIS:

Prioritisation should recognise that a healthy older person who can shelter in place is at different risk from a medically vulnerable older person in crowded housing. It would be important to analyse the efficiency of the vaccines in different settings.

EMA:

EMA is not involved in setting priority groups for receiving the vaccine. These are under the remit of the relevant national authority, who will consider many factors including the need to protect groups of people at higher risk. As mentioned earlier, we would recommend EURORDIS to address this with the relevant body setting immunisation policies in the member state of interest.

The safety and efficacy of the COVID-19 vaccines are studied in different populations, including different age groups, ethnicities and genders as well as different co-morbidities. The effect of different environmental settings as such has not been specifically addressed, although some trials have enrolled individuals at higher risk of exposure to the virus, such as health care workers.

Studies in specific populations not sufficiently included in trials and longer-term studies will continue after authorisation, as for all medicines. Post-authorisation observational studies of effectiveness will show how well the vaccine works in real-life settings (i.e. outside the controlled setting of clinical trials) and on specific populations, such as in people with certain diseases or living in long-term care facilities for example, which could provide useful information with respect to your question.

EURORDIS:

Some expressed concerns about a supposed “collusion between the pharmaceutical industry and governments”, and other interests than public health would prevail. EURORDIS does not subscribe to this theory, and stresses the importance to be able to choose between different vaccines, after discussing the different options with your own doctor, instead of a mandatory vaccination campaign with one product only. This might not be in the remit of the EMA, however it could be useful to publish a comparative information leaflet, that would combine the elements of the different package leaflets.

EMA:

Many different types of vaccines for COVID-19 are under development and it is expected that some would be more suited to certain populations. We agree that it would be desirable if patients, including those in the rare disease community could choose among different options. This however is not within EMA’s remit and is something we would advise EURORDIS to discuss at national level with bodies responsible for immunisation policy.

Medical advice regarding a choice between the different available vaccines should be made in consultation with the healthcare professional who knows the medical history of the patient and is therefore best placed to provide advice. In addition, the risk of exposure to COVID-19 disease will probably be taken into account. The prescribing information and package leaflet, which are approved as part of the marketing authorisation, are useful to support this decision and consultation.

As for other medicines, EMA will not be able to provide a comparative information leaflet as part of the marketing authorisation. It is not in the remit of the EMA to perform comparative analysis across medicinal products, however this can be done by public health bodies at national or European level.

EURORDIS:

There is a growing concern in several rare diseases groups around the AstraZeneca vaccine (and it will  probably be the same with the Janssen one also) because of the use of adeno-associated virus as viral vector. Some people realized this could be an issue for a potential eligibility for a gene therapy if the gene therapy uses same AAV vector due to immunogenicity.

EMA:

We cannot provide definitive answers at this stage because the assessment of the first 2 COVID-19 vaccines based on adenoviral vectors is currently ongoing. The risk of developing immune responses against the vaccine vector is under consideration and will be discussed in light of the available evidence. Theoretically such immune responses might affect how well people may respond to subsequent doses of the vaccine. If such a risk is found, it will be adequately reflected in the EPAR documents. Any contraindications, special warnings or undesirable effects will also be detailed in the prescribing information and package leaflet.

Knowledge on use of adenovirus-based vaccines is also available from other adenovirus-based vaccines currently authorised in EU, most recently the Ebola vaccine Zabdeno. In that context, pre-existing immune responses against adenovirus type 26 (Ad26) used in Zabdeno were assessed in several clinical trials, and in the UK and US studies antibodies were present in few participants (3% - 13%), at low titres, prior to vaccination. Such pre-existing immunity was found not to have any impact on the Ebola-specific antibodies and cell-mediated responses triggered by the vaccine.

EMA:

Allergic reactions (hypersensitivity) have been seen in people receiving the two authorised vaccines (both mRNA vaccines), and a very small number of cases of anaphylaxis (severe allergic reaction) have occurred. Therefore, as for all vaccines, COVID-19 vaccines should be given under close medical supervision, with the appropriate medical treatment available. The product information for the vaccines also states that people should be closely monitored for at least 15 minutes following vaccination. In addition, people who have a severe allergic reaction when they are given the first dose should not receive the second dose. More detailed guidance for healthcare professionals and general public on how to manage potential allergic reactions during the administration of the vaccine will be made available by authorities responsible for immunisation policy at national level.

EMA is evaluating COVID-19 vaccines in the context of a conditional marketing authorisation. This has all the safeguards and controls in place to enable thorough post-authorisation safety monitoring through the collection of additional data in a planned manner. Such safety monitoring will take place more frequently and will include activities that apply specifically to COVID-19 vaccines. Companies for example will provide monthly safety reports in addition to the regular updates required by the legislation and conduct studies to monitor the safety and effectiveness of COVID-19 vaccines after their authorisation. In addition, [independent studies https://www.ema.europa.eu/en/human-regulatory/overview/public-health-threats/coronavirus-disease-covid-19/treatments-vaccines-covid-19#post-authorisation-section] of COVID-19 vaccines coordinated by EU authorities will give more information on the vaccine’s long-term benefit and safe use. These efforts should identify any risks in a timely manner and provide further information on more vulnerable populations, although it may not be possible to address all the existing diseases.

EURORDIS:

As fever seems to be a common side effect to all COVID-19 vaccines, and fever can trigger seizures in patients with rare or common epilepsies, appropriate and close monitoring of this side effect might be needed for this group as part of the pharmacovigilance activities.

For Dravet syndrome in particular, one question is about vaccines using the mRNA technology, and to which extend this could interfere with antisense therapies also based on the mRNA technology.

EMA:

Many patients with rare diseases, such as Dravet syndrome, will not have been specifically studied in COVID-19 vaccine clinical trials. There is also no data available on whether mRNA vaccines might potentially interfere with mRNA therapies. It would be important to highlight all these concerns to the healthcare professional/prescriber to ensure the patient’s specific conditions can be taken into account.

Fever is a side effect of the authorised COVID-19 vaccines. Once a vaccine is authorised and starts to be used in the general population, additional data will be generated on the vaccine’s side effects, as well as long-term safety and benefit, both via safety studies and enhanced safety surveillance. This will help further informing on the side effects and use in special populations.

EURORDIS:

Hereditary Haemorrhagic Telangiectasia (HHT), rare cancers, immune deficiencies, lupus: blood transfusions and immuno-suppressant are standard of care. Will patient need to stop their treatments before receiving the vaccine, and how long before? When can they resume their treatments safely? Would the recommendation apply to all vaccines equally?

The same questions for Auto-inflammatory diseases: is there a need to stop anti-inflammatory treatment before? How long before, when can they be started again?

Rare cancers, melanomas: trials explicitly excluded people with a history of cancer, therefore no data about how effective and safe the vaccines are in people with any kind of e.g. (rare) melanoma. Immunotherapy is a treatment modality that is being used on rare melanomas and although immune checkpoint inhibitors do not cause immunosuppression, they can cause immune-related adverse effects in organ systems. This could constitute a risk regarding vaccination-related complications.

EMA:

The efficacy, safety and immunogenicity of the COVID-19 vaccines so far authorised have not been assessed in immunocompromised individuals, including those receiving immunosuppressant therapy. The efficacy of a vaccine may be lower in immunosuppressed individuals. However, although immunocompromised people may not respond as well to the vaccine, there are no particular safety concerns for the authorised vaccines.

As we have noted, there is limited data in many disease-specific settings. We would refer patients with specific diseases to the product information of each vaccine. EMA cannot advise on the management of specific pathologies beyond the information which is available in the product information.

In addition, questions on clinical practice are outside the remit of EMA. Since many rare disease patients will not have been sufficiently studied in clinical trials, it is important to highlight all these concerns to the healthcare professional/prescriber to ensure specific conditions can be taken into account. Questions concerning patient’s current treatment for existing conditions, whether they should stop before vaccination or when to resume should be discussed with their healthcare professional based on the information in the product information and in line with available advice at national level.

EURORDIS:

In the past, some concerns were shared by a large part of the population and by many healthcare professionals on the possible neuro-toxicity of some vaccines, e.g. vaccine for Hepatitis B infection. Even if these risks were further analysed and finally, the perception prevails in the population.

Are specific monitoring measures planned for patients with neurological diseases, to characterise these risks, or the absence of risks?

People who had an episode of Guillain Barre syndrome for example often renounce to vaccines, and struggle to access the information they need.

EMA:

This has been brought to the attention of EMA’s safety committee (the Pharmacovigilance Risk Assessment Committee, PRAC) for consideration during assessment of the Risk Management Plan (RMP).

Safety data will be collected through spontaneous systems and observational studies, both of which will allow for stratified analyses, detailed in EMA’s Pharmacovigilance plan for COVID-19 vaccines.

EURORDIS:

The Federation indicated some information people with haemophilia will need:

Warnings about people with bleeding disorders to alert on the possibility of an intramuscular haematoma. One measure could be too recommend applying pressure or giving factor replacement coverage to prevent bleeding.

People with bleeding disorders should still be vaccinated according to their priority group (based on age, co-morbidities and healthcare work).

The vaccine should be administered with a thin needle and very slowly.

Having a bleeding disorder should not be a contra-indication to receiving the COVID-19 vaccine.

Any vaccines using AAV can trigger AAV immunity and should only be given to patients who are informed about, and consent to the risk of never being eligible for AAV-based gene therapy

EMA:

There are no contraindications for the COVID-19 vaccines authorised so far except for hypersensitivity to the active substance or to any of the excipients.

As with other intramuscular injections, the authorised vaccines should be given with caution in individuals receiving anticoagulant therapy or those with thrombocytopenia or any coagulation disorder (such as haemophilia) because bleeding or bruising may occur following an intramuscular administration in these individuals.

The Agency is not be able to advise on the management of specific pathologies such as haemophilia beyond the information which is available in the product information.

Also questions on clinical practice are outside the remit of EMA. Since many rare disease patients will not have been sufficiently studied in clinical trials, it would be important to highlight all these concerns to the healthcare professional/prescriber to ensure specific conditions can be taken into account.

EURORDIS:

The paediatric investigation Plans for vaccines for COVID-19 are due in 2024. Children living with a rare disease are vulnerable populations as probably at higher risk of severe COVID-19. They might not want to wait until 2024. Which clinical development is planned? Are clinical efficacy and safety data needed (long development), or would immunogenicity data followed by for example an open label safety study suffice?

EMA:

PIPs are due for 2024, but this is for the youngest part of the paediatric population, children of less than 2 years. For children first from 12 to 18, and then younger ones, immunogenicity data are requested and no large scale long duration clinical trial are requested. Immunogenicity studies are already in progress. This population could benefit from a vaccine before the end of 2021.

It could take more time for the youngest ones, but for the very young (newborn and less than 2), it is unsure a vaccine is needed.

• Health Security Committee - Audio meeting on the outbreak of COVID-19 Summary Report (5 May 2020)

• Coronavirus: Commission issues guidance to mitigate clinical trial disruption in the EU (28 April 2020)

• Guidelines - Guidance on the management of clinical trials during the COVID-19 (coronavirus) pandemic (28 April 2020)

• Crisis preparedness and response / COVID-19 - Health Security Committee Audio meeting on the outbreak of COVID-19 summary report (27 April 2020)

• Coronavirus: European roadmap shows path towards common  lifting of containment measures (15 April 2020)

• Coronavirus: Commission issues guidelines on testing (15 April 2020)

• Notice to stakeholders: Questions and answers on regulatory expectations for medicinal products for human use during the COVID-19 pandemic (10 April 2020)

• Coronavirus: Commission calls on Member States to optimise supply and availability of medicines (8 April 2020)

• Coronavirus: Commission encourages and facilitates cross-border treatment of patients and deployment of medical staff (3 April 2020)

• Commission launches "COVID-19 Clinical Management Support System"(24 March 2020)

• EU CCP Database - Covid-19 convalescent plasma collection and transfusion in the EU

• COVID-19: how EMA fast-tracks development support and approval of medicines and vaccines (4 May 2020)

• Reporting suspected side effects of medicines in patients with COVID-19 (24 April 2020)

• Launch of enhanced monitoring system for availability of medicines used for treating COVID-19 (24 April 2020)

• COVID-19: reminder of risk of serious side effects with chloroquine and hydroxychloroquine (23 April 2020)

• EMA establishes task force to take quick and coordinated regulatory action related to COVID-19 medicines (9 April 2020)

• EU authorities agree new measures to support availability of medicines used in the COVID-19 pandemic (6 April 2020)

• COVID-19: chloroquine and hydroxychloroquine only to be used in clinical trials or emergency use programmes (1 April 2020)

• Draft points to consider on implications of Coronavirus disease (COVID-19) on methodological aspects of ongoing clinical trials (25 March 2020)

• Global regulators map out data requirements for phase 1 COVID-19 vaccine trials (24 March 2020)

• COVID-19: Beware of falsified medicines from unregistered websites (24 March 2020)

• Guidance to sponsors on how to manage clinical trials during the COVID-19 pandemic (20 March 2020)

• European Medicines Agency gives advice on the use of non-steroidal anti-inflammatories for COVID-19 (18 March 2020)

Australia

• COVID-19 pandemic: critical care guidelines for Australians living with a rare disease (Rare Voices Australia, 8 April 2020)

Austria

• Unmet Needs of Rare Disease Patients during the COVID-19 Pandemic - ECRD Poster (Pro Rare Austria)

• Statement, survey and press release (Pro Rare Austria)

• Statement zur Versorgung von Menschen mit seltenen und/oder chronischen Erkrankungen in Zeiten von COVID-19 (Pro Rare Austria, 17 April 2020)

• Auswirkungen der COVID-19 Krisensituation für Menschen mit seltenen und/oder chronischen Erkrankungen in Österreich - Situationsanalyse (Pro Rare Austria, 15 April 2020)

Belgium

• Position de RaDiOrg sur les soins aux personnes atteintes de maladies rares pendant la crise du corona (RaDiOrg, 3 April 2020)

• RaDiOrg met en garde contre la discrimination dans les soins et le traitement des patients atteints d’une maladie rare et complexe pendant la pandémie de COVID-19 (RaDiOrg, 2 April 2020)

• Coronavirus: informatie voor TSC patiënten (be TSC, 15 March 2020)

Brazil

• Perguntas e respostas: Orientações sobre a epidemia de Coronavírus (COVID-19) para as pessoas com doenças raras e seus cuidadores (Casa Hunter, 19 March 2020)

• Guidelines - Orientações gerais de cuidados com as pessoas com doenças raras e seus cuidadores na pandemia de corona vírus (available in Portuguese, English and Spanish, Instituto Vidas Raras)

Canada

• CORD COVID-19 Patient Member Survey

Croatia

• Informacije i važni linkovi – koronavirusa COVID-19 (SARS—CoV-2) (Rare Diseases Croatia, 24 March 2020)

Czech Republic

• Aktuálně o koronaviru (Rare Diseases Czech Republic)

Denmark

• Information om coronavirus – opdateret (Rare Diseases Denmark, 16 March 2020)

France-Alliance Maladies Rares

• ENQUÊTE - maladies rares et COVID

• Alliance Maladies Rares: COVID-19 Déclaration de la présidente

• Santé Info Droits - COVID-19 : Des services d'information à votre disposition

• Maladies Rares Info Services

• Unapei - Informations et recommandations spécifiques pour les personnes handicapées

• COVID-19 recommandations pour les patients et leurs familles (AP-HP)

• COVID-19 - Faire face à une maladie chronique pendant le confinement (Haute Autorité de Santé, 10 April 2020)

• Résultats de l’enquête: Mucoviscidose et coronavirus (Association Muco, 10 April 2020)

• Information Coronavirus (Association nationale Spina Bifida et Handicaps associés (ASBH), 23 March 2020)

• Guidelines - Information sur le COVID-19 pour les adultes et les enfants (FAI2R, 17 March 2020)

• Mesures d'accompagnement pour les enfants/jeunes au domicile suite à la fermeture des IME (Association Française du Syndrome d'Angelman, 19 March 2020)

• AFM-Téléthon: Recommendations pour les malades neuromusculaires

Germany

• „Offener Brief an den Gesundheitsminister“ (Achse, 8 April 2020)

• „Stellungnahme Ressourcenmangel“ (Achse, 6 April 2020)

• Corona Ratgeber für sehbehinderte (DBSV, 31 March 2020)

• „Weiterführende Links und Hilfestellung für Betroffene“ (Achse, 23 March 2020)

• Stellungnahme zum Coronavirus (SARS-CoV-2, COVID-19) (Achse, 13 March 2020)

Hong Kong

• Study on the "Impact of the COVID-19 pandemic on patients with rare disease in Hong Kong" in collaboration with the University of Hong Kong and Rare Disease Hong Kong (12 September 2020)

Ireland

• Research Report - Living with a rare disease in Ireland during the COVID-19 pandemic (Rare Diseases Ireland, 21 May 2020)

• Press Release - Living with a rare disease in Ireland during the COVID-19 pandemic (Rare Diseases Ireland, 21 May 2020)

• Advice on 'cocooning' for extremely medically vulnerable people (Rare Diseases Ireland, 1 April 2020)

• Rare Diseases Ireland statement on coronavirus (COVID-19) (Rare Diseases Ireland, 23 March 2020)

• Resources for the rare disease community in Ireland - regularly updated (Rare Diseases Ireland 2020)

Italy

• COVID-19 e malattie rare (UNIAMO, regularly updated)

• Telemedicina: Un pilastro per il dopo-COVID (UNIAMO, 28 April 2020)

• Associazione - FONDAZIONE ITALIANA HHT “Onilde Carini” APS - per la TELEANGIECTASIA EMORRAGICA EREDITARIA o Sindrome di Rendu-Osler-Weber - Pandemia COVID-19 (HTT Italia, 1 April 2020)

• Lettera aperta al Governo (UNIAMO, 31 March 2020)

• Associazione Fondazione Italiana HHT: 15 Risposte alle piu comuni domande sull'infezione da coronavirus (COVID-19 o sars-cov-2): per le persone affette da HTT.. e non. (HHT, 23 March 2020)

• ELSI-COVID 19 Informed consent for biobanking (UNIAMO)

• UNIAMO COVID-19 webinars (UNIAMO)

Japan

• ASridは、"Advocacy Service for Rare and Intractable Diseases’ multi-stakeholders in Japan" の略であり、「希少・難治性疾患分野における全ステイクホルダーに向けたサービスの提供」を目的としています (ASrid)

• 希少・難治性疾患　海外動向（COVID-19）

Latvia-Aptauju

• Pacientu ar retām slimībām veselības, sociālais un emocionālais pašvērtējums- Rezultātu atskaite 2020

• Par COVID-19 (Latvian Alliance for Rare Diseases)

Luxembourg

• New open letter to the government regarding the health crisis (ALAN-Maladies Rares Luxembourg, 13 August 2020)

• News - Government's response to our open letter (ALAN-Maladies Rares Luxembourg, 14 April 2020)

• COVID-19 newsletter (ALAN-Maladies Rares Luxembourg)

• Lettre ouverte au gouvernement à propos de la crise sanitaire (ALAN-Maladies Rares Luxembourg, 30 March 2020)

• News - COVID-19 (ALAN-Maladies Rares Luxembourg)

Netherlands

• VSOP en de coronacrisis (VSOP, 20 March 2020)

Norway

• Sjeldne diagnoser og korona - Samling av informasjon om koronavirus og sjeldne diagnoser (Oslo universitetssykehus, 23 April 2020)

• Korona-informasjon (Norwegian Federation of Organisations of Disabled People, 19 March 2020)

• Kjernejournal (Summary Care Record) - online service providing healthcare professionals with access to certain important health information about you (helsenorge.no)

Portugal

• COVID 19 – Guia informativo para Pessoas com Doença Rara e seus Cuidadores (Raríssimas, Sociedade Portuguesa de Medicina Interna (SPMI) & Núcleo de estudos de doenças raras (NEDR), 25 April 2020)

Slovakia

• AKO SA SPRÁVAŤ V DOMÁCEJ IZOLÁCII (Slovak Alliance for Rare Disases)

Spain

• El 94% de familias con ER en España han visto interrumpida la atención de su patología con motivo de la pandemia (Federación española de enfermedades raras (FEDER), 27 May 2020)

• La situación de falta de atención sanitaria del colectivo de personas con enfermedades neuromusculares, ante la crisis COVID-19 (Federación ASEM, 12 May 2020)

• Guía para familias: Patología Neurológica Infantil durante el Confinamiento (Duchenne España, 12 May 2020)

• Informaciòn y recomendaciones frente al coronavirus Sars-COV-2 dirigidas a pacientes/familias ADEE (Acondroplasia y otras displasias esqueléticas con enanismo) (Fundación Alpe Acondroplasia (ALPE), 13 April 2020)

• COVID-19 y enfermedades raras: seguimos aquí para ti (FEDER, 24 March 2020)

• Recomendaciones para la prevención frente al Coronavirus Sars-Cov-2 dirigidas a pacientes con enfermedades neuromusculares (Federación ASEM, 24 March 2020)

• Recomendaciones COVID-19 (Coronavirus) - Recomendaciones especificas para pacientes con AME (Fundame, 10 March 2020)

• COVID-19 Piel de Mariposa - Folleto de información y recomendaciones para las personas con EB y sus familias (Debra International)

Sweden

• Sallsynta Diagnoser, Rare Diseases Sweden: Sällsynthet och COVID-19 Published in Altinget

UK

• The Rare Reality of COVID-19: COVID-19 Impact Report (Genetic Alliance UK, 2 July 2020)

• The Ehlers Danlos Society COVID-19 (Coronavirus) update (11 May 2020)

• Genetic Alliance UK information hub for COVID-19 (15 April 2020)

• New general advice regarding Coronavirus (COVID-19) for patients with rare genetic disorders (Unique, 14 April 2020)

• Guidelines - Unique's guide on self-isolation for families with children with rare chromosome or gene disorders (Unique - Understanding Chromosome Disorders, 30 March 2020)

• Brittle Bone Society COVID-19 update for people with rare bone disorders (25 March 2020)

• Coronavirus and TSC (Tuberous Sclerosis Complex): Information for the TSC community (TSA: Tuberous Sclerosis Association, 24 March 2020)

• Duchenne UK update on Coronavirus (COVID-19) (Duchenne UK, 20 March)

• MAJOR NEW MEASURES TO PROTECT PEOPLE AT HIGHEST RISK FROM CORONAVIRUS (Genetic Alliance UK, 20 March 2020)

USA

• NORD COVID-19 Resource Center

• NORD Community survey results reveal the significant impact of COVID-19 on Americans living with rare diseases

• NIH-supported research survey to examine impact of COVID-19 on rare diseases community

• COVID-19 Rapid Response Leadership Series (National Organization for Rare Disorders (NORD))

• COVID-19 Community Survey Report (National Organization for Rare Disorders (NORD), 5 May 2020)

• COVID-19 resources for the rare community (National Organization for Rare Disorders, 19 March 2020)

• Concerns about the Coronavirus / LAM (Lymphangioleiomyomatosis) (LAM Foundation USA, 18 March 2020)

• National Fragile X Foundation: COVID-19 - My child is home from school or work or a day program! What do I do? (13 March 2020)

• NORD’s Peter L. Saltonstall on Coronavirus Prevention and Risk for the Rare Community (National Organization for Rare Disorders, 5 March 2020)

• ELF (European Lung Foundation)* - Q&A: your questions answered by a respiratory expert (1 April 2020)

• ESC (European Society of Cardiology) - Q&A for COVID-19 and Heart Patients (31 March 2020)

• Osteogenesis Imperfecta Federation Europe (OIFE)* - COVID-19 info for people with OI (25 March 2020)

• HHT Europe* - Indicazioni su Covid-19 e HHT (23 March 2020)

• Cystic Fibrosis Europe* (23 March 2020)

• Lupus Europe Update on Hydroxychloroquine* (21 March 2020)

• Guidelines - Myeloma Patients Europe - Information on Coronavirus and Myeloma* (20 March 2020)

• European Alliance Of Neuromuscular Disorders Associations* (19 March 2020)

• European League Against Rheumatism (EULAR) Policy statement on COVID-19 (19 March 2020)

• The European Connected Health Alliance (ECHAlliance) Position Paper on the impacts of COVID-19 on European health R&I activities (18 March 2020)

• Guidelines - Paediatric Rheumatology European Society (PReS) Paediatric Rheumatic Diseases - Recommendations for the COVID-19 outbreak, translated in many languages (17 March 2020)

• Guidelines - European League Against Rheumatism (EULAR) Guidance for patients COVID-19 outbreak (17 March 2020)

• Statement on COVID-19 from the European Cancer Organisation’s Board of Directors (17 March 2020)

• European Lung Foundation*: your questions answered by a respiratory expert - available in 9 languages (16 March 2020)

• European Patients’ Forum statement on COVID-19 (12 March 2020)

• European Haemophilia Consortium Statement* (11 March 2020)

• Survey - Global Rheumatology Alliance - Surveys on how COVID-19 may affect people with rheumatic, autoimmune and autoinflammatory diseases

• SMA Europe* statement on COVID-19

• European Paediatric Neurology Society - COVID-19 and neurological disorders

• European Patients Forum - COVID-19 Resource Point

• European Lung Foundation* - COVID-19 resources

• EUnetHTA (European Network for Health Technology Assessment) COVID-19-related repository of publications and outputs

• ERN BOND have a dedicated 24hr helpline during the coronavirus outbreak (24 March 2020)

• ERN ITHACA - General advice regarding Coronavirus (COVID-19) for patients with rare genetic disorders (23 March 2020)

• Guidelines - COVID-19 recommendations from VASCERN's Rare Disease Working Groups (23 March 2020)

• eUROGEN newsletter - European Commission launches COVID-19 Web Conferencing support system (23 March 2020)

• ERN-EYE: Update about COVID-19 and Rare Eye Diseases (20 March 2020)

• VASCERN HHT statement on COVID-19 (20 March 2020)

• ERN Rare-Liver : Information for all patients with rare liver diseases (10 March 2020)

• Survey - from European Reference Network for Neuromuscular diseases to increase understanding of the effects COVID-19 has on patients with existing neuromuscular conditions

• COVID-19 information from EuroBloodNet (ERN for rare hematological diseases)

• ERN BOND: COVID-19 indications and recommendations

• Survey - ERN-LUNG called all its members to share information on their experience with COVID-19 in patients with a rare lung disease

• ERN-RND: list of official websites containing up-to-date information on the COVID-19 pandemic

• MetabERN: Recommendations for all rare inherited metabolic diseases patients and caregivers about treatment adherence during the COVID-19 emergency

• Daily updates on COVID-19 and Duchenne & Becker muscular dystrophy (World Duchenne Organization*)

• RDI Statement on COVID-19 response and recovery (6 July 2020)

• Chronic Myeloid Leukemia (CML) and COVID-19 (updated frequently) (CML Advocates Network*) (18 April 2020)

• The COVID-19 pandemic and haemoglobin disorders - Blood and COVID-19: An informational guide from TIF (Thalassaemia International Federation*) (10 April 2020)

• IBTA's COVID-19 international information page - COVID-19 information for the international brain tumour community (International Brain Tumour Alliance*) (31 March 2020)

• The COVID-19 pandemic and haemoglobin disorders - a TIF-proposed Haemoglobinopathy Patient Pathway (Thalassaemia International Federation*) (30 March 2020)

• Frequently Asked Questions about COVID-19 and CDG (Congenital Disorders of Glycosilation), available in multiple languages (World CDG Organization) (29 March 2020)

• COVID-19 Information for the HI Community (Congenital Hyperinsulin International (CHI)) (23 March 2020)

• Evidence-based and expert validated information for the global primary immunodeficiency (IPOPI*) (21 March 2020)

• Statement from Retina International and its Scientific and Medical Advisory Board on COVID-19 (Retina International*) (21 March 2020)

• Statement from the International Gaucher Alliance on COVID-19 (International Gaucher Alliance*) (20 March 2020)

• Statement on COVID-19 from the World CDG Organization, available in many languages (19 March 2020)

• Guidelines - Key Recommendations toward a disability-inclusive COVID-19 response (International Disability Alliance) (19 March 2020)

• How is the coronavirus affecting people with Spina Bifida and Hydrocephalus? (International Federation For Spina Bifida And Hydrocephalus*) (17 March 2020)

• Coronavirus and Mitochondrial Disease (International Mito Patients*) (13 March 2020)

• COVID-19 update from the World Alliance Of Pituitary Organizations (WAPO*) (11 March 2020)

• Inherited Metabolic Disease and Coronavirus (COVID-19) advice for patients / parents / guardians (International Niemann-Pick Disease Alliance*) (5 March 2020)

• COVID-19 updates from the International Patient Organization For Primary Immunodeficiencies (IPOPI*)

• COVID-19 advice for patient groups from the Lymphoma Coalition*

• Message from the president of Thyroid Federation International*

• Statement on COVID-19 from the World Federation of Incontinence Patients*

• The European League Against Rheumatism (EULAR) database to monitor and report outcomes of COVID-19 occurring in patients with rheumatic and musculoskeletal diseases-

• International Alliance of Patients' Organizations (IAPO) Coronavirus (COVID-19) Resources Hub-

• International Federation for Spina Bifida and Hydrocephalus (IFSBH) COVID-19 resources

• Webinar - Webinar on Second Wave of COVID-19: Preparatory actions - Consultation with Clinicians (11 June 2020)

• Webinar - Webinar on the impact of COVID 19 for people living with a Rare Disease (9 June 2020)

• Webinar - Webinar on “COVID-19 and the Kidney" in collaboration with ERKNet (8 June 2020)

• Webinar - COVID-19 and Rare Liver Diseases (4 June 2020)

• Webinar - COVID-19 and Intensive Care Medicine (2 June 2020)

• Webinar - Webinar on Providing high-quality care remotely to patients with Rare Bone Diseases during COVID-19 pandemic (19 May 2020)

• European Medicines Agency Press Briefing 14 May (14 May 2020)

• Webinar - 6th webinar by the “COVID-19 Clinical Management Support System” on “COVID-19: Endocrine conditions with increased risk - Endo-ERN” (12 May 2020)

• Webinar - Webinar on COVID-19 in patients with cardiomyopathies and myocarditis  (7 May 2020)

• Webinar - 4th webinar of the COVID-19 Clinical Management Support System on “COVID-19 in patients with rare diseases of the respiratory system - ERN-LUNG and the European Respiratory Society (ERS) joint webinar” (30 April 2020)

• Webinar - Third webinar of the COVID-19 Clinical Management Support System on “COVID-19 and anti-epileptic drugs” (27 April 2020)

• Webinar - Webinar on COVID-19 and inherited arrhythmia syndromes (webinar presentations) (23 April 2020)

• Webinar - EFPIA Virtual Event "Cancer and COVID-19 – short term reality, long-term vision" (22 April 2020)

• Webinar - Webinar on Sarcomas and COVID-19 patients (20 April 2020)

• Webinar - Evaluating the potential of drug repurposing for rare diseases and COVID-19 (webinar recording, Findacure) (8 April 2020)

• Webinar - EHA & ERN-RND webinar: “Huntington’s disease and the Covid-19 pandemic - a difficult combination” (webinar recording) (6 April 2020)

• Webinar - EASPD: Ensuring Staff Continuity in Social Services during the COVID-19 pandemic (25 March 2020)

• Webinar - Inherited Metabolic Disease & Coronavirus (COVID-19) British Inherited Metabolic Disease Group (BIMDG) and Metabolic Support UK (23 March 2020)

• Webinar - Marfan Europe Network (19 March 2020)