Coronavirus and rare diseases: update
In the current Coronavirus pandemic, all stakeholders in the field of rare diseases are mobilised to ensure that rare disease patients receive adequate treatment and support. In the previous issue of OrphaNews, we provided a list of resources, including expert guidelines, that we continue to update, in order to ensure that those concerned with a rare disease have access to the appropriate information.
European Commission
The European Commission (EC) has initiated several actions to respond to this crisis. This response to the crisis is articulated through the following actions:
• Coordination with all Member States, of the response epidemic at the EU level through three key mechanisms, including the Early Warning and Response System, as according to the Cross-Border Health Threat Decision;
• Technical guidance regarding risk assessment and case reporting in collaboration with European agencies;
• Coordination of assistance to China, as well as financing of transport costs of repatriated EU citizens
• Global preparedness, prevention and containment of the virus.
In addition to this response, on 17 March, the EC launched of a Commission Advisory Panel on Covid-19, composed by epidemiologists and virologists, whose activities will center on crisis response for each stage of disease and national contexts, in order to avoid gaps, inequalities and inconsistencies in measures taken so as to contain the virus. The panel will also make recommendations on community measures, such as social distancing, that have proven to be efficient in ‘flattening the curve’, slowing the transmission of the disease.
The EC has also issued recommendations regarding testing strategies with a focus on timely and accurate and robust laboratory testing strategies for slowing down the epidemic and to improve virologic knowledge of Covid-19. On 30 January 2020, the EC launched a request of expressions of interest for 17 new research projects on the Covid-19 for a total amount of €10 million. 136 research teams from the EU will be involved in the 17 projects funded by the standing project line for emergency research funds in connection with the Horizon 2020 annual work programmes. The EC, the European Medicines Agency (EMA), and the national Heads of Medicines Agencies (HMA) have published recommendations addressed to sponsors to advise on the way to deal with clinical trials during the coronavirus epidemic.
The EC has also drawn on the experience of the European Reference Networks (ERNs) to formulate its response to the crisis. A Covid-19 Communication Support System (Covid-19 CMSS) has been created to foster exchange knowledge and on available information based on the experience to date in ERNs to benefit to the healthcare professionals who are dealing with the Covid-19. A Covid-19 Communication Support System (Covid-19 CMSS) Web system was set up to ease communication among clinicians and across the EU. A helpdesk managed by DG SANTE has been set up for clinicians to facilitate web conferences and exchanges with colleagues across Europe on treatment options, and on how to handle severe and complex cases. It is hoped that this initiative will improve treatment of patients.
In a statement, the ERN Board of Member States called on European health systems actors to strengthen their cooperation regarding operating principles for good quality care and solidarity.
European Reference Networks
The ERNs have also launched several initiatives to inform professionals and rare disease patients during the coronavirus epidemic. A full list of these resources, including statements, guidance and recommendations issued by the ERNs, is provided in the previous edition of OrphaNews and is updated regularly. To date around 40 specific resources have been made available to the community by the ERNs
Patient organisations
Other networks also released statements following the Covid-19 outbreak. The International Rare Disease Research Consortium (IRDiRC) sent a message to the IRDiRC community to express their support. The organisation launched a survey asking people to share their experience on how the epidemic is affecting their work or activities, and which measures were taken by their organisation of affiliation. EURORDIS also launched a number of statements (see Spotlight) aimed at protecting rare disease patients during the current crisis and suggesting policy measures to achieve this. Another organisation, the National Organisation for Rare Diseases (NORD) advocated for the access to medical treatment, with an emphasis on adequate and affordable coverage for people with rare disease condition during the coronavirus pandemic. EURORDIS and NORD issued a joint statement calling for rapid development of covid-19 treatments without the use of orphan drug incentives. Rare Voices Australia (RVA), in collaboration with the Scientific and Medical Advisory Committee, also released a statement on covid-19 following the EURORDIS press release. RVA called for the urgent development and use of critical care guidelines for people with rare diseases to guide hospitals and clinicians with adequate information on the coronavirus.
Learned societies and other organisations
The British Society for Genetic Medecine (BSGM) gave general advice for patients with rare and undiagnosed genetic disorders on how to manage their disease and the risks associated to the covid-19. The European Data Protection Board (EDPB) issued a statement on the processing of personal data, for example the General Data Protection Regulation (GDPR), during the current period of the covid-19 outbreak. And finally, the United Kingdom Genetic Alliance communicate about major measures to be observed by people at high risk from coronavirus, including people with genetic, rare and undiagnosed conditions.
Research
In the line of other European actions, on 3 March, the Innovative Medicines Initiative (IMI) launched a fast track call for proposals on Covid-19 for €45 million to fight current and future coronavirus outbreaks, focused on diagnostics and development of therapeutics. The call for proposals includes drugs in an advanced or earlier stage of development, approved drugs for repurposing, and the development of diagnostic tests. Preventive vaccines are not concerned by the call. Another IMI’s project, IMI-funded ZAPI (Zoonotic Anticipation and Preparedness Initiative) project, focused on the potential of nanobodies, believed to be genetically-engineered small antibodies founded mostly in Ilamas, dromedaries, and camels, and that are able to fight against zoonotic diseases transmitted to humans from animals, like vertebrates, such as coronaviruses. IMI launched ZAPI project to establish tools and platforms for a rapid response in the production of vaccines in case of such a kind of the covid-19 outbreak. ELIXIR, to support of the fight against the Covid-19, created a website with a full range of services provided by the organisation for more information on the coronavirus, including resources for studying SARS-CoV-2 coronavirus and the COVID-19 disease. The ELIXIR website is regularly being updated and readers are invited to contact the network through an electronic message in case they have questions.