World Orphan Drug Congress 2021
From 16 to 19 November 2021
At : Barcelona, Spain
Events
Genomics in Health Implementation Forum (GHIF) meeting
From 17 to 18 November 2021
At : Online
Webinar: Pseudopubertas praecox and the tumor rupture
On 18 November 2021
At : Online
Webinar: Vasculitis- From cell to service
From 18 to 19 November 2021
At : Online
Webinar: Reinvigorating Family Health History in the Era of Genomics
On 19 November 2021
At : Online
Webinar: Muscle and Statins
On 19 November 2021
At : Online
International Conference On Rare Diseases and Paediatric Research
From 19 to 20 November 2021
At : Online
10th International Conference of the International Lymphoedema Framework
From 19 to 21 November 2021
At : Copenhagen, Denmark
Webinar: Pure autonomic failure: really that pure?
On 24 November 2021
At : Online
Resource Webinar: Orphanet
On 26 November 2021
At : Online
Webinar: Challenges in the management of non-metastatic thyroid microcarcinoma
On 26 November 2021
At : Online
Webinar: ERN Italia- points to consider for the ERN RDs Italian community
On 01 December 2021
At : Online
Third ITHACA webinar on Solve-RD
On 01 December 2021
At : Online
Patient-Focused Drug Development — How RDCA-DAP Can Help
On 01 December 2021
At : Online
Marche des Maladies Rares
On 05 December 2021
At : Paris, France
Webinar: Capturing the patients’ voices on patient safety
On 15 December 2021
At : Online
Webinar: Congenital Brain Tumor: Diagnostic and Therapeutic Challenges
On 16 December 2021
At : Online
ERN GENTURIS 5-year anniversary conference
From 11 to 12 February 2022
At : Online
International Symposium for Researchers and Clinicians on Wiskott Aldrich Syndrome- WAS2022
On 19 March 2022
At : Munich, Germany
The Translational Science of Rare Diseases - From Rare to Care IV
From 31 March to 02 April 2022
At : Tutzing, Germany
20th International Vasculitis and ANCA Workshop
From 04 to 07 April 2022
At : Dublin, Ireland
International Primary Immunodeficiencies Congress – IPIC 5th Edition
From 28 to 30 April 2022
At : Vilamoura, Portugal
ECTS 2022: 49th edition of the European Calcified Tissues Society Congress
From 08 to 11 May 2022
At : Helsinki, Finland
World Congress on Rare Skin Diseases
From 08 to 10 June 2022
At : Paris, France
13th International Congress on Autoimmunity
From 11 to 14 June 2022
At : Athens, Greece
7th Annual TREAT-NMD International Conference
From 17 to 18 June 2022
At : Vancouver, Canada
European Conference on Rare Diseases & Orphan Products- ECRD 2022
From 28 June to 02 July 2022
At : Online
Grants
European Commission Horizon Europe Framework Programme (HORIZON)
The European Commission is funding a call on the development of new effective therapies for rare diseases with the aim of “Tackling diseases diseases and reducing disease burden.” The topic will support proposals covering several different stages in the continuum of the innovation pathway ( translational, preclinical, clinical research, validation in the clinical and/or real-world setting etc.), as relevant.
To that end, proposals under this topic should aim for delivering results that are directed, tailored towards and contributing to some of the following expected outcomes:
- Researchers and developers make the best use of the state-of-the-art knowledge and resources for a fast and effective development of new therapies for rare diseases.
- Researchers and developers increase the development success rate of therapies for rare diseases by employing robust preclinical models, methods, technologies, validated biomarkers, reliable patient reported outcomes and/or innovative clinical trials designs.
- Developers and regulators move faster towards market approval of new therapies for rare diseases (with currently no approved treatment option) due to an increased number of interventions successfully tested in late stages of clinical development.
- Healthcare professionals and people living with a rare disease get access to new therapeutic interventions and/or orphan medicinal products.
The deadline for this call is a two-stage model, including 1 February 2022 and 06 September 2022.
The EJPRD is offering services to help applicants for this call, notably in the fields of Data FAIRification, Clinical Studies Support Office and Mentoring.
EJP RD Research Mobility Fellowships 4th call
The call for Research Mobility Fellowships aims to support PhD students, Postdocs and medical doctors in training to undertake scientific visits fostering specialist research training outside their countries of residence. Successful applicants should acquire new competences and knowledge related to their research on rare diseases, with a defined research plan and demonstrable benefit to the ERN of the home and/or host institution.
Either home or host institution must be a Full Member or Affiliated Partner of an ERN at the time when the application is submitted, as well as during the proposed period of the training stay. Successful applicants should acquire new competences and knowledge related to their research on rare diseases, with a defined research plan and demonstrable benefit to the ERN of the home and/or host institution.
- Fellows should take into consideration several elements such as:
• Relevance and impact of the project, for the fellow, home and host institutions and the ERN as a whole
• Clear added value for the ERN(s)
• Quality of the research proposal
• Organisation and proposed methodology of the training
• Relevance of timelines and of required resources and budget
The research mobility fellowships are meant to cover stays of 4 weeks to 6 months duration.
Applications are due by 28 November 2021.
The 2nd Share4Rare Call for Research Projects
The objective of this call is to select two projects from research groups and/or patient organisations to be implemented and developed from January to December 2022. This call is thus open for patient-driven research projects. Applications are due before 10 December 2021.Patients’ organisations or academic institutions, and other related stakeholders interested to promote patient-centred research in rare diseases are welcome to apply.
Selected proposals will be co-financed by Sant Joan de Déu Research Foundation, which will assume 50% of the costs related to the implementation of the research project.
Second wave of the EU4Health calls for project grants
The second wave of the EU4Health calls for project grants under the 2021 Work Programme has been published. The 13 topics range from health data, healthy lifestyles, infections, mental health, cancer, genomics and more.
Applications are acceptable either by a sole applicant or by a consortium. In case of a consortium, the consortium must be a consortium of at least 3 applicants, and it must comply with the condition of minimum 3 entities from 3 different eligible countries.
Eligible countries are EU Member States or eligible non-EU countries. Eligible non-EU countries are
EEA countries, and countries associated to the EU4Health Programme.
Applications are welcome until 25 January 2022.
Repurposing of authorised medicines: pilot to support not-for-profit organisations and academia
The EMA and the Heads of Medicines Agencies (HMA) are launching a pilot project to support the repurposing of medicines. The aim of this project is to support not-for-profit organisations and academia to gather or generate sufficient evidence on the use of an established medicine in a new indication to have this new use formally authorised by a regulatory authority. As part of the pilot, EMA and the national medicines agencies will provide regulatory support, primarily scientific advice, to help these stakeholders generate a data package robust enough to support a future application by a pharmaceutical company. Sponsors wishing to seek EMA scientific advice should complete the File drug repurposing submission form by 28 February 2022.
Student Voice Prize
The Student Voice Prize is an annual, international essay competition that focuses on raising the profile of rare disease within the medical field, particularly with medical students, nurses and scientists who may never have considered working in rare disease.
The following categories are encourage to apply:
- Medical students
- Trainee nurses
- Undergraduate and masters students enrolled in biological and health sciences studies (pharmacy, biology, genetics, biomedical science, pharmacology, public health etc)
Applicants are invited to answer one essay question in 1,500 words or less.
The deadline to submit the essay is on 17th November 2021 by 12:00 pm GMT.
EJP RD – Networking Support Scheme (NSS) Call
The EJP RD Networking Support Scheme (NSS) call is open.
The first aim is to encourage sharing of knowledge on rare diseases and rare cancers of health care professionals, researchers and patients. The second aim is to enable or increase the participation of usually underrepresented countries in Europe (see below) in new and existing research networks. The scheme will provide financial support to applicants to organize workshops or conferences. The focus of these workshops or conferences should be (the implications of) research results and innovative solutions in compliance with the vision set by the International Rare Diseases Research Consortium (IRDiRC) : "“enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention”.
In this Networking Support Scheme applicants can apply for a budget of a maximum of 30,000 € per event.
The collection date is December 2, 2021 at 14.00 (CET).
The most important change from June 3, 2021 is that applicants can choose between the format of a face-to-face meeting, an online meeting or a hybrid meeting if networking is secured. The Call documents have been changed acoordingly.
Please note: Are you planning to apply for the next rounds of the Networking Support Scheme? Please note that the documents have been updated in June 2021. Take care not to use earlier documents as they will not be eligible anymore.
Courses and educational initiatives
Radboudumc Adrenal Masterclass
Radboud UMC is organizing its fourth international Radboudumc Adrenal Masterclass to presents the state-of-the-art lectures and workshops on clinical and pathophysiological aspects of adrenal diseases. The masterclass is aimed at adult and paediatric endocrinologists, clinical fellows, internists and clinical chemists who are involved in the management of patients with adrenal disease.
The masterclass will take place on January 24-28, 2022 in Amsterdam, the Netherlands. It will cover major adrenal disorders and will include lectures as well as interactive sessions on patient cases with challenging diagnostic and therapeutic issues.
Introduction to real world data
The European Health Data & Evidence Network (EHDEN) and the European Patients’ Forum (EPF) offer a free course to patient organisations on real world data. The course focuses on data protection and the vital role they can play in the healthcare ecosystem and health research.
The course is available upon registration through the EHDEN Academy.
Paediatric Surgery Courses
A new series of Paediatric Surgery Courses organized by the Radboud UMC – Amalia Children’s Hospital (Nijmegen, The Netherlands) will be held online in 2022.
The courses will take place on the following dates:
- Pediatric Colorectal Course 13-14 January 2022
- ECMO & CDH course 10-11 March 2022
ERN EpiCare e-learning modules
The ERN EpiCARE has developped new e-learning modules in collaboration with the ILAE Academy. The modules are dedicated towards professionals who wish to practice diagnosis of rare and complex epilepsies. The first online six modules are patient-centered cases.
EJP-RD and the Foundation for Rare Diseases : "Diagnosing Rare Diseases - from the Clinic to Research"
The European Joint Programme on Rare Diseases has launched an online academic education course on rare diseases research topics. The development of the courses is coordinated by the Foundation for Rare Diseases, one of EJP RD's partners.
The first MOOC (Massive Open Online Course) “Diagnosing Rare Diseases: from the Clinic to Research and back” started on April 26th. This course aims to gain insight into patients’ experiences and discuss key issues relating to this topic.
It has been co-developed with representatives from ERN Ithaca (Prof Laurence Faivre, Dijon University Hospital), ERN Genturis (Dr Chrystelle Colas, Curie Institute) and Foundation for Rare Diseases (Roseline Favresse).
This course is designed for individuals with a keen interest in diagnostic research and rare diseases. While primarily designed for medical students and PhD/post-doc students in biomedical sciences, it will also be of interest to Patients Advocacy Organisations’ representatives, Healthcare professionals or paramedics who want to further their knowledge of rare diseases diagnosis.
This course will cover the following topics :
- The diagnostic process and the types of genetic tests available for rare diseases
- The differences in rare genetic diseases patient pathways
- Technological advances for diagnostic research
- The role of collaborative studies and data sharing in rare diseases diagnosis
- The impact of having or lacking a diagnosis on patients’ lives
- The role and place of physiopathology approaches as well as social sciences research in the context of rare diseases diagnosis.
The 8th Rare Diseases Summer School
Because of the pandemic covid-19, the 8th Rare Diseases Summer School has been postponed and will be held from 13 July to 15 July 2022 in Kartause Ittingen. The rare diseases summer school is part of the curriculum of the PhD programs. It aims to provide an environment for informal exchange between PhD students and experts in the field of rare disease research. The application procedure will open in 2022.
International Neurofibromatose Educational Resources: Masterclasses in NF
INFER (International NF Educational Resources) is a series of online educational lectures for medical professionals by leading neurofibromatosis experts.
The next lecture will be held on:
- 10th December 2021, 4pm CET:
- NF1 Associated Glioma in Children - the Optic Pathway and Beyond
- Chaired by Amedeo Azizi, Head of the Paediatric Neurofibromatosis Program and Paediatric Neuro-Oncology Program (Vienna, Austria)
ERN-Skin: Series of webinars
The ERN-Skin launches a series of scientific webinars on rare skin diseases. Each ERN-Skin thematic group organizes a one hour session, with 30 minutes presentation and 30 minutes discussion.
The next webinars will be held on:
- 30th November 2021, 1pm - 2pm CET :
- Acquired immunological low prevalence and complex adult diseases of the skin
- 14th December 2021, 1pm - 2pm CET :
- Mendelian connective tissue disorders
- 25th January 2022, 1pm - 2pm CET :
- Cutaneous diseases related to DNA repair disorders
Job opportunities
Orphanet Nomenclature & rare diseases and X-eHealth Project Manager
Please send your CV and cover letter to jobs.orphanet@inserm.
Orphanet Rare disease clinical trials and orphan medicinal products data curator
Please send your CV and cover letter to Charlotte Gueydan at jobs.orphanet@inserm.fr
Orphanet database curator
Please send your CV and cover letter with the reference US14-2021-01 to:
Charlotte Gueydan
Tel : +33 (0)1 56 53 81 41
jobs.orphanet@inserm.fr
Media
The Rare Disease Podcast 4 Medics
Medics4RareDiseases have launched a new podcast entitled "The Rare Disease Podcast 4 Medics". Interviews with people from across the rare disease field will be added each week to share their experiences and perseptives as well to provide advice for healthcare professionals. The interviewees include patients, clinicians, advocates, students as well as researchers focusing on rare diseases.
The podcast is available on Apple Podcast and Spotify.