GA4GH 6th Plenary Meeting
From 04 to 06 October 2018
At : Basel, Switzerland
Events
The Glasgow Rare Disease Showcase
10 October 2018
Venue : Glasgow, Scotland
11th International Society of Neonatal Screening European Regional Meeting
From 15 to 18 October 2018
At : Bratislava, Slovaquie
NORD's Rare Diseases and Orphan Products Breakthrough Summit
From 16 to 17 October 2018
At : Washington D.C., USA
Orphan Drugs and Rare Diseases
From 18 to 19 October 2018
At : Holiday Inn Kensington Forum, 97 Cromwell Road, London, UK
At this year's Orphan Drugs and Rare Diseases conference, taking place on 17th – 18th October in London, industry experts will address the opportunities and challenges within the rare disease treatment field.
HIGHLIGHTS IN 2018:
- Discuss the pricing and reimbursement of orphan drugs
- Understand the challenges of patient recruitment and patient-centric research
- Hear the MHRA’s perspective on benefit-risk assessment in rare diseases
- Explore the clinical development of orphan drugs for rare disease
- Gain insight into potential synergies between regulators and assessors
FEATURED SPEAKERS:
- Xavier Ortega, Project Manager, Rare Diseases, Minoryx
- Nigel Nicholls, Director and Country Manager UK/Ireland, BioMarin Europe Ltd
- Yolanda Barbachano, Senior Statistical Assessor, MHRA
- Michale Bouskila-Chubb, Head of Business Development, Healx
- Nicholas Sireau, CEO and Board Chair, AKU Society
- Peter Rutherford, Medical Director and Patient Access Leader, Vifor Pharma
- Solange Corriol-Rohou, Senior Director Regulatory Affairs and Policy, EU, GRAPSQA - GMD, AstraZeneca
- Neil Dugdale, Vice-President and General Manager, UK and RoI, Sobi
There will also be an interactive workshop on 16 October, run by JG Zebra Consulting who will be looking into the topic of: "Working together for HTA in rare diseases - A step too far or the way forward?"
ICT4Life Final Conference
18 October 2018
Venue : Brussels, Belgium
American Society of Human Genetics
From 17 to 21 October 2018
At : San Diego, USA
Family Conference CGD society
From 20 to 22 October 2018
At : Cobham, Surrey, UK
Annual Meeting of the Histiocyte Society
From 23 to 24 October 2018
At : Lisbon, Portugal
Fifth International Symposium on ARSACS Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS)
From 01 to 02 November 2018
At : Montreal, Quebec
9th World Orphan Drug Congress
From 07 to 09 November 2018
At : Barcelone, Espagne
Rare Voices Australia: 2018 Rare Disease Summit
From 17 to 18 November 2018
At : Melbourne, Australia
1st global congress on epidermolysis bullosa
From 20 to 24 January 2020
At : London, UK
Courses & Educational Initiatives
International course "Clinical practice guidelines for rare diseases: development and quality assessment"
From 10 to 11 October 2018
At : Rome, Italy
Register before: 25 septembre
Second ERN-EYE workshop
From 11 to 13 October 2018
At : Florence, Italy
This ERN-EYE workshop will be mainly dedicated to genetic testing and will allow the publication of a white paper on genetic testing for rare eye diseases in the European Union, coordinated by the chairs of the TWG6 genetic testing group, Prof. Graeme Black and Prof. Frans Cremers.
Second ROAD Network Winter School
From 07 to 08 December 2018
At : Paris, France
This event is dedicated to « New Progress in Osteoarticular Research : From Cell Environment to Human Ecosystem ».
Abstract submission is now open, until 14 September 2018. Registration is free but required on account of limited places.
EURORDIS Winter School 2019
From 12 to 16 March 2019
At : Imagine Institute, Paris, France
Applications are now open. Applicants will be notified of the outcome of their application by mid October.
Grants
Rare Diseases Clinical Research Network Funding Opportunity Announcement (FOA)
The National Center for Advancing Translational Sciences (NCATS) Office of Rare Diseases Research (ORDR) has published two Funding Opportunity Announcements for the Rare Diseases Clinical Research Network (RDCRN) :
- Data Management and Coordinating Center (DMCC): The DMCC will facilitate and support the activities of each Rare Diseases Clinical Research Consortium (RDCRC) along with trans-network activities that broadly facilitate the advancement of rare disease research via four Cores: 1) Administrative; 2) Data Management; 3) Clinical Research and; 4) Engagement and Dissemination.
- Rare Diseases Clinical Research Consortia (RDCRC): The RDCRCs are intended to advance the diagnosis, management, and treatment of rare diseases with a focus on clinical trial readiness. Each RDCRC will promote highly collaborative, multi-site, patient-centric, translational and clinical research with the intent of addressing unmet clinical trial readiness needs.
Letters of intent are due for 9 September 2018, application are due for 9 October 2018. Applicants are encouraged to apply early to allow adequate time to make any corrections to errors found in the application during the submission process by the due date.
The French Haemophilia Association (AFH) 2018 call for proposal
This call for proposals aims to support an innovative project on new strategies for basic, preclinical or clinical research for patients with Von Willebrand Disease.
Potential applications of this project should be of benefit to patients. The proposal should foster progress on the diagnosis of the disease, on its understanding, to provide answers in the improvement of existing treatments or even to go towards new innovative therapies. All requests concerning all or part of these criteria will be considered.
Funding of €50,000, awarded for one year, will be awarded in the form of a one-year fixed-term contract (established by agreement with the host organizations) to a postdoctoral researcher (clinician or scientist).
Depending on the proposal(s) selected and the results obtained, the financial support of the AFH may be renewed annually, one or more times, or readjusted by the Board of Directors (CA) of the AFH. The amount of this future support is currently not defined and will depend on the donations collected by the association at their maturity.
The deadline for submitting applications is 10 Octobre 2018.
Completed applications must imperatively be sent by mail to Geneviève Piétu (genevieve.pietu@afh.asso.fr).
Innovative Medicines Initiative (IMI) : new calls for proposals
The Innovative Medicines Initiative (IMI) has launched several calls for proposals. The topics address, among others, brain disorders (e.g. Alzheimer's disease, Parkinson's disease, Huntington's disease, major depression) and immune-mediated diseases (e.g. rheumatoid arthritis and lupus as well as inflammatory bowel diseases such as Crohn's and colitis, and skin diseases like dermatitis and psoriasis). The aim of the topics is to make clinical trials more patient-centric, contribute to medicine safety, and apply blockchain technologies to the drug development and health sectors.
- Call 15
- Topic 1: Integrated research platforms enabling patient-centric drug development
- Topic 2: Blockchain Enabled Healthcare
- Topic 3: Microenvironment imposed signatures in tissue and liquid biopsies in immune-mediated diseases
- Topic 4: Emerging translational safety technologies and tools for interrogating human immuno-biology
- Topic 5: Development and validation of translational platforms in support of synaptopathy drug discovery
- Topic 6: Digital endpoints in neurodegenerative and immune-mediated diseases
Stage 1 submission deadline: 24 October 2018
Job Research Foundation will award several grants
The Job Research Foundation is offering grant funding for researchers who are interested in researching the causes of, and treatments for, Job Syndrome.
For the remainder of the 2018 calendar year, the Foundation will award two 2-year grants of up to $200,000 each, and two 2-year grants of up to $50,000 each to qualified grantees. Special Consideration will be given to research focused on Job Syndrome and Pulmonary complications due to Job Syndrome, and therapies to help them. It is anticipated that these grants will be available on an annual basis for new applicants. Previous applicants can apply in subsequent years to extend their research.
The submission deadline for all grant applications is 15 Novembre 2018. Grants will be awarded by 17 Decembre 2018.
International Prize for Scientific Research Arrigo Recordati 2019
The International Prize for Scientific Research Arrigo Recordati has announced the Call for Nominations for the Ninth Edition of the Award.
The 2019 €100,000 Award will be dedicated to the promotion and recognition of excellence in orphan disease treatment research.
International projects within the area of rare/orphan disease treatment in all therapeutic areas (except for oncology, hematology and immunology) are eligible to apply as long as the disease concerned has a prevalence of not more than 1 in 2000.
The 2019 Award is open to researchers of all nationalities who are not in any way directly affiliated with pharmaceutical or medical device companies.
Nominations procedure will follow a two-step selection approach:
Deadline of the preselection: 15 December 2018
Full Applications: 30 April 2019
Contacts: recordatiprize2019@recordati.com
International fund Congenital Adrenal Hyperplasia: new call for proposals
The international fund Congenital Adrenal Hyperplasia (IFCAH) is opening a call for proposals in October 2018.
IFCAH is an endowment fund created to support research dedicated to improve the lives of those living with Congenital Adrenal Hyperplasia. Over the past 8 years 112 different European proposals have been submitted.
Letters of intent are due by January 2019.
Job opportunities
VASCERN hires: IT helpdesk/end-user support and data manager
The VASCERN coordination team, located in Paris, is looking for a full-time IT helpdesk/end-user support specialist and a full-time data manager.
Read the full job descriptions (in English and French):
Job offer: VASCERN – IT HELP DESK END-USER SUPPORT SPECIALIST (EN-FR)- Sept 2018
Job offer: VASCERN Data manager (EN-FR) – Sept 2018
Document resource manager - Clinical signs
Please send your CV and cover letter to:
- Annie OLRY
- E-mail : jobs.orphanet@inserm.fr
- Tél : +33 (0)1 56 53 81 37
French documentary resource manager - Editor of "Focus Handicap" collection - Disability fact sheets
Please send your CV and cover letter citing the reference US14-2018-05 to:
- Ana RATH
- E-mail : jobs.orphanet@inserm.fr
- Tél : +33 (0)1 56 53 81 51
Media
Openly Rare with Paul Kidwell podcast launches with conversation about rare disease film festival
"Openly Rare", a monthly podcast series dedicated to rare diseases, launched in August. The podcast is aimed at featuring discussions with researchers, newsmakers, patient advocates, business leaders and rare diseases experts. The podcast launched with a conversation between Paul Kidwell and the co-creators of "DISORDER: The Rare Disease Film Festival", of which the second installation will take place in 2019 in San Francisco.
A NORD webinar on genetic testing
In this recording, NORD welcomed Debra Regier, MD, Director of Genetic and Genomic Education at Children's National Hospital in Washington, DC, to give an overview of the different types of genetic tests, indications for their use, and their benefits and limitations. The recording also includes stories from patients and families who have had genetic testing and the impact it has had on their lives.