ERN Workshop on Endocrine cancer: A challenge in adults and children.

In the context of EJP RD’s ERN Workshops, a face-to-face workshop on “Endocrine cancer: A challenge in adults and children” aimed at giving an update on the molecular background and clinical management of rare endocrine malignancies is being organised by Prof. Barbara Jarzab of the M. Sklodowska-Curie National Research Institute.
The in-person event will take place on May 4th, 2022 at the M. Sklodowska-Curie National Research Institute in Gliwice, Poland.

Registration closes on January 28th, 2022 Read

 
 
 

EMA is launching a pilot to support the repurposing of established medicines for use in new indications by NGOs and academia.

EMA and the Heads of Medicines Agencies (HMA) are launching a pilot project to support the repurposing of medicines as a follow-up to the European Commission’s Expert Group on Safe and Timely Access to Medicines for Patients (STAMP) discussions on a proposal for a medicines repurposing framework.

The aim of this initiative 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 with the view to have this new use formally authorised by a regulatory authority. This is a way of making new treatment options available to patients. Read

 
 
 

Training Webinar: Does Randomization matter in RD clinical trials?

As part of the EJP RD Training series, an advanced-level training webinar on the topic “Does Randomization matter in RD clinical trials?” will be held on December 14th.

The training consists of two parts: a 1.5-hour training in less frequently applied randomization procedures and their performance on the level of evidence, followed by 1 hour of panel discussion with experts from regulatory, pharma-industrial, academic, and clinical fields.. The event is is open to the international research community, clinicians, medical specialists, healthcare professionals and advocacy patient groups with knowledge of rare disease clinical trials.

Registration closes on December 10th.

More information: https://www.ejprarediseases.org/event/ejp-rd-training-webinar-does-randomization-matter-in-rd-clinical-trials/

 
 
 
 
 
 

Call for Members: IRDiRC Task Force on “Enabling and Enhancing Telehealth for Rare Diseases Across the Globe

The Funders Constituent Committee (FCC) have set up a Task Force to identify barriers and opportunities for the use of telehealth to improve diagnosis, care, and research experiences for rare disease patients – including technological, legal, cultural, linguistic, healthcare system, and patient/provider factors.

This will be accomplished through survey and systematic review of existing models of telehealth, their uptake and usage by the rare disease community, and their specific value and effectiveness. The Task Force will then develop “best practices” for introducing telehealth services into communities where they would be most beneficial using realistic and culturally sensitive approaches. Read

 
 
 

EJP RD Joint Transnational Call 2022, a funding opportunity for RD research projects

The European Joint Programme on Rare Diseases (EJP RD) announced the upcoming launch on December 14th of the fourth EJP RD Joint Transnational Call (JTC) 2022, aimed at funding multilateral research projects on rare diseases under the EJP-COFUND action.

The aim of the call is to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project, with the call topic being “Development of new analytic tools and pathways to accelerate diagnosis and facilitate diagnostic monitoring of rare diseases.”

Read

 
 
 

Share4Rare Call4Projects 2022 for patient-centred research initiatives

The 2nd Share4Rare Call for Research Projects will be open until December 10th 2021.

Share4Rare aims to put rare diseases patients at the centre of research, not only by asking them directly regarding their condition but also by providing them a safe platform where they can participate in collaborative research projects.

The Share4Rare platform enables to gather data from patient and caregivers on disease related questions and other areas of scientific interest: burden and impact of the disease, its natural history, how the disease affects quality of life, etc. Read

 
 
 

ERKReg Reaches 10,000 Patients

The ERKReg, a Web-based registry for all patients with rare kidney diseases, recently established by the European Rare Kidney Disease Reference Network (ERKNet), has just crossed the 10,000 patients mark.

This important milestone was reached after the 10,000th patient has been enrolled in the Registry by Ghiles Larbi from the Necker Hospital in Paris, France. Almost 1800 patients have already been enrolled from the Necker Hospital! Read

 
 
 

Announcement of the launch of “Open Research Europe”

Open Research Europe (OCE) is an open access publishing platform for the publication of research stemming from Horizon 2020 funding across all subject areas. The platform makes it easy for Horizon 2020 beneficiaries to comply with the open access terms of their funding and offers researchers a publishing venue to share their results and insights rapidly and facilitate open, constructive research discussion. Read