Explore the Rare Diseases Clinical Trials Toolbox

Clinical Trials Toolbox

Drug development programmes in rare diseases have many challenges, some of which differ from those facing researchers working on common diseases, like the lack of clinical research experts and the scarcity of patients. Moreover, academic sponsors and investigators end up getting involved not only in the scientific aspects of the research but also having to navigate the operational coordination and management themselves. The Rare Diseases Clinical Trials Toolbox has been developed as a practical aid for developers of clinical trials on medicinal products for human use regardless of therapeutic area.

The toolbox aims to collect the accumulated knowledge, experience, and [...]



ERICA WP3 ‘Patient centred research’ Expert Working Group Kick-off Meeting 8-10 March

WP3 PCEWG meeting
On the 8th and 10th of March it has been held the Kick-off Meeting of the ERICA WP3: during the 2 sessions the team and participants have discussed about PROMs for Rare Diseases and how to build an accessible repository.

View the event

The general framework of the ERICA project’ WP3 is Patient Centered Research for Rare diseases. WP3 is led by Ana Rath and María del Mar Mañú Pereira, respectively from Orphanet and ERN-EuroBloodNet, in collaboration with MAPI Research Trust.

Specifically, [...]



The European Rare Disease Research Coordination and Support Action (ERICA) 1 year!


One year ago, on March, 1st 2021 the European Rare Disease Research Coordination and Support Action (ERICA) started officially! The aim of the ERICA consortium, in which all 24 European Reference Networks (ERNs) take part, is to build on the strength of the individual ERNs and create a platform that integrates all ERNs research and innovation capacity.

It has been challenging year due to Covid circumstances, but nevertheless productive year with great start of the different WP-specific activities and their Expert Working Groups. Great work has been done by ERICA’s enthusiastic WP-leads/board members, [...]



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

ERN Workshops (9)

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



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

EMA medicines

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 Read


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

Irdirc call

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 [...]



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.”