News - Rare Disease Moonshot

14 May 2025

Rare Disease Moonshot’s contribution to the Consultation on the next Multiannual Financial Framework

Introduction The Rare Disease Moonshot is a multi-stakeholder initiative uniting public and private actors, researchers, clinicians, patients, and funders to advance research and care for the 30 mill...

18 Apr 2025

Rare Disease Moonshot’s contribution to the Call for Evidence on the EU Life Science Strategy

Our submission on the call for evidence on the new Strategy for European Life Sciences can be found here. The Rare Disease Moonshot welcomes the European Life Sciences Strategy (LSS) as a transf...

14 Mar 2025

Rare Disease Moonshot contribution to the Call for Evidence on the EU Start-up and Scale-up Strategy

Start-ups and scale-ups play a crucial role in the development of innovative medicines in high unmet needs areas, such as rare disease, paediatric medicines or personalized medicines. To overcome regu...

19 Feb 2025

Rare Disease Moonshot welcomes the EU Competitiveness Compass

The European Commission’s Competitiveness Compass recognizes biotechnology, life sciences, and innovation as key drivers of economic growth and competitiveness. This communication builds on previous...

04 Feb 2025

The Rare Disease Moonshot and Together for Rare Diseases coalitions advocate for reimagining ERNs as research powerhouses with PPPs at their core

The European Commission's recent evaluation of European Reference Networks (ERNs, representing a structured network comprising more than 1,600 highly specialised healthcare units from almost 400 hospi...

18 Nov 2024

Breaking barriers in Rare Disease Research: The Rare Disease Moonshot at two years

Rare diseases, despite affecting millions worldwide, remain an underserved area in medical research. Over 7,000 rare diseases have been identified, yet fewer than 5% have available treatments. This si...

18 Oct 2024

Virtual Event – Innovating for Impact: Shaping the Future of Rare Disease Treatment

Millions of Europeans are living with rare diseases where no treatment option exists. How can the work from the public and private sector help in accelerating research and raise awaren...

16 Apr 2024

Translational research needs recommendations

The Rare Diseases (RD) Moonshot was set up to boost public private collaborations in the areas where there are with no treatment options and there is no R&D going on. The RD Moonshot part...

29 Feb 2024

Rare Disease Moonshot supports the EURORDIS-Rare Diseases Europe’s call for an EU action plan for Rare Diseases

On the rarest of days, we, the members of the Rare Diseases Moonshot, stand together to celebrate the resilience, strength, and perseverance of those living with rare diseases. This day serves as...

12 Feb 2024

Recording Now Available: EJP RD-EFPIA Joint Advanced Webinar on Real-World Data, Machine Learning, and Deep Analytics

We are delighted to announce the successful outcome of the EJP RD-EFPIA Joint Advanced Webinar on Real-World Data, Machine Learning, and Deep Analytics in rare diseases, which accumulated enthusiasti...

17 Jan 2024

Training Webinar: Real-World data, Machine learning and Deep analytics in rare diseases: Regulatory grade data collection for marketing authorization submissions – what is buzz, what is realistic?

Marc Van Dijk (UCB) and Luis Pinheiro (EMA) will present views of industry and regulators on various facets of collecting, processing, and using data for research, regulatory and healthcare decisio...

10 Jan 2024

Rare Disease Moonshot: Europe’s Public-Private Coalition to Erase the Rare Disease “White Spots”

The knowledge and infrastructure required to unlock rare disease “white spots,” diseases where there is currently no science or translational capability, call for a different strategic paradig...

18 Dec 2023

Diagnostic research needs recommendations

The Rare Diseases (RD) Moonshot was set up to boost public private collaborations in the areas where there are with no treatment options and there is no R&D going on. The RD Moonshot part...

11 Jul 2023

Clinical trials research needs recommendations

The Rare Diseases (RD) Moonshot was set up to boost public private collaborations in the areas where there are with no treatment options and there is no R&D going on The RD Moonshot partners id...

14 Jun 2023

Rare Diseases Moonshot partners welcome the pre-publication of the draft future Innovative Health Initiative (IHI) call topic: ‘Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases’

The Rare Disease (RD) Moonshot partners welcome the inclusion of the rare and ultra-rare diseases in a recent IHI call topic which aims to establish novel approaches to improve clinical trials to ulti...

28 Feb 2023

How the Rare Disease Moonshot can unlock science for patients

A guest blog by Magda Chlebus, Executive Director Science Policy and Regulatory at EFPIA Sometimes the most exciting areas to work in are those facing complex challenges, where the scope for making...

12 Dec 2022

TOWARDS A RARE DISEASE MOONSHOT: SCALING UP PUBLIC PRIVATE PARTNERSHIPS TO ACCELERATE RESEARCH

If you missed the launch of the Rare Disease Moonshot, you can watch it here...

08 Dec 2022

‘Rare Disease Moonshot’ – Scaling-up public-private partnerships to accelerate research into world’s rarest diseases

A new initiative to boost research and development into rare and paediatric diseases will launch today at the European Health Summit. The ‘Rare Disease Moonshot’ is a commitment and collabora...