The ERDERA Strategic Research & Innovation Agenda (SRIA) sets out a clear route map for turning Europe into the global engine room of rare-disease research and care. Its ambition is straightforward yet bold: improve the health and wellbeing of the 30 million people in Europe who live with a rare condition, while making the EU the world leader in rare-disease science and innovation.

To reach that ambition, ERDERA’s vision is to weave a truly pan-European, multi-stakeholder ecosystem—uniting researchers, clinicians, patients, industry, regulators and funders—so that discoveries move from laboratory bench to patients’ bedsides faster and more efficiently.

Three intertwined mission strands steer the work: bring Europe’s best expertise and services under one roof; ensure every consenting patient can be found and enrolled in an appropriate study through rich, FAIR-compliant data sharing; and align regional, national and EU investments to keep Europe at the cutting edge of innovation and job creation.

The SRIA translates this mission into three headline goals:

• Faster answers – a diagnosis (or enrolment in systematic research) on average within six months of first medical attention.
• More treatment options – a surge of new, effective therapies, prioritising diseases that currently have none.
• Better lives – deeper understanding of the day-to-day impact of rare diseases to drive policies and services that genuinely raise quality of life.

Delivering on these goals hinges on practical, patient-centred action. Annual trans-national funding calls will back high-quality, needs-led research, while a Europe-wide clinical research network and state-of-the-art data infrastructure will give projects the tools and expertise they need to succeed.

Dedicated training programmes, from online masterclasses to “train-the-trainer” schemes, will grow the next generation of rare-disease specialists, and an incentivised framework ensures patients are equal partners at every step. Crucially, ERDERA builds on the strong foundations laid by the European Joint Programme on Rare Diseases and other EU initiatives, knitting them into one coherent, sustainable pipeline from discovery to care.