How ERDERA addresses PPIE

ERDERA embeds Public and Patient Involvement and Engagement (PPIE) as a core, cross-cutting function within its governance and all work packages, ensuring that people living with rare diseases are involved as co-designers, implementers, and evaluators of research. 

How PPIE is put into practice

Acts as the central coordination hub for patient involvement across the programme 

Monitors work packages and works with their leaders to embed meaningful patient participation 

Identifies where and how patients can contribute across the research lifecycle (design, calls, governance, dissemination)

Supports onboarding and capacity-building of patient advocates

Ensures research priorities, communication, training, and national alignment activities remain patient-centred

Overall, PPIE in ERDERA is not a standalone activity but a system-wide approach integrated into governance, funding processes, and research implementation. 

Learn more on what ppie is

Who drives PPIE in ERDERA

EURORDIS

AFM-Téléthon 

World Duchenne
Organisation

Rare Diseases
International

Health Research
Charities Ireland

Genetic Alliance
UK 

These partners form a multi-organisation patient-led group that meets regularly and acts as the focal point for patient engagement across ERDERA. 

International perspective

Funders such as the National Institute for Health and Care Research (NIHR), the Health Research Board (HRB) in Ireland, the Canadian Institutes of Health Research (CIHR) and the National Institutes of Health (NIH) increasingly expect meaningful PPIE in funded projects.

Across these systems, the shared goal is co-production of research with those it is intended to benefit.

Key takeaway

ERDERA operationalises PPIE through a dedicated, patient-led coordination group embedded in governance, ensuring that patient perspectives systematically shape strategy, research design, funding, and implementation across the entire partnership.

Resources related to PPIE are available within the Learning Portal and can be accessed by applying the relevant filters.

Discover available resources

Black Pearl Awards

EURORDIS Black Pearl Awards to recognise rare disease community contributions in 2027

Established in 2012, the EURORDIS Black Pearl Awards recognise contributions across the rare disease community. The programme will return to Brussels and online on 15 February 2027.
Blueprint EU flag

European Blueprint sets out community priorities for a coordinated EU Action Plan on Rare Diseases

EURORDIS’ European Blueprint proposes 18 priority actions across six areas, offering a community-led foundation for a future EU Action Plan on Rare Diseases.
Engaging children, adolescents and young adults living with rare diseases as research partners: practical considerations for meaningful involvement

Engaging children, adolescents and young adults living with rare diseases as research partners: practical considerations for meaningful involvement

An International Rare Diseases Research Consortium (IRDiRC) working group developed practical guidance and a checklist for involving young people meaningfully in rare disease research.
Sveriges riksdag (house of parlament).

Sweden adopts first national strategy for rare health conditions

Sweden has adopted its first national strategy for rare health conditions, aiming to improve coordination, equity and access to care between 2026 and 2030.
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Access our training and learning offer

Multi Format

Open Academy x ERDERA Training on Data, Ethics and AI

Hybrid Open Academy x ERDERA training equipping rare disease advocates and early‑career researchers to navigate health data, ethics and AI in European rare disease research.
multi-format
•
intermediate
  • 2026   |    AI technologies   |    Data Collection and Sharing   |    Data Stewardship   |    Early Career Researchers   |    ERDERA   |    Ethics   |    EURORDIS–Rare Diseases Europe   |    Open Academy   |    Patient Advocacy   |    Regulatory
Workshop

2nd ERDERA Training for Young Advocates for Rare Diseases

This training aims to empower young patients by providing them with the knowledge, confidence and skills needed to actively take part in paediatric and rare disease research.
workshop
•
basic
  • 2026   |    Clinical Trials   |    ERDERA   |    Genetic diseases   |    Institut Imagine   |    Paediatric   |    TEDDY   |    Young patients
E-Learning

Management of multinational clinical trials for rare diseases

Practical online course on planning and managing multinational clinical trials across Europe, including regulatory/ethics, roles, budgets, and patient engagement.
e-learning
•
basic
  • All rare diseases   |    Clinical research   |    Clinical Trials   |    ERDERA   |    Funding   |    Multinational trial management   |    Policy and Regulation   |    PPIE

Closed training

Materials available

Enhancing Patient-Centricity in Rare Disease Clinical Trials

This webinar explores patient-centred approaches to rare disease clinical trials, focusing on how patient priorities can be incorporated into trial design and analysis.
webinar
•
intermediate
  • Clinical Trials   |    Patient Advocacy   |    Patient engagement   |    Patient Public Involvement and Engagement (PPIE)   |    Ultra-rare diseases
In Person

Open Academy x ERDERA School on Scientific Innovation & Translational Research

The Open Academy School on Scientific Innovation & Translational Research is part of the Open Academy Schools x ERDERA and consists of a 4-day in-person training in Barcelona.
in-person training
•
intermediate
  • 2027   |    ERDERA   |    EURORDIS–Rare Diseases Europe   |    Innovation   |    Open Academy   |    Translational Research
In Person

Open Academy x ERDERA School on Medicines Research and Development

The School on Medicines Research & Development is part of the Open Academy Schools x ERDERA and is a 4-day face-to-face training, held in June in Barcelona.
in-person training
•
intermediate
  • 2027   |    Data Stewardship   |    ERDERA   |    EURORDIS–Rare Diseases Europe   |    Medicines Research and Development   |    Open Academy
Multi Format

Open Academy x ERDERA Training on Data, Ethics and AI

Hybrid Open Academy x ERDERA training equipping rare disease advocates and early‑career researchers to navigate health data, ethics and AI in European rare disease research.
multi-format
•
intermediate
  • 2027   |    AI technologies   |    Data Collection and Sharing   |    Data Stewardship   |    Early Career Researchers   |    ERDERA   |    Ethics   |    EURORDIS–Rare Diseases Europe   |    Open Academy   |    Patient Advocacy   |    Regulatory
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