Ongoing Studies & Research
Ongoing Studies & Research
Patient-Centred Research
Patient-Centred Research
Rare Disease Resources
Rare Disease Resources
Websites & Organisations
Websites & Organisations
Conferences
Conferences
Training
Training
Upcoming Events
Upcoming Events
PPI Opportunities
PPI Opportunities

Be Part of Research

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A search tool designed to connect people with a condition to ongoing NIHR trials of interest.
Trials
Studies
Involvement
Visit

GRACE Project: OpenLearn

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The GRACE project researches and engages directly with communities, giving them a voice, and identifying barriers and concerns which prevent their participation in genetics.
Genetics
Involvement
PPIE
Engagement
Visit

Newcastle Centre for Rare Disease

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Collaborates across disciplines for patient-centred impact and innovation; improves therapy options, outcomes, and quality of life for people with a rare disease.
Clinical Trials
Funding
Care Delivery
Visit

NIHR BioResource

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Bringing together researchers and willing volunteers to contribute to leading research that will benefit everyone.
Samples
Data access
Centres
Recruiters
Visit

NIHR Rare Diseases Research Landscape Report

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Presents an overall picture of rare disease research taking place across the UK over the last 5 years.
Funding
Treatment
UK Rare Diseases Framework 2021
Industry
Visit

Rare Autoimmune Rheumatic Disease Alliance (RAIRDA)

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Conglomerating research across these conditions and promoting research priorities.
Research
Collaboration
Visit

Rare Disease Research UK

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UK-wide Platform, aiming to impact the Rare Disease research landscape and improve the lives of those directly or indirectly affected by Rare Diseases.
Treatments
Platform
Clinical Network
Visit

Rudy Study

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Rare disease study ongoing (NIHR-funded). Headed up by a research team at the University of Oxford, Rudy aims to transform clinical care for participants through patient-driven research.
Funding
Patient
Forum
Visit

We R Rare

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Steering Group with direct experience of rare conditions across Manchester and North West England. Members use their experiences of rare conditions to shape the research, and services, provide strategic advice to the Manchester Rare Conditions Centre and the Manchester Biomedical Research Centre, and influence the latest research projects.
Patient Voice
Involvement
Steering Group
Visit

Co-production Collective

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Supports co-production projects and enables others to co-produce, bringing people together to share learning and ideas, and build evidence and influence to make the case for co-production.
Co-production
PPIE
Networking
Visit

Elinor Ostrom’s design principles and co-creating public value (study)

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Study on co-production and group collaboration: key principles.
Co-production
Co-design
Collaborative group working
Visit

Equality, Diversity and Inclusion Toolkit: NIHR

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Developed to support researchers and research advisers to better understand how to embed equality, diversity and inclusion (EDI) in research design and to meet the National Institute for Health and Care Research (NIHR) Research Inclusion.
EDI
Toolkit
Visit

Global Genes Research Readiness Fellowship

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Provides patient advocacy leaders with the opportunity to make a tangible difference in the lives of rare disease patients and caregivers across the globe.
Mentorship
Training
Visit

Guidance on Co-producing a Research Project: NIHR

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A first step in explaining what is meant by co-producing a research project, setting out the key principles and features of co-producing a research project and suggesting ways to realise them whilst outlining some of the key challenges.
PPIE
Co-production
Visit

‘How to’ toolkit for creating accessible condition-specific information: Genetic Alliance

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Alongside Genomics England, Genetic Alliance UK has developed a ‘how to’ toolkit to write genetic and rare condition-specific information that is accessible for people with a wide range of requirements.
Toolkit
Language
Guide
Visit

I am Rare Programme: NORD

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Easy to use system that allows patients and organisations to inform and shape medical research and translational science for rare diseases by launching high-quality, customised registries to collect the data needed to define the natural progression of their disease.
Translational research
Registries
Natural history studies
Visit

Involving Children and Young People as Advisors: NIHR

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Guidance on engaging younger populations in research, ensuring their perspectives are included in studies that affect them.
PPIE
Children
Young people
Visit

Patient-Centred Outcomes Research Institute (PCORI)

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Funds patient-centred comparative clinical effectiveness research (CER) and research-related awards; promotes the use of patient-centred evidence.
Funding
Comparative research
Awards
Visit

Patient Engagement Training and Resources Repository: Synapse Connect

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Resource and training sharing repository for patient engagement across the globe.
Training
Resources
PPIE
Visit

PEQG – Patient Engagement Quality Guidance

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This tool focuses on enhancing patient engagement in the development of medicines, ensuring that patient input is integral to the process.
Clinical trials
PPIE
Guidance
Medicines
Visit

Patient Engagement Resource Centre

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Outlines the basics of patient engagement through the different phases: from planning to conducting and evaluating.
PPIE
Impact & evaluation
Training
Visit

Patient Experience Library

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Puts a wealth of patient experience evidence in one place with free search and unlimited downloads; can create a diary for specific research needs.
Lived Experience
Patient data
Visit

Plain English Summaries: NIHR

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Tools and guidance to help you write clear, accessible research summaries that are easily understood by a wide audience.
Guide
Language
Plain Language Summaries
Visit

PPI review (national: UK paper)

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Research paper nationally evaluating patient and public involvement in the UK.
Best practice
Co-creation
PPIE
Visit

Rare Diseases Clinical Research Network (US-based)

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Designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing.
Clinical studies
Data sharing
Translational research
Visit

Reaching Out - A Guide to Inclusive Public Involvement: NIHR

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Guidance on how to involve diverse communities in your research, ensuring your work is inclusive and representative.
EDI
PPIE
Visit

Toolkit for patient-focused therapy development: US Health Services

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Provides information and resources about the growing role of patient voices from rare disease communities in treatment & development.
Patient Voice
PPIE
Treatments
Visit

Beacon Resources Hub

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Beacon is a UK-based charity building a united rare disease community with patient groups at its heart. The Resources Hub is a proud part of Beacon’s patient group training programme.
Training
Patient voice
Resources
Visit

Great Ormond Street Hospital for Children NHS Foundation Trust

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Research across Rare Disease Cohorts (RDC) often requires and uses similar resources despite occurring across a vast range of diseases. In order to create cohesion across rare disease research taking place at NIHR BRC at GOSH, they have created a platform for exemplary resources to be accessed.
Resources
Platform
NIHR
Visit

Medscape Rare Disease Resources section

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Online global destination for physicians and healthcare professionals worldwide, offering the latest medical news and expert perspectives; essential point-of-care drug and disease information; and relevant professional education and CME.
Medical news
Disease information
CME
Clinicians
Visit

NHS Rare Disease Collaborative Networks

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Signposting to specialist hospitals and researchers working on rare conditions.
Clinicians
Researchers
Signposting
Visit

PFMD (Patient Focused Medicines Development)

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PFMD aims to transform how we understand, engage, and partner with patients globally in the design and conducting of research and development of medicines by focusing on unmet patient needs. They are bringing together relevant experts and synergising disparate but complementary efforts that integrate the voice of the PATIENT across the lifecycle of medicine.
PPIE
Multi-stakeholder
Medicines
Visit

UK Rare Disease Research Platform

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Brings together expertise from across the UK's rare disease research system to foster new and innovative treatments for those directly and indirectly impacted by rare conditions.
Funding
Policies
Collaboration
Visit

Action

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The leading UK-wide charity dedicated to funding vital research to help sick and disabled babies, children and young people.
Funding
Events
Research
Visit

CamRARE

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CamRARE supports the rare community, builds multi-stakeholder networks, hosts events, undertakes research and facilitates and gives access to experts through lived experience.
Networking
Events
Families
Community
PPIE
Industry forum
Visit

Contact

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CamRARE supports the rare community, builds multi-stakeholder networks, hosts events, undertakes research and facilitates and gives access to experts through lived experience.
Family
Community
Advocacy
Visit

EURODIS

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EURORDIS-Rare Diseases Europe is a unique, non-profit alliance of over 1000 rare disease patient organisations from 74 countries that work together to improve the lives of over 300 million people living with a rare disease globally.
Advocacy
Diagnosis
Events
Visit

GARD

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Public health resources supporting people with a rare disease and their caregivers by providing access to easy-to-understand information that is free and reliable.
Disease database
Data access
Visit

GeNotes Knowledge Hub (KH)

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Quick, concise information to help healthcare professionals make the right genomic decisions at each stage of a clinical pathway. KH = encyclopaedia of resources, designed to support your understanding of genomics in medicine.
Clinicians
Support groups
Diagnosis
Visit

LDA Research

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International medical market research for the pharmaceutical industry and medical device sector; Specialists at recruiting hard-to-find individuals from across the globe.
Podcast
Recruitment
Medical devices
Visit

Medics4RareDiseases

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Medics4RareDiseases is a registered charity driving an attitude change towards rare diseases amongst medical students and doctors in training.
Clinicians
Training
Events
Visit

NORD

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NORD advances practical, meaningful, and enduring change so people with rare diseases can live their fullest and best lives.
Support groups
Patient voice
Rare disease database
Visit

Orphanet

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Orphanet is a unique resource, gathering and improving knowledge on rare diseases to improve the diagnosis, care and treatment of patients with rare diseases.
Drug development
Thematic reports
Directory of patient organisations
Visit

Our Health Service

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Provides information about genetic and rare diseases and social care support.
Support groups
Clinical trials
Healthcare policy
Visit

Rare Disease Clinical Trial Network

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Collaboration hub that links with international expertise, and positions Ireland to be world-leading in patient access to rare disease clinical trials.
Clinical trials
Feasibility
Collaboration
Visit

Rare Disease Collaborative Networks: CUH

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University hubs for rare diseases for patient care & medical.
Networking
Collaboration
Hub
Visit

Shared Learning Group on Involvement & Charities Research Involvement Group

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A group that meets every few months to discuss charities research involvement and PPI.
PPIE
Training
Best practice
Visit

Wales Gene Park

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Provides researchers and industry partners access to genomic expertise and technologies.
Bioinformatics
Sequencing
Genomics
Visit

Cambridge Rare Disease Network

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Rare Summit & Rare Fest (biennial) - bringing the rare disease community together for networking, expert speakers, cutting-edge science and technology, celebrating patient stories, and raising awareness.
Community
Networking
Raising awareness
Patient voice
Multi-stakeholder
Visit

EJP Rare Disease Events

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The European Joint Programme on Rare Diseases (EJP RD) work on specific impacts such as improving the alignment of national/ regional activities and policies in rare diseases.
Diagnostic tools
Policies
Virtual platform
Visit

EURORDIS Open Academy

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Offers rare disease-specific comprehensive training programmes that empower advocates with the knowledge, skills and confidence they need to engage with different stakeholders as equal partners.
Leadership
Communications
Research process
Visit

EUPATI

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Provides education and training to increase the capacity and capability of patients and patient representatives to understand and meaningfully contribute to medicines research and development (R&D), and to improve the availability of medical information for patients and other stakeholders.
Clinical development trials
Medicines
Research and development
Visit

International PPI Network

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Expertise sharing & evidence-based PPI good practice.
Communications
PPIE
Research Process
Visit

Learning for Involvement Course: NIHR

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An online, free course that helps researchers understand and implement effective PPI, with modules available in accessible formats.
PPIE
Guide
Accessible
Visit

Introduction to Patient Public Involvement: University of Edinburgh

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This free course includes videos and downloadable PDFs that introduce key concepts and practical steps for successful PPI.
PPIE
Guide
Visit

Patient Engagement Management Suite

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A wide variety of training tools and resources were created using multi-stakeholder working groups, including patients and patient organisations.
Patient engagement
Working groups
Multi-stakeholder
Visit

CamRARE

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Rarefest24. Friday 22 Nov, in person, Cambridge. ‘Strictly Rare’ - art music, performance, talks. A celebration of creativity, diversity and people living with rare conditions. Saturday 23 Nov, in person, Cambridge. An interactive science, technology, community and arts exhibition with hands-on activities, talks, film, art. The RDRN platform officially launches at RAREfest24 - join our panel at 11:00am and visit our stand for fun activities. Find more info and get tickets for each event by clicking the link.
Technology
Science
Community
Visit

People in Research

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Opportunities for public involvement in NHS, public health and social care research in the United Kingdom.
PPIE
Research
Visit

James Lind Alliance

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Non-profit organisation supported by the NIHR, bringing patients, carers and clinicians together in Priority Setting Partnerships (PSPs) to identify and prioritise unanswered questions.
Collaboration
PPIE
Research priorities
Visit
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