PPIE
Patient and Public Involvement and Engagement, or PPIE, includes many ways which people with lived expeirence get involved alongside researchers to help shape:
What research gets done
How it’s carried out
How the results are shared and applied in practice
About PPIE
PPIE is important in ensuring that research is relevant, ethical and impactful. It is an integral part of the LifeArc Centre for the Acceleration of Rare Disease Trials (ARDT), and is delivered through our Lived Experience Advisory Panel (LEAP).
Members of the LEAP were recruited to represent a diversity of experiences with rare conditions and a mix of characteristics such as their age, ethnicity and where they live in the UK. This diversity helps ensure that the perspectives informing the Centre’s work reflect the breadth of the rare disease community.
At ARDT, PPIE is embedded across the Centre’s activities. People with lived experience of rare conditions provide advice that help shape how research is designed, communicated and delivered. Their involvement helps ensure that studies consider the practical realities faced by patients and families, from recruitment and participation to the outcomes that matter most to those living with rare conditions.
By working in partnership with patients and the public, ARDT aims to strengthen the quality and impact of rare disease research. Embedding lived experience into our work helps ensure that clinical trials are more accessible, meaningful and responsive to the needs of the communities they are intended to serve.
The Lived Experience Advisory Panel
The Lived Experience Advisory Panel (LEAP) is the central mechanism through which patient and public involvement is delivered across ARDT. The panel brings together people who have personal experience of rare conditions, either as patients themselves or as carers and family members. Their insights help ensure that the Centre’s work remains closely connected to the needs and priorities of the rare disease community.
LEAP members are embedded across the ARDT work packages, where they contribute their perspectives to different aspects of the Centre’s activity. This allows lived experience to inform a wide range of work, from the development of clinical trial approaches and recruitment pathways, to the ways in which research is communicated and shared.
In addition to their involvement across the work packages, the LEAP meets once every quarter as a group. These meetings provide an opportunity for members to share their experiences, discuss the progress of the Centre, and provide collective feedback on key areas of ARDT’s work.
Through this combination of regular panel meetings and ongoing involvement across the project, the LEAP helps ensure that patient voices are consistently represented throughout the Centre’s activities. This approach strengthens ARDT’s commitment to conducting rare disease research that is informed by lived experience and responsive to the communities it aims to support.
PPIE Jargon Buster
Research can sometimes use unfamiliar words. This guide explains common terms used in rare disease research and clinical trials, along with useful acronyms.
Common Terms
Clinical Trial
- A study that involves people and tests whether a treatment or approach is safe and works well.
Rare Disease
- A condition that affects a small number of people (usually fewer than 1 in 2,000 in the UK).
Protocol
- The plan for a study. It explains what will happen, who can take part, and how the research will be carried out.
Recruitment
- How people are found and invited to take part in a study.
Eligibility Criteria
- The rules that decide who can or cannot take part in a study.
Informed Consent
- Making sure people understand a study before they agree to take part.
Patient-Reported Outcomes (PROs)
- Information shared directly by patients about how they feel or function, often through questionnaires.
Endpoint
- What the study is measuring (for example, whether symptoms improve).
Placebo
- A “dummy” treatment with no active ingredient, sometimes used for comparison.
Randomisation
- A way of assigning participants to different groups by chance (like flipping a coin), to keep the study fair.
Data
- Information collected during a study.
Ethics Approval
- Approval from an independent group to make sure the study is safe and fair.
Translational Research
- Research that turns scientific discoveries into real treatments or changes in healthcare.
Rare Disease–Specific Terms
Registry
- A database that collects information about people with a specific rare disease. It helps researchers understand the condition and plan studies.
Natural History Study
- A study that follows people over time to understand how a disease develops without treatment.
Recruitment Portal
- An online system that helps match patients to relevant clinical trials.
Small Patient Population
- Rare diseases often affect small numbers of people, which can make research and recruitment more challenging.
Decentralised Trial
- A study where some or all activities happen remotely (for example, from home) rather than in a hospital.
Biomarker
- A measurable sign in the body (such as a blood test result) that can show how a disease is progressing or how a treatment is working.
Genetic Testing
- Testing DNA to help diagnose rare conditions or understand how a disease works.
Undiagnosed Patient
- Someone who has symptoms but does not yet have a confirmed diagnosis.
Trial Design
- How a study is set up to answer a research question (for example, how many people take part and what is measured).
Common Acronyms
ARDT – Acceleration of Rare Disease Trials
PPIE – Patient and Public Involvement and Engagement
LEAP – Lived Experience Advisory Panel
NHS – National Health Service
PROs – Patient-Reported Outcomes
EDI – Equality, Diversity and Inclusion
PI – Principal Investigator (lead researcher)
CI – Chief Investigator (overall lead for a trial)
RCT – Randomised Controlled Trial
HRA – Health Research Authority
MHRA – Medicines and Healthcare products Regulatory Agency
GDPR – General Data Protection Regulation
IP – Intellectual Property
PhD – Doctor of Philosophy (research degree)
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Interested in working with the team?
We are always looking for talented researchers, clinicians, and industry partners to join our efforts in accelerating rare disease trials. Whether you’re interested in research collaboration, clinical trial design, or patient engagement, we’d love to hear from you.