Making a difference in clinical trials for rare conditions
Research type
Research Study
Full title
Ethical Legal and Social Issues (ELSI) in Rare Conditions Research: making a difference in clinical trials for rare conditions
IRAS ID
343088
Contact name
Tara Clancy
Contact email
Sponsor organisation
University of Manchester
Clinicaltrials.gov Identifier
NHS002188, Sponsor Reference
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
Collectively, rare conditions affect >3,5 million people in the UK. One thing rare conditions have in common is the need for a timely diagnosis and access to effective treatments. Evidence about the impact of treatment for rare conditions is limited because of the lack of consistency and availability of outcome measures to capture the impact on patient health, well-being and quality of life. In addition, the available evidence often uses outcomes that do not reflect the experiences and/or priorities of patients and families living with the rare condition.
We will use a qualitative interview-based study to explore patients’ and families’ needs, hopes, expectations and what they consider to be meaningful improvement in the context of clinical trials. People eligible to take part are: adults living with a rare condition who have taken part in/are currently taking part in a clinical trial; parents/carers of children and young people living with a rare condition who have taken part in/are currently taking part in a clinical trial; and, children and young people living with a rare condition who have taken part in/are currently taking part in a clinical trial. The research interviews will last 30-45 minutes and can be virtual, by phone or face-to-face (depending on the participants preference).
Potential participants will be identified by the clinicians and research teams involved in clinical trials for rare conditions which are in progress/have been undertaken at Manchester University NHS Foundation Trust.
The study will last for 3 years and is partly funded through a grant provided by the Medical Research Council and the National Institute for Health and Care Research, and by the Manchester Biomedical Research Centre.
REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
24/YH/0248
Date of REC Opinion
11 Dec 2024
REC opinion
Further Information Favourable Opinion