PREFER
Research type
Research Study
Full title
PREFER Project Case Study: Quantifying Patient Preferences in Neuromuscular Disorders
IRAS ID
285949
Contact name
Grainne Gorman
Contact email
Sponsor organisation
Newcastle University
Duration of Study in the UK
1 years, 8 months, 31 days
Research summary
This study forms part of the Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) project.
PREFER is a public-private collaborative research project under the Innovative Medicines Initiative (IMI) aiming to develop expert and evidence-based recommendations on how and when patient preferences can be assessed and used to inform medical product decision making.
This study (PREFER project case study: Quantifying patient preferences in neuromuscular disorders) consists of a large-scale online survey (consisting of questionnaires) that will be delivered internationally via Patient Organisations, Patient Registries and via the NHS Highly Specialised Mitochondrial Service for Adults and Children.
Participants (patients with myotonic dystrophy type 1 [DM1] and mitochondrial disease, and caregivers of patients with these conditions) are asked to complete a series of online questionnaires about their sociodemographics, their health, how their condition affects their day-to-day life, and about their opinions/preferences regarding potential treatments for their condition.
This case study aims to explore NMD patients and caregivers’ preferences for future NMD treatments. Additionally, it will contribute to the overall PREFER Project final recommendations.
Initially the study was designed for delivery outside of the NHS (via patient organisations and charities only). However in recognition of the challenges associated with recruitment into studies in rare disease, and due to the sample size required, it is felt that extending the study to allow recruitment via the NHS will improve recruitment and allow patients with less access to social media and patient organisations to participate.
The NHS organisation involved in the project (the Newcastle upon Tyne Hospitals NHS Foundation Trust) will be acting as a Participant Identification Centre rather than a ‘full’ study site.
REC name
North West - Greater Manchester West Research Ethics Committee
REC reference
20/NW/0367
Date of REC Opinion
14 Oct 2020
REC opinion
Further Information Favourable Opinion