Widening patient engagement for orphan drugs trials v.1.0
Research type
Research Study
Full title
Widening patient engagement for orphan drugs trials
IRAS ID
304904
Contact name
Julia Frost
Contact email
Sponsor organisation
University of Exeter
Duration of Study in the UK
2 years, 5 months, 30 days
Research summary
Background:
Orphan drugs are medicines for diagnosis, prevention or treatment of life-threatening or progressive disorders that are rare (affecting less than 1 in 2,000 people). An example is Idiopathic Pulmonary Fibrosis (IPF).
The pharmaceutical industry has identified the importance of developing orphan drugs for medical and financial reasons. While the UK is a leader in the early stages of drug development, it falls behind other counties in the later phases, which assess drug safety or how it compares with other treatments. Furthermore, the UK government and pharmaceutical industry acknowledge gaps in current knowledge about the experience of patients in clinical trials of orphan drugs who do not receive the active treatment or who drop out of a trial.
To help bridge this gap, an experienced qualitative researcher has been seconded to a European-wide pharmaceutical company (Galapagos NV) to facilitate knowledge exchange and identify opportunities for deep learning for the UK pharmaceutical industry and patients with orphan conditions.
Aims:
Learn more about orphan drug development and patient experience of IPF.
Embed qualitative research in pharmaceutical practice enabling a wider group of patients to participate in orphan drug research.
Expand orphan drug engagement to widen their reach and benefit, improve clinical trials and medicine use, to benefit both people with orphan conditions and the pharmaceutical industry.
Methods:
* In-depth interviews with patients who have been diagnosed with IPF, healthcare professionals, and clinical trial staff.
* Interviews with staff and observations of Galapagos' values and practice.
* Training Galapagos staff to conduct qualitative research, and assess their effectiveness.
* Patient and Public Involvement (PPI) representatives will regularly input into the research findings and co-design accessible resources for patients, and organisations that recruit patients to orphan drug trials. Outputs will address industry objectives and include patient-facing resources for recruitment to trials of orphan drugs, seminars for scientists and academics, and peer reviewed publications.REC name
London - Riverside Research Ethics Committee
REC reference
22/LO/0186
Date of REC Opinion
28 Mar 2022
REC opinion
Favourable Opinion