OBJECTIVES: Patient-centred trial designs improve research quality and efficiency. Our qualitative work suggests quantifying 6 key trial attributes could define priorities for patient-centred trials: type of medicine, trial design (placebo vs open label), washout, stipend, location of visits, and post-trial drug access.
METHODS: Preferences were determined using an online discrete choice experiment (Oct 20-Jan 21). pwCF age 16+ were recruited through social media. Respondents considered 12 scenarios, choosing between 2 hypothetical trials (or ‘no trial’) characterised by attribute levels based on current CF trials. Levels were combined using experimental design. The cross-sectional data were explored using an RPL model.
RESULTS: Responses: n= 207. Never participated in a trial: 57%. (1) The strongest influence on the decision to participate was trial location, pwCF favouring their usual centre and being less likely to participate the greater the distance from home. (2) Post-trial drug access ranked next. pwCF are less likely to take part if they do not gain access to the drug on completion. Open-Label extension phases or Managed Access Programmes were similarly persuasive in decisions to participate. Priority access to later trials did not encourage participation. (3) At this time, pwCF favoured trials of CFTR modulators over other drugs, with no strong preference between antibiotics and anti-inflammatories. (4) A larger stipend was associated with greater willingness to participate. (5) pwCF did not favour a washout period but were more prepared to washout non-modulators than modulators. (6) There was minimal difference in intention to take part in placebo vs open label trials. There was a complex interaction between placebos and washouts. Subgroup data will be presented.
CONCLUSION: We identified factors that are most important to pwCF when deciding whether to join a trial. This evidence could inform patient-centred trial design with benefits to recruitment and retention.
