VALU-CF

• Research type

  Research Study

• Full title

  Evidence-based valuation of patient-centred outcomes in Cystic Fibrosis

• IRAS ID

  258284

• Contact name

  Siobhan Carr

• Contact email

  S.Carr@rbht.nhs.uk

• Sponsor organisation

  Royal Brompton and Harefield Foundation Trust

• Duration of Study in the UK

  1 years, 6 months, 1 days

• Research summary

  The proposed research will improve our ability to value outcomes in cystic fibrosis (CF) from the perspective of those for whom they are most relevant – the patients and their families. It will focus clinicians and health services on delivering these outcomes and considering them when treatment choices are made.
  We propose a mixed methods study (survey and interview) to enable us to understand the relative importance of different treatment outcomes in CF for the patients perspective, and to derive utility weights for two of these outcomes for use in future for use in economic evaluations of CF interventions: reduced pulmonary exacerbations with/without hospitalisation, and reduced treatment burden. The survey will include validated instruments to help us better understand both perceived treatment burden in CF and its relationship with health related quality of life (HRQoL). To measure treatment burden, we will use the generic Multimorbidty Treatment Burden Questionnaire (MTBQ) which has been validated for use in older adults with chronic conditions. We will also collect perceived treatment burden using the three items that form the treatment burden domain of the CFQ-R condition-specific HRQoL measure and the three treatment burden items included in the CFQoL HRQoL measure. To measure HRQoL, we will use the EQ-5D-5L tool which is a generic tool with limited clinical utility but it is a recognised measure for use in cost utility analyses For participants who have taken part in the LwCF study, we will also ask permission to link their reponses with data from the LwCF study (which is collecting HRQoL data using three HRQoL questionnaires). At the time of the survey and interviews, clinical information on disease severity will be recorded. This is routinely collected data that is part of normal clinical care and no extra tests or measures will be made for this research.

• REC name

  Yorkshire & The Humber - Leeds West Research Ethics Committee

• REC reference

  19/YH/0423

• Date of REC Opinion

  18 Feb 2020

• REC opinion

  Favourable Opinion