Evidence-based valuation of patient-centred outcomes in Cystic Fibrosis - HEP02


This is a National Institute for Health Research (NIHR) Research for Patient Benefit (RfPB) project, focused on improving the measurement and valuation of patient-centred outcomes in cystic fibrosis.


Cystic fibrosis (CF) is a genetic condition that affects more than 10,000 people in the UK. People with CF accumulate thick, sticky mucus in their lungs, gut and other organs. They need to follow a time-consuming treatment routine every day to maintain their health. For example, most adults with CF dedicate between one and four hours every day to their treatments. This treatment burden can impact everyday life significantly for people with CF and their families, interfering with their work, school, university or home life. It often makes routine activities more difficult and limits social activities. In addition to their usual treatments, a lot of extra treatment – such as antibiotic injections, hospital visits and stays in hospital – is necessary when they are unwell. Feedback from the CF community has indicated that reducing the treatment burden is considered to be their top research priority.

Health-related quality-of-life and treatment burden are particularly important to patients and caregivers but are poorly considered in assessments of new treatments. This research aims to address this issue and ensure a more accurate valuation of patient-centred outcomes within the decision-making process for access to new treatments and clinical practice. In the longer term, this research will bring a more precise understanding of what it means to say that a CF treatment 'works'.

Project aims

This research is designed to improve our ability to incorporate patient-centred outcomes (specifically health-related quality of life (HRQoL) and treatment burden), systematically in economic evaluations and funding decisions. There are three specific objectives:

  1. To investigate the relative importance of different treatment outcomes for people with CF.
  2. To measure the HRQoL impact associated with reduced daily treatment occasions.
  3. To compare two validated HRQoL measures to determine which measures the best capture the aspects of quality-of-life which are important to people with CF.

Project activity 

The project started in October 2019, with fieldwork conducted between July 2020 and March 2021.

People with CF were involved in all stages of the project, from grant development through to survey instrument design and interpretation of results.

People with CF attending the adult CF clinic at the Royal Brompton hospital in London were invited to participate in the research. A series of two web-based surveys and an online interview were conducted with participants.

The potential or actual impact 

Outputs will encourage the NHS to deliver the interventions that are most valued by CF patients. Patients will benefit because a better understanding and valuation of quality-of-life and treatment burden will enable better-informed decisions around the funding of interventions. Decision-makers will benefit from a more rigorous evidence base on which to make funding decisions. Society more broadly will benefit from increased health system efficiency (that is, more of the health outcomes most important to patients will be provided for the same cost).  

In the longer term, this research will support patient access to CF treatments and help ensure the sustainability of our NHS. Moreover, the focus on outcomes that are important for patients will be publicised and promoted to clinicians, leading to an anticipated uptake of their routine measurement in CF clinical care. This project has the potential to directly and rapidly benefit patients through promoting a focus on patient-reported outcomes in clinical practice.

Papers/ resources

Much of the research conducted in this study will form the basis of Rana Altabee’s PhD.

Two papers are currently in development and are expected to be submitted in the coming months, with at least two more expected to follow later in 2021/22.

A poster abstract has been accepted for the European Cystic Fibrosis Society conference in June. A further two abstracts were recently submitted for the North American Cystic Fibrosis Conference in September.

Next step 

Finalising analysis, writing up and disseminating findings.

Recent activity 

Fieldwork was completed in March 2021.

Analysis has been finalised for one major component of the research and is underway for the remainder of the study.

Related projects


Who is involved?

Co-PI: Dr Siobhan Carr

Consultant in Paediatric Respiratory Medicine, Imperial College London, Royal Brompton and Harefield NHS Foundation Trust

Co-PI: Jennifer Whitty, ARC EoE HEP

Researchers and Institutions 

Health economics lead:

Dr Rory Cameron, ARC EoE HEP/UEA


Rana Altabee,

Health Economics PhD student, UEA


Host organisation:  Royal Brompton and Harefield NHS Foundation Trust

Collaborating institutions:

University of Central Lancashire

Imperial College London

London School of Hygiene and Tropical Medicine


Dr Siobhan Carr

Consultant in Paediatric Respiratory Medicine, Imperial College London, Royal Brompton and Harefield NHS Foundation Trust