Title: The AtOM CF Study - How can Adherence to Oral Medication be improved for the benefit of people with cystic fibrosis.
Funder: Health Education England

Abstract: BackgroundCystic Fibrosis (CF) is an inherited, chronic, multisystem disease caused by mutations in the CF Transmembrane conductance regulator (CFTR) gene1. CFTR modulator medicines are a novel, expensive treatment for CF that correct the baseline defect in CFTR dysfunction2,3. They have been shown to improve lung function, nutritional status and stabilisation of the disease, improving survival2-7. Despite positive health benefits adherence has been shown to be variable8. Understanding the reason for non-adherence is vital in assisting with intervention design to optimise the health benefits of this new treatment. Optimisation is essential as treatment costs approximately 110,000 per patient per annum9. Research Question. The AtOM CF Study - How can Adherence to Oral Medication be improved for the benefit of people with Cystic Fibrosis?Aim To develop a patient-centred complex intervention to enhance and optimise adherence to oral medication with a focus on CFTR modulators in the person with cystic fibrosis (PwCF). Objectives: To synthesise existing literature on adherence to oral medication, including CFTR modulators in PwCF, and characterise the impact of interventions tested to improve adherence. To determine adherence to CFTR modulators and assess the burden of prescribed medication in adults attending two adult CF centres in England. To explore the perceptions and experiences of CFTR modulator therapy in PwCF including in relation to health status, adherence and necessity of other routinely prescribed CF medications. Co-design an intervention using a modified Experience Based Co-Design approach to support long-term adherence to oral medication with a focus on the CFTR modulators in PwCF.Methods and timeline Work package 1 (month 1-8): Mixed methods systematic review focusing on adherence to oral medication, in the PwCF and assessment of the impact of interventions to improve adherence.Work package 2 (month 7-11) : Medicines possession ratio (MPR) will be utilised to calculate adherence rates of the CFTR modulators in the target population. The burden of prescribed medication will be quantified utilising the Living with Medicines Questionnaire version 3.Work package 3 (month 11-25): Semi-structured Interviews, utilising photo elicitation in two adult CF centres, to provide a conceptual framework for work package 4. Work package 4 (month 23-30): PwCF and staff will co-design an intervention to optimise adherence to oral medication in CF. The last six months will be dedicated to write up and dissemination. Impact and dissemination Research findings will be published in international peer-reviewed journals and presented at regional, national and international conferences. They will be shared with patients and public through social media, written summaries such as a patient newsletter and oral presentations, co-authored with PPI. Photographs obtained during work package 3 of the research will displayed in an exhibition and through social media.The intervention will be based on theory and co-designed utilising the patient's perspective. Following the MRC Framework for design and evaluation of complex interventions10, it is anticipated that there will be a feasibility post-doctoral study, providing the basis for a definitive trial in order to provide rigorous evidence regarding the impact of the intervention on adherence and patient outcomes.

Lay abstract: BackgroundCystic fibrosis (CF) is a genetic illness that affects more than 10,500 people in the United Kingdom. It is life limiting, with average life expectancy around 47 years. The CF gene affects how salt and water moves in and out of cells. This means that people with CF get thick, sticky mucus in their lungs, digestive system and other organs. Until recently treatment could only help control and reduce the effects of mucus build up. It includes medication, physiotherapy, exercise and a high calorie diet. Daily treatment regimens are complex, take around two hours to complete and they are life-long. A new treatment is now available - tablets called modulators. They improve how salt and water move in and out of cells. This improves symptoms, stabilises the illness and prevents further damage to organs such as the lungs. People taking modulators as intended should live longer and with fewer periods of severe illness. However, it appears that some people do not take modulators as prescribed. This not only affects the life of the person with CF, but also NHS resources as frequent admissions to hospital are needed. The new treatment itself is very expensive and costs around 110,000 for each patient, each year. Therefore careful management to prevent wastage is required. Aim.This project aims to find new ways to support and encourage the regular taking of this life-long treatment, as well as other regular medication. This will be done with the help of a group of people with CF. Project Outline The project will be carried out in four stages: I will review all the published research about how well people take medicines for CF. In particular, I will look at new ways to improve how well people take these medicines - and how effective these are. I will measure how well people take the new modulator medicines in adults attending two CF centres in England. I will also find out about the problems these people have using prescribed medicines -h; using something called the 'Living with Medicines Questionnaire'. I will ask around 40 people from the two CF centres to take part in an interview. I will ask them to bring along photographs they have taken to show how the new modulator treatment has affected their lives. These photographs will help to stimulate discussion at the interviews. I will then work with people with CF to devise new ways to support and encourage people with CF to take medicines , especially the new modulator medicinesPPI InvolvementPatients will be essential to the success of this project and will be involved in all stages. I will form a Research Panel of six people with CF and will meet regularly, every four to six months. The role of the panel will include: advising on how best to recruit people to take part reviewing any patient information during the project giving feedback on patient interview questions making sure we get a full picture of the patient point of view -h; reviewing information we get from the first three stages of the project helping to design new ways to support medicine taking in the final stage help with plans to share what we find DisseminationI will publish what I find in international high-quality research journals. I will also talk about what I find at regional, national and international conferences. I will also share with patients and the public through social media, written summaries such as a patient newsletter and presentations at patient meetings. Photographs from stage three of the research will be shown in an exhibition.

Research activity codes: 7.1 INDIVIDUAL CARE NEEDS
Health categories: CONGENITAL DISORDERS