Parental Experiences of CFTR Medication
Research type
Research Study
Full title
Parent’s experiences of their child starting Cystic Fibrosis transmembrane conductance regulator (CFTR) modulator therapy
IRAS ID
321325
Contact name
Hannah Edwards
Contact email
Sponsor organisation
University of East Anglia
Duration of Study in the UK
1 years, 2 months, 28 days
Research summary
Summary of Research
Cystic Fibrosis (CF) is a life limiting genetic condition, which affects approximately 10,800 people in the United Kingdom. People with CF struggle with a range of difficulties including reduced lung function and difficulties absorbing nutrients from food. Treatment for CF has traditionally been invasive and time consuming. Parents of children with CF quickly become experts in the care of their children and work closely with the multi-disciplinary CF team to support their child’s health and wellbeing. Parents of children with CF are known to experience high levels of stress as well as high levels of anxiety and depression.A new tablet-based treatment called Cystic Fibrosis transmembrane conductance regulator (CFTR) modulator therapy has been made available on the NHS. This treatment targets the cause of CF symptoms and although it is not a cure, is expected to revolutionise the lives of people with CF. Large research studies looking into how well CFTR modulator therapy works have already found it to improve the physical health of people with CF.
This qualitative study aims to explore what it is like for parents of a child with CF to start this medication. We hope that by focusing on the parents we can explore the impact of the medication on more than the physical health of the child and in doing so understand how they can be best supported.
Eight to 10 parents of children with CF will be invited to take part in interviews about their experiences. These interviews will be recorded and then transcribed so they can be analysed.
Analysis will focus on parent's journey to getting and starting this medication and how they made sense of this. The study’s findings will be written up in a report and used for an information booklet for other parents who are starting this same journey.
Summary of Results
Tentative Hope: An exploration of the experiences of parents of children with Cystic Fibrosis as they commence the new Cystic Fibrosis conductance regulator modulator therapies.Background
Cystic Fibrosis (CF) is a genetic disorder that affects approximately 11,000 people in the United Kingdom. CF is a life limiting condition and people with CF have traditionally had to undergo numerous and length treatments. Both people with CF and their familes are known to struggle with their wellbeing because of this.More recently a new type of medication called Cystic Fibrosis Conductance Regulator Modulator Therapies have been developed, which have been found to significantly improve the physical health of people with CF, without being as intrusive. The introduction of this medication is felt to be an important milestone in CF treatment.
What were the aims of this project?
This project aimed to explore what it was like for parents of a child with Cystic Fibrosis, for their child to start the new modulators: Kaftrio, Kalydeco, Symkevi or Orkambi.We wished to understand parents’ experiences of their child starting the modulators and how these were similar and different.
What we did?
We interviewed a total of ten different parents about their experiences. We asked a series of questions that had been chosen by the research team with the help of a parent of a child with Cystic Fibrosis. These interviews were recorded and transcribed.We then reviewed each of the interviews using a research approach called Interpretative Phenomenological Analysis (IPA). IPA is intensive approach, where researchers identify patterns in parents’ experiences. IPA is particularly interested in identifying patterns which represent how people make sense of their experiences. These patterns are summarised into themes.
What we found?
We identified five main themes, which contained nine smaller or subthemesExperience as Context (including impact of diagnosis, child’s relationship with CF and stories of other unknown people with CF ).
We found that that parents’ feelings about the medication were quite complicated. All parents spoke about the medication being important, particularly because of how devastating the diagnosis had been. For many the medication represented a new kind of future. However, for some parents this was complicated by worries they had for their child having to have additional procedures in order to have the medication, which could be upsetting for them.
Many parents spoke about hearing about the medication through social media. For some this had encouraged them to try the medication, whilst others found those stories differed wildly from their own experiences, as their children had not been as unwell.
Living alongside CF (including unconscious adjustment and conscious adjustment)
Nearly all parents talked about how important it was for their children to feel normal and live normal lives and that they made decisions not to make CF too big of a deal in their day to day lives. For some parents, starting the medication was like this, and it was just another day. Other parents celebrated starting the medication and it was more of a milestone.
All parents, however, found that life very quickly moved on and that day-to-day life became more of the focus than this medication.
Impact of the medication (including tangible and intangible) All parents spoke about feeling varying levels of relief after their child started the medication. Many felt that they could take their foot of the accelerator a bit.
Some parents talked about the relief they felt when their child was able to come off medications and how this freed up more of their headspace and time.
Many parents talked about how difficult they found it to know if things had changes for their children since starting the medication, as their children were changing all the time.
Special role of the CF team
All parents talked about how important the CF team was, and how special and unique their relationship with them was. They spoke about how accessible they were and how important this had been to them.
Socio-political context (including privilege and uncertainty of removal) Many parents spoke about feeling really lucky to have access to the medication, but that this could mean they felt pressured to make sure their children were taking it correctly. They were also aware of how much the medication costs, which added to that pressure.Many parents also spoke of the sadness and anger they felt at the announcement by NICE that they were re-considering whether they could continue to offer this medication on the NHS.
What does this suggest?
Overall, these themes suggest that although modulators are felt to be important, parents’ feelings about them are not straightforward. It found that these feelings were influenced by their experiences of their child’s CF, parents own coping strategies and the opportunity to reflect.
What we hope for the future?
This project showed that starting new life changing medication can bring up lots of different feelings for people and we think that it is important for people to be able to share this. We hope that more time can be given to parents to talk about this.This project also suggested that parents’ own wellbeing may be changing after starting this medication due to this sense of relief. We feel it would be important to explore this further to see if this is the case.
We are glad that NICE has decided to continue to recommend that modulators are offered on the NHS and hope that research such as this one offers a new view on why it is important.
REC name
HSC REC A
REC reference
23/NI/0020
Date of REC Opinion
16 Mar 2023
REC opinion
Further Information Favourable Opinion