Date published: 28 August, 2024
Date last updated: 5 September, 2024
NHS RightCare, Patient care

RightCare cystic fibrosis toolkit

Introduction

Cystic fibrosis is one of the most common, life-limiting, recessively inherited conditions in the UK affecting approximately 9,259 people in England, (11,148 people in UK) (Cystic Fibrosis Trust). Since 2007 new born screening for cystic fibrosis has routinely been undertaken in the UK, leading to early diagnosis in most cases.

Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that affects the production of the CFTR protein. If the CFTR protein is not made correctly, it affects the balance of salt and fluids inside and outside of the cell.

This imbalance leads to thick, sticky mucus in the lungs and digestive tract, which can cause breathing problems and increased risk of lung infections alongside gastro-intestinal and nutritional issues. Over time, the lungs may stop working properly.

People with the condition can also develop problems with their pancreas and other organs leading to several other related conditions, including diabetes mellitus, thin, weakened bones (osteoporosis), infertility in males, subfertility in females and liver problems.

Living with cystic fibrosis can also have significant psychological and social impacts, and health outcomes may be affected by poor emotional health and social adversity.

Purpose of this toolkit

This RightCare toolkit will help support systems to understand key priorities and ways of optimising cystic fibrosis care, by providing key actions for improvement.

It presents several priority areas across the cystic fibrosis pathway that highlight new and emerging areas of care, and where we know there is unwarranted variation that would benefit from further review and improvement.

The toolkit includes a self-assessment questionnaire which provides a method for assessment and benchmarking of current services.

This toolkit should be used in conjunction with the NHS England service specification and the Cystic Fibrosis Trust Standards of Care. It is primarily aimed at commissioners and providers of cystic fibrosis care.

The toolkit has been developed with reference to NICE guidelines. It is not intended to duplicate NICE guidelines or cover all aspects of cystic fibrosis care. Wider consultation has taken place with patient representatives, clinicians, professional bodies and other expert stakeholders.

How to use this toolkit

We recommend users first complete the accompanying self-assessment questionnaire, as this will highlight which priority areas you should explore based on your answers.

A Microsoft Word version of the questionnaire is available from the NHS England website, while an interactive version is available at the RightCare National FutureNHS site (please note that you will need to register as a FutureNHS member).

We do not intend systems or services to read this toolkit in full. We advise that you focus on the areas that the completed self-assessment questionnaire suggests would benefit from further investigation. Because of this, information/actions may be duplicated throughout the toolkit in multiple priority areas, where relevant.

For each priority area, there is a link to further guidance, references, tools and resources, and examples of good practice and case studies (where available).

Cystic fibrosis in the UK

66% of people with cystic fibrosis are in some form of education and/or employment

53% are male and 47% are female

  • 11,148 people in the UK have been diagnosed with cystic fibrosis
  • 63% of people with cystic fibrosis are aged 16 or over
  • by December 2022, 71% of people with diagnosed cystic fibrosis in the UK were on a CFTR modulator, compared to 10% in 2018
  • forced expiratory volume (FEV1) – the amount of air that a person can force out of their lungs in 1 second – is showing an increasing trend in both children and adults
  • parenthood: in 2022, 140 women with cystic fibrosis had a baby, compared to 58 in 2019; an increase of 141%
  • antibiotics: antibiotic use is decreasing. In 2022, 22.3% of people with cystic fibrosis had at least 1 course of intravenous antibiotics, compared to 44.5% in 2019

(Cystic Fibrosis Trust)

Vision of the future population

The care of people with cystic fibrosis is already changing for the better and will continue to improve following the introduction of CFTR modulators for many patients. People with cystic fibrosis born today are less likely to develop significant lung disease earlier in their lives than their predecessors.

Over the next 10 to 20 years it is expected that there will be a differential need for medical care for adults compared to children. Overall, the number of children with cystic fibrosis is not changing but due to increased life expectancy, there will be significantly more adults surviving with cystic fibrosis.

It is expected that overall, both children and adults with cystic fibrosis will need less hospital-based care but will require enhanced support at home using appropriate evidence-based clinical platforms without significant reduction in quality of care.

The cystic fibrosis multidisciplinary clinical team (MDT) includes physicians, nurses, physiotherapists, dietitians, pharmacists, psychologists and social workers.

They will remain integral to the care of people with cystic fibrosis to manage disease progression, drug therapies and the psychological and social impacts of the condition, but particularly those patients who cannot be prescribed CFTR modulators.

How may care change?

Advanced disease

By 2026 a very small number of children and approximately 20% of adults will have advanced disease. They will require high levels of support including inpatient treatment of exacerbations and may be on the lung transplant pathway.

Moderate disease

By 2026 a very small number of children and approximately 55% of adults will have moderate disease. They will require regular support from their hospital team including some inpatient treatment of exacerbations. A higher proportion of care will be delivered remotely.

Low disease

By 2026 most children and 25% of adults will be dependent on CFTR modulators to manage their disease, and so will need hospital team oversight of prescribing and monitoring of disease control. Much care will be delivered outside of hospital for this group.

Please note: this information assumes severity is calculated based upon lung function alone. In the future, the severity of illness may need to be viewed by other levels of organ support/failure such as liver and renal disease. Psychosocial needs are also currently under-represented in the severity model.

Future treatments and considerations

CFTR modulator therapy is currently not suitable for everyone due to their cystic fibrosis gene variant.

For some people, even if they are eligible, treatment may not be suitable for several reasons, such as significant interactions with other medications they are taking, side effects such as a change in mental health, or they have been advised not to take it due to physical health risks such as severe underlying liver disease.

As eligibility and new therapies become available it is important that teams keep abreast of developments in research and novel therapies.

It is important to remember that those who are eligible and respond well to CFTR modulators still have cystic fibrosis, and that clinical guidelines still need to be followed. Irrespective of whether someone is on CFTR modulators or not, cystic fibrosis care should continue as per NICE guidelines, with emphasis on patient-centred care and shared decision making.

People on CFTR modulators still need monitoring and support to remain well, and may need additional support, including mental health support, to help them adapt to their treatment.

Conversely, people who cannot take CFTR modulators – and their families and carers – may need specific psychosocial support to help them process and cope with the difficult emotions they may feel about this.

With recent marketing authorisations extending access criteria, 94% of all people with cystic fibrosis are now eligible for CFTR modulators.

This is an emerging picture, although we now have a better understanding of the impacts that CFTR modulators can have on people in the cystic fibrosis community. It is still a relatively new therapy, and more information will become available as our knowledge improves, particularly for younger age groups (Cystic Fibrosis Trust).

Risks of infection

People with cystic fibrosis who have been prescribed CFTR modulators – such as Orkambi, Symkevi, Kalydeco and Kaftrio – are still susceptible to lung infections and should be aware of and take precautions against cross-infection.

These drugs can reduce the frequency and severity of infections that would require hospitalisation, but there is currently no evidence to suggest that they can prevent them altogether.

There is still a significant risk of cross-infection between people with cystic fibrosis, and close contact should be avoided (Cystic Fibrosis Trust). 

System improvement priorities

As noted above, this toolkit has identified 6 areas across the cystic fibrosis pathway where system improvement can be undertaken.

These highlight new and emerging areas of care, and areas where we know there is unwarranted variation that would benefit from further review and improvement.

These are described below, along with key focus areas, recommended actions and appropriate resources and reference information.

Priority 1: Optimal management and personalised care

Co-ordination of care should be provided by a cystic fibrosis MDT based at a specialist cystic fibrosis centre.

Due to the risks associated with the condition, services should implement optimal patient-centred care that is flexible, accessible and available throughout the whole pathway.

By 2026, many adults and some children will still have moderate to advanced disease and will require regular and high level support from their cystic fibrosis care team.

Services need to forward plan to ensure inpatient care meets demand and the needs of their cystic fibrosis community.

Key areas of focus:

1.1 Supporting self-management (risk factors)

There are several risk factors that can prevent people with cystic fibrosis from effectively managing their condition. Systems and cystic fibrosis services need to ensure that they identify what these risks are and put in place actions to mitigate against them.

Different risks can affect different people and at different times. Risks may include:

  • lack of education and understanding about their condition
  • social determinants of health, such as housing and income
  • issues with emotional wellbeing and mental health
  • digital exclusion

System improvement priority 6 on health inequalities below gives further information regarding risk factors.

1. Ensure a risk management and identification approach is embedded within all aspects of cystic fibrosis services, considering a person’s specific needs and circumstances.

2. Services should provide regular reviews by specialist cystic fibrosis MDTs as part of routine care. These should be accessible by people with cystic fibrosis for education and support purposes, as well as clinical care.

The core team should include specialist doctors, nurses, physiotherapists, dietitians, pharmacists, social workers and psychologists.

Other MDT members such as youth workers to support adolescents and transition, and occupational therapists to support ageing people with cystic fibrosis, should be considered as appropriate.

3. Screening should be in place as part of routine cystic fibrosis care for additional support needs to identify any additional psychosocial needs associated with living with a chronic and progressive condition.

4. Ensure a clear transition process is in place, that has good communication between adult and paediatric cystic fibrosis teams, to enable smooth transition for the person with cystic fibrosis, and to highlight and mitigate any risk factors (see section 2.3 adolescents).

Further information

NICE guidelines:

  • NG43 – Transition from children’s to adults’ services for young people using health or social care services
  • QS140 – Transition from children’s to adults’ services

1.2 Outpatient care

Outpatient care needs to be accessible and available when needed.

Led by experts, services need to adapt and grow in line with an increasing and ageing population of people with cystic fibrosis and to consider different models, for example a mix of face-to-face and virtual consultations (hybrid model) as appropriate and as part of shared decision making conversations.

1. Outpatient care must be accessible for people with cystic fibrosis when needed and without delay. They should be seen by doctors and MDT staff with experience in cystic fibrosis.

2. People with cystic fibrosis should be reviewed regularly with a frequency appropriate to their individual needs. As health improves for those on CFTR modulators, frequency of appointments may be reduced, especially for those without pre-existing significant lung disease.

3. Infection control guidelines should be adhered to within outpatient care to prevent cross infection.

4. Secondary and primary care should work closely together and communicate effectively ensuring a joined-up approach to care for people with cystic fibrosis.

5. Commissioners and adult centres will need to plan for growth in line with increased numbers of people with cystic fibrosis, with regards to both capacity and resources; for example, additional outpatient capacity.

Plans for growth in numbers will need to consider the likely increased prevalence of age dependent cystic fibrosis and non-cystic fibrosis complications within the population.

6. Close relationships with non-cystic fibrosis clinical teams not previously accessed regularly will need to be built and maintained, for example elderly care, to provide continued quality care.

7. Trusts should provide the infrastructure and resources to provide virtual consultations as appropriate, including IT tools and virtual monitoring capability. This may help to manage an increased population size, but should not replace face-to-face care where it is more appropriate, dependent on clinical need, or preferred by individuals.

Further information

NICE guidelines:

  • NG78 – Cystic fibrosis: diagnosis and management
  • NG94 – Emergency and acute medical care in over 16s: service delivery and organisation
  • NG27 – Transition between inpatient hospital settings and community or care home settings for adults with social care needs
  • NG142 – End of life care for adults: service delivery
  • Health technology evaluation HTE13 – Virtual ward platform technologies for acute respiratory infections

NHS England resources:

Cystic Fibrosis Trust:

1.3 Enhanced treatment at home

Virtual consultations may relieve the stress of hospital appointments for adults and children with cystic fibrosis who have other complex needs, but this must not compromise the quality of care.

1. Services should implement a personalised care approach to virtual consultations and home monitoring as an alternative model of care, where deemed suitable and safe to do so depending on the person’s clinical need.

A shared decision-making approach is needed to ensure that this is appropriate for the individual. Factors such as adherence to treatment, digital access and confidence should also be considered.

2. Services should provide digital technology devices that measure key indicators such as lung function, blood oxygen levels, weight and temperature, and adherence to preventative inhaled therapy, to support the management and monitoring of their condition.

3. Services need to mitigate against digital exclusion. For example, by screening for challenges that technology may pose for those with limited electronic skills or low health literacy; those with limited English proficiency; and communities with no access to devices or poor internet connection.

(Further actions can be found in section 6.5, equity of access).

Further information

NHS England resources:

FutureNHS:

NICE:

1.4 Screening

Increased life expectancy is likely to increase co-morbidity risk in the older cystic fibrosis population.

Cystic fibrosis centres need to consider changes in associated risk screening, dependent on age and cystic fibrosis-specific risk factors, and adapt accordingly; for example, audiology screening, gastrointestinal cancer screening, cardiometabolic risk factor screening.

1. Genetic testing for partners should be provided as part of the fertility/pregnancy support for people with cystic fibrosis and can be requested via a number of specialities, as set out in the National Genomic Test Directory for R185 Cystic Fibrosis carrier testing.

2. Mitochondrial screening for risk of deafness with aminoglycosides should be offered to children following confirmation of diagnosis of cystic fibrosis. Older children and adults who have not been tested may be at risk, and screening should be considered.

3. Annual eye tests should be recommended to all children on CFTR modulators to detect any significant visual difficulties which may prompt referral to hospital eye services for further assessment.
Consideration should also be given to infants who have been exposed to CFTR modulators in utero or during lactation.

4. People with cystic fibrosis should be encouraged and informed to engage with other relevant healthcare providers and sectors, such as primary care, and for the older population to attend routine health checks which are not available in cystic fibrosis centres, such as primary health care cardiovascular screening and women’s health screening (cervical and breast cancer screening).

5. Cystic fibrosis annual assessments should include screening for mental health, emotional wellbeing and social factors.

6. Cystic fibrosis services need to have a robust process in place for annual screening of all patients by the cystic fibrosis psychologist.

With increasing numbers of people with cystic fibrosis, especially within adult services, it may be important to consider which groups may benefit from enhanced or more frequent screening by the psychologist.

For example: those diagnosed ‘late’, those not receiving modulator treatments, pre and post-natal mothers and fathers with cystic fibrosis, and neurodivergent individuals.

7. Due to a higher incidence of hearing impairment in people with cystic fibrosis and an ageing population of people with cystic fibrosis, audiology screening should be offered.

8. Dental health checks should be proactively encouraged for people with cystic fibrosis to reduce the risk of dental cavities and decay.

9. Technology should be used where appropriate to increase the opportunity for remote screening of cystic fibrosis related dysglycemia; that is, flash glucose monitoring with review from specialist teams (cystic fibrosis and diabetes teams).

10. Cystic fibrosis centres should consider changes in cystic fibrosis and non-cystic fibrosis risk factors around bone health, and amend annual reviews and screening appropriately, for example in peri- and post-menopausal women.

11. Due to an increased risk of colorectal cancer within an ageing population of people with cystic fibrosis, screening should be provided.

For colonoscopies, appropriate cystic fibrosis bowel preparation should be followed.

This should also be done as routine post-lung transplant. (See section 2.5 Lung transplantation and post-transplant care for more information)

12. Annual screening for musculoskeletal issues (including continence) is essential to address any issues and prevent unnecessary complications.

Further information

NICE:

European Cystic Fibrosis Society:

Cystic Fibrosis Trust:

Oxford Children’s Cystic Fibrosis Network:

NHS Greater Glasgow and Clyde:

Royal Brompton Hospital NHS Trust:

Journal articles:

1.5 CFTR modulator pathways

CFTR modulator therapies are designed to correct the malfunctioning protein made by the CFTR gene.

Because different mutations cause different defects in the protein, the medications that have been developed so far are effective only in people with specific cystic fibrosis mutations.

1. Services should follow NICE guideline NG78 for the management of all people with cystic fibrosis, irrespective of eligibility for CFTR modulators.

2. Services will need to adapt to the evolving challenges for those patients on CFTR modulators, including excess weight gain and the need for body composition measurements, and the greater need for obtaining lower respiratory tract samples from patients with reduced sputum load, such as use of induced sputum.

3. With an increasing number of adults and children on modulator therapies, care must be taken to avoid drug interactions from other prescribed medicines.

4. Where patients are not responding to CFTR modulators, they should be assessed to understand the cause, for example re-checking genotype, sweat test, adherence, concomitant medications, etc.

5. A person’s eligibility should be continuously/regularly assessed as this can change.

In addition, a person’s willingness or decision on whether to take CFTR modulators (or their ability to adhere to treatment) can also change and should be reassessed at least annually.

6. Services need to be aware of the potential for psychological/psychiatric/cognitive impacts of CFTR modulators and follow guidance to monitor any such impacts (see section 4.2 Psychosocial impacts of modulators for more information).

7. Services also need to be aware of the emotional health of those not taking modulators for any reason.

8. Annual eye tests should be recommended to all children on CFTR modulators to detect any significant visual difficulties which may prompt referral to hospital eye services for further assessment.

Consideration should also be given to infants who have been exposed to CFTR modulators in utero or during lactation.

9. If an expectant mother is taking Kaftrio during pregnancy and/or during lactation, services need to be aware that newborn screening may deliver false/negative result.

Further information

NICE guideline:

  • NG78 – Cystic fibrosis: diagnosis and management

Journal articles:

Cystic fibrosis inpatient services must be maintained. Although inpatient admissions will reduce for those on CFTR modulators, there will be 5-10% patients who are ineligible or cannot tolerate this treatment.

Admissions will also continue to be a vital aspect of management for people on modulators with pre-existing significant lung disease, and for other complications.

1. People with cystic fibrosis must be able to access inpatient care when needed, without delay.

2. Infection control guidelines should be adhered to within inpatient care to prevent cross infection.

3. People with cystic fibrosis should be looked after on a ward by doctors and MDT staff with experience in cystic fibrosis.

4. Due to reduced numbers of admissions since the introduction of modulators there is the risk of inpatient staff being less familiar with managing people with cystic fibrosis when admissions are needed.

Therefore, staff must remain up to date with relevant training in managing people with cystic fibrosis and maintain appropriate competencies.

Further information

NICE guidelines:

  • NG78 – Cystic fibrosis: diagnosis and management
  • NG94 – Emergency and acute medical care in over 16s: service delivery and organisation
  • QS61 – Infection prevention and control

NHS England:

  • Cystic fibrosis service specification – adults
  • Cystic fibrosis service specification – children

Cystic Fibrosis Trust:

1.7 Treating emergencies and exacerbations

Cystic fibrosis can cause a wide range of complications that vary depending on the genetic profile of that person’s particular cystic fibrosis mutation, age, and gender.

Almost everyone with cystic fibrosis experiences significant respiratory and gastrointestinal complications eventually, but the age when these begin, and the severity, can be quite different from person to person.

It is important that people can access care when experiencing an emergency or exacerbation due to the potential severity of the risks and complications. A personalised care plan that is held with the patient, GP and cystic fibrosis team may be helpful.

1. Cystic fibrosis services must be able to provide for urgent and specialist care needs and advice 24 hours a day, 7 days a week.

This will include management of emergencies such as haemoptysis, pneumothorax and bowel obstruction (including distal intestinal obstruction syndrome [DIOS]).

2. Telephone advice must be available, and clear contact numbers should be given to patients to enable them to obtain advice from the specialist team at any time.

During out of hours contact, a process must be in place to ensure a clear line of communication with a cystic fibrosis specialist.

3. Beds on a ward suitable for cystic fibrosis care must be available within 24 hours for an emergency admission, as well as capacity to ensure elective and non-urgent admissions can be managed appropriately when they have a respiratory exacerbation.

4. Provisions for emergencies and exacerbations need to be maintained for those not eligible or intolerant of CFTR modulators and those on CFTR modulators with pre-existing cystic fibrosis related lung disease or conditions.

Although emerging data suggests people on CFTR modulators will likely need less emergency care and have less exacerbations, they still need access to emergency and in-patient care and this should be factored into service planning.

5. Where intravenous antibiotic therapy is administered at home, the quality and standard of care should be equal to that of inpatient services with support from the cystic fibrosis MDT as appropriate.

Further information

NICE guideline:

  • NG94 – Emergency and acute medical care in over 16s: service delivery and organisation

NHS England:

  • Cystic fibrosis service specification – adults
  • Cystic fibrosis service specification – children

Cystic Fibrosis Trust:

1.8 End of life care

One part of end of life care is advance care planning. This involves thinking and talking about the person’s wishes for how they want to be cared for in the final months of their life. This can include treatments they may not want to have.

Planning ahead like this can help people document their wishes and feelings while they are still able to.

Involving family and carers ensures they know about the person’s wishes and could help them if they have to make decisions about their care.

1. Specific guidance on the management of advanced disease and palliative care – that is, complexity of care and co-morbidities – should be followed, and cystic fibrosis teams should work with other involved specialties as required.

2. Cystic fibrosis teams should work closely with specialist palliative care services in both hospital and community settings, including at home where possible and requested.

3. Services should recognise the increased emotional burden at this time and refer patients for psychosocial interventions and support where needed. Services should ensure workforce education is in place to support bereaved children and families.

4. Teams should be aware of the financial burden associated with end of life care on individuals and their families. They should screen for any financial stresses in end of life care and refer to specialist cystic fibrosis social workers where needed.

5. A standard model of end of life care should be followed such as the Gold Standards Framework for End of Life Care.

Further information

NICE guideline:

  • NG142 – End of life care for adults: service delivery

NHS England:

Cystic Fibrosis Trust:

The Gold Standards Framework

European Cystic Fibrosis Society:

All-Party Parliamentary Group for Terminal Illness:

Marie Curie:

Nuffield Trust:

UK Commission on Bereavement:

Journal articles:

EducateCF:

Priority 2: Supporting specific patient groups

Some people with cystic fibrosis may require additional or more specialised support. This could be due to the development of co-morbidities, a temporary change such as pregnancy or a patient that has additional needs.

Having timely and correct information and access to the additional support when needed will mean that their condition can continue to be managed safely.

Key areas of focus:

2.1 Late diagnosis in adults

While most cases of cystic fibrosis are now diagnosed through the newborn screening programme, this has only been in place nationally since 2007.

Therefore, there may be some people who remain undiagnosed until adulthood.

1. A sweat test and genotyping should be arranged for everyone with clinical features suggestive of cystic fibrosis (irrespective of newborn screening test result) and results interpreted in line with the latest diagnostic guidelines for cystic fibrosis and CFTR-related disorder.

2. Extended genetic testing should be made available and additional CFTR functional tests (such as nasal potential difference and intestinal current measurement) should be considered where sweat test and genotype results lead to an inconclusive diagnosis.

3. Where a diagnosis of cystic fibrosis is confirmed, every effort should be taken to establish the genotype, to ensure appropriate access to CFTR modulator medications.

4. If indicated, psychosocial support should be offered for the individual throughout the diagnostic process and following confirmation of the diagnosis.

5. Genetic counselling should be offered following diagnosis at any age of cystic fibrosis, cystic fibrosis screen positive, inconclusive diagnosis (CFSPID) or CFTR-related disorder.

6. Those with a confirmed diagnosis of CFTR-related disorder should be offered follow-up, ideally in a cystic fibrosis centre or a specialist clinic led by a cystic fibrosis physician, with other speciality follow-up as dictated by clinical symptoms, as per latest guidelines.

Further information

Journal articles:

2.2 Ageing population and retirement planning

Advances in the understanding of cystic fibrosis mean that there are more effective treatments, so people with the condition are living longer than ever.

However, the ageing process can bring its own challenges with additional cystic fibrosis complications and non-cystic fibrosis complications, for example bowel cancer, poor bone health, menopause, musculoskeletal issues, cardiovascular disease, chronic pain, etc.

As well as increasing clinical demand on services, this can also increase the emotional burden on the individual and their families.

1. Services need to ensure screening for age-related complications is made available and that people with cystic fibrosis and their families are made aware of the possible complications (see section 1.4 Screening for more information).

2. Members of the MDT should extend their scope to manage the evolving needs of the population (for example, non-medical prescribing). Additional clinical input by other non-cystic fibrosis specialist clinical teams may be required to care for a more complex ageing population.

3. Good, clear processes of communication should be embedded between the cystic fibrosis MDT and other teams involved in the care of people with cystic fibrosis to provide joined up, holistic care for the ageing population.

4. Cystic fibrosis physiotherapists will need to develop their musculoskeletal expertise or centres will need to provide ‘in-house’ musculoskeletal physiotherapy services.

5. Systems need to plan for new cystic fibrosis services that will be required for an ageing population, for example specialist cystic fibrosis occupational therapists, or build links with local services if there is no occupational therapist available within the service.

6. Systems need to plan for increased demand on social work and psychology support to help patients through the emotional burden of changing health status, increased life expectancy and associated impacts on their life plans such as education, employment, family planning, menopause, pension, retirement and benefits, including at diagnosis and transition.

Further information

NICE:

  • NG27 – Transition between inpatient hospital settings and community or care home settings for adults with social care needs
  • Mental health and wellbeing

Cystic Fibrosis Trust:

Journal article:

2.3 Adolescents

NHS England now considers the transition phase to extend to age 25, highlighting the need for recognition of possible ongoing needs for transition work in adult services.

Although processes for transition are well established, it is important to recognise that children and young people going through adolescence will experience many physical and emotional changes – also new friendships and routines will be formed (whether starting secondary school or university, or a new job and living independently).

This can make adherence to their treatment challenging. It can be a difficult time for both the individual and their families.

Additional support, including psychosocial support, may be needed to help them adjust and learn to make informed decisions around their care.

Adolescence and transition between paediatric and adult care

1. Services should have early and ongoing conversations with young people and their families to help them understand the changes and potential challenges that come with growing up having cystic fibrosis.

These conversations should be tailored to the young person’s developmental age and stage, and should include discussions on the importance of maintaining treatment and avoiding risk-taking behaviours such as smoking, vaping and excessive alcohol use.

Adult cystic fibrosis services need to ensure they continue any required work on transition after transfer to adult care.

2. Cystic fibrosis teams in both paediatric and adult services may need more education and skills in working with young people during adolescence, so that the needs of this age group are met.

This is especially true where complexity exists in terms of mental health, neurodiversity, or difficulties in managing cystic fibrosis care around sexual and reproductive health and future fertility/family planning/contraception.

3. Cystic fibrosis teams must ensure that any education/information about services and cystic fibrosis care is available in formats that children and young people will readily access and engage with, for example web-based formats that target areas of particular interest to this age group.

4. The membership of the cystic fibrosis MDT will need to evolve to meet the needs of the service.

Access to youth workers enhance the support provided to young people with cystic fibrosis in paediatric care and through transition to adult care and services should consider including them in the MDT, where appropriate.

Adolescence and mental health

5. Services need to forward plan for young people and families who need additional support at the stage of adolescence.
Increased psychosocial support may be needed to help families adjust to change and to help young people independently manage their condition.

6. Young people with cystic fibrosis and their family members who are 6-25 years old should be signposted to the Cystic Fibrosis Trust’s youth programme for additional support and peer events.

Adolescence and health management/adherence to treatments

7. During this period, services need to be aware that young people may have lower participation and adherence with treatments at home and remote monitoring activities.

Services should plan alternative models of care, as required, to mitigate the risk of poor adherence.

8. Cystic fibrosis services need to ensure that robust, multidisciplinary processes are in place locally to support adolescents specifically (and their caregivers) with adherence to treatment issues, in both paediatric and adult services and throughout the potentially difficult time of transition between services. (See section 5.2 Treatment burden).

9. Cystic fibrosis teams need to ensure that engagement is taken into account when considering use of digital technology/remote monitoring for this age group.

10. Currently, there are no universally agreed upon screening tools to facilitate the assessment of adolescent health-compromising behaviours including smoking and vaping, substance use, diet, physical activity, sleep, risky sexual activities, and healthcare seeking behaviours.

Services should consider creating and using their own screening tools locally to identify areas of concern and provide appropriate interventions such as referral to educational resources or local substance misuse, weight management or physical activity services. Information captured locally can be used to inform future service delivery.

Adolescents and future planning

11. Cystic fibrosis services should ensure that discussions around education, career and employment planning are embedded within MDT transitional care; and that adolescents and their families have access to support that provides information, advice, and guidance to achieve their future goals.

12. Services should provide workforce education to support health professionals in transitional care to address the vocational development of adolescents with cystic fibrosis.

They should also provide interventions to support and improve educational and employment outcomes and deliver flexible and optimal disease management care that minimises disruptions to education and work.

13. The cystic fibrosis specialist social worker should support young people to navigate their educational and career paths. This includes:

  • deciding when and how to reveal their health status
  • worries about facing discrimination
  • balancing their health needs with academic or work demands
  • dealing with symptoms or treatment in their place of education or work
  • managing interruptions in their education or employment caused by their illness

Further information

NICE guideline:

  • NG43 – Transition from children’s to adults’ services for young people using health or social care services

Cystic Fibrosis Trust:

(Formerly) NHS Digital:

Journal articles:

2.4 Fertility, pregnancy and parenting

Due to an increase in the variety of treatments, parenthood is now a more realistic prospect for people with cystic fibrosis.

Those considering parenthood may need education and support from the specialist cystic fibrosis MDT around the complex decision making process of planning a pregnancy, especially in those with poor lung function, challenging infections, and breastfeeding.

There may be a greater need for counselling around pregnancy and planning.

1. Education on male and female sexual and reproductive health should have started by the age of 10-12 and continue through transition and beyond, with signposting to contraception and education on outcomes for parents with cystic fibrosis.

2. Family planning counselling should be provided for patients and their families so they are fully informed about how health and parenting can intersect.

Adequate screening should take place to address any psychosocial concerns such as discussing cystic fibrosis care with their child and/or the need for increased support during periods of acute illness and/or as a parent’s health declines.

3. Family planning counselling should begin early enough to capture pre-conception optimisation discussions and plan appropriate maternal health and medicine network pathways.

That is: shared care with local outreach obstetric/maternity cystic fibrosis support or maternal medicine hub centre care, depending on risk factors and patient wishes regarding location of care setting.

4. Genetic screening for partners should be provided as part of the fertility and pregnancy support for people with cystic fibrosis, with a referral to genetic counsellors if required.

5. People with cystic fibrosis should be referred to appropriate departments for fertility testing and treatment.

6. Medication reviews should be carried out when women with cystic fibrosis are planning to conceive and during pregnancy, with adjustments being made as appropriate.

7. Centres should offer joint cystic fibrosis and obstetrics clinics or have systems in place to allow a coordinated approach from the obstetric and cystic fibrosis teams during pregnancy.

8. Services should be aware of the increased risk of gestational diabetes during pregnancy and manage care accordingly. Screening, timely diagnosis, and prompt treatment of diabetes are critical to reduce risks of maternal and foetal complications.

9. Pregnant women with cystic fibrosis should have access to the full cystic fibrosis MDT support around decision making and interventions for any potentially complex situations/complications.

10. There has been an increase in pregnancies for women on CFTR modulators;1 services need to plan accordingly for this and provide appropriate education and support, and consider adapting the service model for new parenthood as well with peer-to-peer support models and lactation/dietetic support during breastfeeding.

11. If an expectant mother is taking Kaftrio during pregnancy and/or during lactation, services need to be aware that newborn screening may deliver false/negative result, and that the babies should be tested for cataracts.

Further information

NICE:

Cystic Fibrosis Trust:

Journal articles:

2.5 Lung transplant and post-transplant care

There can come a point when standard cystic fibrosis treatments are no longer effective and people with cystic fibrosis can become very unwell, requiring more care and support.

This is the point when cystic fibrosis teams may consider whether referral for assessment for suitability for lung transplantation is appropriate and this is a treatment option that the individual wishes to explore.

1. Equity of access to specialist cystic fibrosis care needs to be ensured for management of non-transplant cystic fibrosis-related complications.

2. Systems must ensure that people have equity of access to specialist psychosocial professionals post-transplant.

3. The potential use of CFTR modulators should follow an informed discussion with patients with both cystic fibrosis and transplant teams.

4. Patients who have received a solid organ transplant must be given earlier screening for bowel cancer (see screening section). Screening should be provided ensuring appropriate cystic fibrosis bowel preparation is followed.

Further information

Cystic Fibrosis Trust:

Journal article:

2.6 Neurodiversity

There is an increased recognition of neurodiversity including autism spectrum conditions (NHS Digital: Mental Health of Children and Young People in England 2022; Russell G, et al, 2022), attention deficit hyperactivity disorder (ADHD), attention deficit disorder (ADD), dyslexia, dyscalculia and dyspraxia and their impact (including on mental health) in the general community.

Higher rates of ADHD (Cohen-Cymberknoh, et al, 2018; Georgiopoulos AM, et al, 2018; Lemiere & Havermans, 2018) have been observed in the cystic fibrosis population and it is important for teams to be aware of the potential for any form of neurodiversity and how it may impact on outcomes.

People with cystic fibrosis who are also neurodivergent may have additional care needs that may require supporting to help them manage their condition (Paul, Pasley, Krivchenia, et al., 2022; Pasley, et al, 2023).

1. Cystic fibrosis teams need to be alert to the possibility of undiagnosed neurodivergence and implement screening and referral for specialist assessment as appropriate.

2. Systems and services should offer continuous professional development for cystic fibrosis teams regarding neurodiversity and the impacts on cystic fibrosis management.

3. Systems and services should have clear pathways and processes in place to support people with cystic fibrosis who receive a diagnosis of neurodivergence.

The MDT should ensure that support is offered in managing their diagnosis alongside their cystic fibrosis and put in place any required reasonable adjustments.

Further information

NICE guidelines:

  • NG87 – Attention deficit hyperactivity disorder: diagnosis and management
  • CG128 – Autism spectrum disorder in under-19s: recognition, referral and diagnosis
  • CG142 – Autism spectrum disorder in adults: diagnosis and management
  • CG170 – Autism spectrum disorder in under-19s: support and management

NHS England:

(Formerly) NHS Digital:

Department of Health and Social Care:

Legislation.gov.uk:

Attention Deficit Disorder Association (adda):

Autism Research Centre:

Journal articles:

2.7 Learning disability

Over 1.2 million people in England have a learning disability and face significant health inequalities compared to the with the rest of the population (Mencap; Hosking, F., et al, 2016).

Despite suffering greater ill-health, people with a learning disability often experience poorer access to healthcare (Lenehan, C., 2017).

Cystic fibrosis services need to ensure that reasonable adjustments are made to help improve the health and wellbeing of patients with learning disabilities.

1. Services should review the (King’s College London) Learning Disability Mortality Review (LeDeR) Programme Report 2021 to understand mortality in people with learning disabilities relevant to cystic fibrosis.

2. Reasonable adjustments must be made according to individual patient need and information provided in an accessible and easy read format.

3. Services should ensure there is specialist learning disability knowledge to support reasonable adjustments and awareness of the risks to people with profound and multiple disabilities to ensure appropriate treatment plans are in place.

4. Ensure that the Learning Disability Standards are known and followed across the service.

5. Refer to NICE learning disabilities products for people with learning disabilities (guidance, advice, NICE Pathways and quality standards).

Education and training should be provided to ensure medications are taken appropriately and avoid the over medication of people with a learning disability, autism or both (STOMP), if appropriate.

6. Refer to NICE guideline NG96 – care and support of people growing older with learning disabilities.

Further information

NICE:

NHS England:

Kings College London:

  • Learning from lives and deaths – people with a learning disability and autistic people (LeDeR) – annual report (2021)

2.8 Diversity and inclusion

Diversity and inclusion is about recognising and valuing difference in its broadest sense.

Cystic fibrosis services need to ensure that their services are inclusive and adapt to the needs of all patients.

See also section 6. Health inequalities.

1. Staff must undertake equality and diversity training and be familiar with the NHS patient plan for equalities which encompasses equality frameworks and information standards.
These include the Accessible Information Standard, the Sexual Orientation Monitoring Standard and the NHS Equality Delivery System.

This will help staff develop their awareness of cultural and gender differences and how this might impact patient needs, communication and care.

2. Services should have an appointed equality, diversity and inclusion lead to drive work forward in this area and identify solutions.

3. Services should proactively understand the diversity within their population and the barriers which prevent people accessing care.

This should not be limited to ethnicity but include other aspects of diversity, such as gender identity.

4. Services must be regularly reviewed and adapted so that they adequately fit the needs of a diverse community.

5. All efforts should be made to allow for equitable and inclusive access to research and clinical trials for all patients with cystic fibrosis.

6. Educational materials must be available in different formats and languages as needed, and translators must be available as required.

Further information

NICE:

NHS England:

Cystic Fibrosis Trust:

Journal article:

Priority 3: Future demand considerations

Overall, the number of children with cystic fibrosis is not changing, but due to increased life expectancy, there will be significantly more adults surviving with cystic fibrosis.

Over the next 10 to 20 years, it is expected that there will be a differential need for medical care for adults compared to children.

The demand on cystic fibrosis services and the need for wider joined up multidisciplinary care with other services will increase.

Key areas of focus:

3.1 Adults living longer with cystic fibrosis

Advances in the understanding of cystic fibrosis and the development of new treatments mean that most people with cystic fibrosis are living longer, and so are now able to plan for the possibilities of further education, careers, family planning, retirement and beyond.

However, the increase in life expectancy is also likely to increase the risk of co-morbidity in the older cystic fibrosis population and local services will need to plan for this.

The plan for growth in numbers will need to consider the likely increased prevalence of age dependent cystic fibrosis, non-cystic fibrosis complications and co-morbidities within the population.

Virtual consultations and remote monitoring technology may support adult centres to manage an increased population size, but this should be used to enhance patient care not to deal with capacity.

1. Systems need to plan for growth in line with increased numbers of people with cystic fibrosis, with regards to capacity and resources; for example, specialist MDTs to provide the recommended level of care, including preventative approaches.

This will need to consider the likely increased prevalence of age dependent cystic fibrosis and non-cystic fibrosis complications within the population.

2. Cystic fibrosis centres and services should provide virtual consultations and remote monitoring technology as part of a personalised care approach.

This may help to manage an increased population size but should not replace face-to-face care (if more appropriate) and should consider both clinical need and preference of individuals.

3. Close relationships with non-cystic fibrosis clinical teams not previously accessed regularly will need to be built and maintained, for example elderly care, to provide continued quality care.

4. The membership of the cystic fibrosis MDT will need to continuously evolve to meet the needs of the changing and increasing population.

This may include extended scope roles including non-medical prescribing as well as additional and emerging roles, for example occupational therapists, youth workers or advanced care practitioners.

5. With the expectation of many people living longer due to CFTR modulators, there is currently increased need for psychosocial support with the shift to wellness and considering future opportunities.

More young people and adults with cystic fibrosis may need help with issues such as family planning, education, employment, pension advice, retirement and benefits.

6. Screening should be offered for age related complications (See also section 1.4 screening).

7. Paediatric and adult centres will need to continue to adapt the education and support provided to people with cystic fibrosis and their families, to reflect the change in expectations of both life expectancy and future complications associated with living longer.

Further information

NHS England:

3.2 Future models of care

While adults with cystic fibrosis are now living longer, the population size in paediatric centres is not likely to increase.

As CFTR modulators become available to younger age groups, health is likely to improve and there will be a shift from acute care to more preventative care.

Shared care models and associated funding arrangements are likely to need to evolve as care needs change.

The use of virtual consultations and community and home-based care may improve convenience to the person with cystic fibrosis, as well as reducing the risk of hospital based cross infection.

1. Systems need to plan for growth in-line with increased numbers of people with cystic fibrosis, with regards to capacity and resources; for example, a specialist MDT to provide the recommended level of care, including preventative approaches.

This will need to consider the likely increased prevalence of age dependent cystic fibrosis and non-cystic fibrosis complications within the population.

3. People with cystic fibrosis on CFTR modulators will likely need less emergency care and have fewer exacerbations.

However, provisions for emergencies and exacerbations need to be maintained for those not eligible or tolerant of CFTR modulators and those on CFTR modulators with pre-existing cystic fibrosis related lung disease or conditions.

3. Trusts should provide the infrastructure and resources to provide virtual consultations as appropriate, including IT tools and virtual monitoring capability.

This may help to manage an increased population size, but should not replace face-to-face care if more appropriate and dependent on clinical need or preferred by individuals.

4. Technology for remote monitoring can be used to optimise preventative care, make best use of financial resources and improve patient activation. Patient activation has been identified as important by the Kings Fund (The King’s Fund: Supporting people to manage their health An introduction to patient activation).

5. Systems should offer home-based (or virtual) care provided by the specialist cystic fibrosis MDT to improve convenience for people with cystic fibrosis and to reduce the risks of hospital based cross infection.

6. The cystic fibrosis MDT should be given the funding and permission to attend educational meetings and conferences to ensure opportunities to share best practice and ensure practice remains up to date.

Further information

NHS England:

The King’s Fund:

Journal article:

3.3 Feedback on quality of services

Centres are encouraged to regularly gather and consider feedback on quality of care/service.

The Cystic Fibrosis Trust undertakes national patient experience related surveys (patient reported experience measures [PREMs]) which provide cystic fibrosis centres with feedback on their service and opportunities for improvement, as well as identifying areas of best practice.

1. Services should encourage patients to engage with patient-reported outcome measures (PROMs) and PREMs to help healthcare providers, commissioners and other stakeholders make informed changes to their services and ensure an enhanced service.

2. Cystic fibrosis teams should have an active quality improvement audit programme that includes an assessment of quality of service.

It should use Cystic Fibrosis Trust PROMs, including benchmarking themselves against existing standards, for example clinical standards of care, dashboards and UK Cystic Fibrosis Registry data.

3. Patient involvement should be embedded in all stages of cystic fibrosis service development to support healthcare professionals to understand the needs of their community and provide a better service.

Services should liaise with the hospital trust patient, public and involvement lead for advice and support.

Further information

Cystic Fibrosis Trust:

3.4 Research and clinical trials

There is a need for ‘research ready’ clinical and healthcare staff and increased research capacity to operate in a modulator era, where the evidence base is young and there are significant gaps in knowledge.

The James Lind Alliance has published the refreshed top 10 research priorities for cystic fibrosis.

1. Centres should be supported to work closely with research networks:

  • Respiratory Clinical Research Network (CRN)
  • Clinical Trials Accelerator Platform (CTAP)
  • Clinical Trials Network (CTN)

3. Local cystic fibrosis teams should work with local research and development departments to facilitate and engage with research and registry-based trials.

3. Local cystic fibrosis teams should be enabled to receive consent from people with cystic fibrosis to input data UK Cystic Fibrosis Registry.

Further information

James Lind Alliance:

Cystic Fibrosis Trust:

Priority 4: Psychosocial wellbeing and mental health

Living with cystic fibrosis may impact on emotional wellbeing and mental health (The King’s Fund), and on social outcomes.

Similarly, poor emotional health and social adversity may impact negatively on health management and cystic fibrosis outcomes.

Integration of specialist psychosocial care into the work of the cystic fibrosis team is therefore essential. All people with cystic fibrosis (and caregivers) must have access to cystic fibrosis psychologists and social workers.

The MDT also needs access to consultation with cystic fibrosis psychosocial colleagues to ensure that care provided by the team is psychologically and socially informed.

Key areas of focus:

4.1 Emotional wellbeing and mental health

Rates of anxiety and depression have been shown to be 2 to 3 times higher in people with cystic fibrosis and their parents (Quittner AL, et al, 2014), with impacts on quality of life (Riekert KA, et al, 2007), physical health outcomes (Fidika A, et al, 2014; Schechter MS, et al, 2021) and healthcare use, as well as emotional wellbeing (Smith BA, et al, 2010).

Cystic fibrosis centres are seeing an increase in the demand for help in the areas of emotional health and social adversity.

This is likely due to national trends in mental health needs but also due to specific cystic fibrosis issues including impacts of COVID-19, treatment, modulators, and the changing needs of the ageing population.

The importance and benefits of joined up physical health and mental health care is recognised and recommended (Psychological Professions Network (PPN)).

1. Cystic fibrosis services need to have a robust process in place for annual screening of all patients by the cystic fibrosis psychologist.

With increasing numbers of people with cystic fibrosis, especially within adult services, it may be important to consider which groups may benefit from enhanced or more frequent screening by the psychologist.

For example, those diagnosed ‘late’, those not receiving modulator treatments, pre and post-natal mothers and fathers with cystic fibrosis, neurodiverse individuals, and those with advanced/end stage disease, including those being assessed for or waiting for a transplant.

2. Systems and services need to plan for an increased access to consultation with cystic fibrosis psychosocial professionals, so they can provide psychologically and socially informed care to increasing numbers of people in adult care.

3. Psychological therapies can be delivered to people with cystic fibrosis virtually (Bathgate, et al, 2022), but further evaluation of these is required.

Group interventions/education can now also be offered virtually to people with cystic fibrosis although barriers to access should be explored.

4. Access to liaison psychiatry services should be available to people with cystic fibrosis, as needed, preferably on both an inpatient and outpatient basis.

5. Cystic fibrosis psychosocial professionals should liaise with external mental health services as required, for example community mental health teams, child and adolescent mental health services (CAMHS) and eating disorder services.

6. The membership of the cystic fibrosis MDT will need to evolve to meet the needs of the service.

In addition to existing cystic fibrosis psychosocial professionals, access to youth workers may enhance the support provided to young people with cystic fibrosis in paediatric care and through transition to adult care.

7. Services should be aware that new psychological support and interventions may be required around ‘survivorship with cystic fibrosis’ (Sala, Marc A et al, 2022).

Further information

NICE:

Cystic Fibrosis Trust:

European Cystic Fibrosis Society:

Journal articles:

Kings Fund:

4.2 Psychosocial impacts of modulators

There is emerging data on the impact of new modulator treatments on emotional health and wellbeing, with some reports of negative impact on mental health and cognitive function (Bathgate, et al, 2022).

The Journal of Cystic Fibrosis has published guidance on monitoring psychological impacts of modulator treatments.

1. Cystic fibrosis teams need to be aware that they may need to provide monitoring, support and interventions in multiple new areas of cystic fibrosis care, including:

  • complex decision making for people with cystic fibrosis and their families, for example, whether to take modulator treatments
  • when people with cystic fibrosis have to stop taking modulators
  • new weight management issues
  • life adjustment, future planning (sometimes unexpected), for example career choices or family planning
  • indirect emotional impacts of modulators, for example changes to role, relationship to illness, existential issues in adjusting to wellness, etc
  • any apparent direct psychological, psychiatric or cognitive impacts of modulators, including a possible causal relationship between Kaftrio and depression and suicidality
  • emotional wellbeing/mental health/health optimisation of those not taking modulators
  • adherence to modulator treatments (and impact of taking modulators on adherence to conventional treatments)

Further information

Cystic Fibrosis Trust:

Journal articles:

4.3 Equity of access to cystic fibrosis psychology and social work

Everyone with cystic fibrosis should have access to specialist cystic fibrosis psychologists and social workers.

However, available data (UK PREMs survey – Children’s Cystic Fibrosis Services Report and UK PREMs survey – Adult Services Report) indicates inequity of access to such services.

1. Access to cystic fibrosis psychosocial support should be equitable and offered to all patients, as appropriate.

2. Services should engage with audit tools such as PROMs and PREMs to identify staffing gaps and equality, diversity and inclusion access that need addressing.

3. New guidelines for cystic fibrosis clinical psychology and social work services should be implemented and followed (to be published in 2023).

Social work practice interventions could be benchmarked and guided by NICE guideline NG216: Social work with adults experiencing complex needs.

Further information

NICE:

  • NG216 – Social work with adults experiencing complex needs

Cystic Fibrosis Trust:

4.4 NHS staff wellbeing

It is important for services and organisations to recognise the pressures on staff caring for a young, chronically ill patient population.

Organisations have a responsibility to ensure staff are supported and have a healthy work life balance.

Rapid changes in cystic fibrosis care also means that significant changes to working practices are required within teams.

Teams should be supported to adapt services and skills to adjust to significant change at such a time.

1. Services and organisations need to be aware of the responsibilities of the NHS and of managers/team leaders to support the mental health and wellbeing of their staff; and to facilitate access to any help and resources required by the cystic fibrosis MDT.

2. Psychosocial professional members of the cystic fibrosis MDT have a role to play to help achieve a healthy, functioning MDT, for example using reflective practice to work through any issues that arise.

3. Following significant difficult events (including deaths of their patients), the cystic fibrosis MDT could use facilitated peer-to-peer team support through ‘post event reflection’ (The Association of Clinical Psychologists UK).

NHS trusts may also provide opportunities for such reflection through local employee wellbeing services.

4. Services should allow and provide opportunities for the cystic fibrosis MDT to network with their professional counterparts for peer support, particularly where staff are the sole practitioner, as is often the case with cystic fibrosis social workers.

Further information

NHS England:

NHS Employers:

Kings Fund:

The Association of Clinical Psychologists UK Group:

Priority 5: Medicines optimisation

The aim of medicines optimisation is to help improve patient outcomes by ensuring that medicines are prescribed and taken correctly, thus avoiding the intake of unnecessary medicines, reducing wastage of medicines, and improving medicines safety.

It is about ensuring that the right patients get the right choice of medicine at the right time and encouraging patients to take ownership of their treatment.

The NICE guideline NG5 ‘Medicines optimisation: the safe and effective use of medicines to enable the best possible outcomes’ and related Quality Standard provide guidance on the safe and effective use of medicines in health and social care for people taking one or more medicines.

The pharmacist should be responsible for medicines management within the service and be accessible to the person with cystic fibrosis.

It is important to consider the psychological and social factors for people with cystic fibrosis and their families, including health and treatment beliefs and including perceived risks and benefits.

Because of this, a shared decision making approach with the patient is needed when it comes to changing medication, to ensure that all new dosing regimens, potential side effects and drug-to-drug interactions are discussed and fully understood.

Further information

NICE guidelines:

  • NG5 – Medicines optimisation: the safe and effective use of medicines to enable the best possible outcomes
  • NG78 – Cystic fibrosis: diagnosis and management
  • NG197 – Shared decision making
  • CG76 – Medicines adherence: involving patients in decision about prescribed medicines and supporting adherence

NICE quality standards:

  • QS120 – Medicines optimisation

NHS England:

Areas of focus:

5.1 Medication routine

Ensuring patients are confident in managing and following their medication regimen is important for the optimal management of cystic fibrosis.

There is no ‘one size fits all’ approach; the medication routine should be appropriate and tailored to the patient’s needs where possible.

1. Services should work in partnership with people with cystic fibrosis and their families to ensure that the medicines routine is suitable and appropriate for individual’s needs.

2. Services should implement NICE guideline NG78 – cystic fibrosis: diagnosis and management.

This states that specialist pharmacists should advise people with cystic fibrosis on medicines optimisation at outpatient clinic visits, during inpatient admissions, on discharge from hospital, and at annual review.

They should advise healthcare professionals on all aspects of medicines use and prescribing, and support GPs, community pharmacists and homecare providers to ensure that people with cystic fibrosis get the medicines they need without interruption.

3. In clinic when changes are made to medicines and treatment, one member of the team should take the lead to discuss the changes with the patient and agree how these will fit into their medicine taking routine.

4. With an increasing number of adults and children on modulator therapies, care must be taken to avoid drug interactions from other prescribed medicines.

5. Regular medication reviews should be standardised to ensure they are normalised, accepted and expected.

These reviews should include not just whether patients are taking their medicines as instructed, but that they are right for them.

6. A specialist cystic fibrosis pharmacist should undertake a comprehensive medicines review at least annually for all patients.

7. Ensure that ‘starting to stop’ conversations take place initially and at the subsequent clinic visit after a new treatment is added, to review how the patient is managing with it and consider stopping treatment if appropriate to do so.

8. Ensure inhaled medication technique is reviewed regularly. This should include equipment cleaning/sterilisation routines.

9. People with cystic fibrosis and their families should be educated and supported to use appropriate technology to monitor and manage their condition.

10. The psychosocial team should be involved either in direct work with patients or in consultation with the MDT where there are difficulties with medicine taking, as there may be significant psychological and social factors involved.

Further information

NHS England:

5.2 Treatment burden

Time, lack of routine, and number of medicines and treatments required are often cited as the biggest challenges for patients with cystic fibrosis.

New treatments are often added in isolation without consideration for how this will fit into the wider treatment regimen.

This creates an additional burden to patients and their families and can impact on the patient’s ability to follow their treatment regimen.

1. Regular medication reviews should be collaborative and incorporate shared decision making, where patients are supported to understand the medication and treatment options and are able to make decisions based on their individual needs and preferences.

Appropriate NICE guidelines (NG5 and NG197) should be followed.

2. Review of electronic medicine taking or delivery data (where possible) to promote open and realistic discussions about taking of medication, and to facilitate negotiated treatment plans and goal setting.

3. Where needed, implement regular standardised home visits by members of the cystic fibrosis MDT as appropriate, to allow a holistic review of patients in their living environment (looking at medications usage, equipment, techniques and sterilisation routine).

Any issues should be taken back to clinic and mitigated. Learning should be captured and shared.

4. Access to modern, faster nebulisers should be offered to everyone, where appropriate, to improve treatment times and reduce burden.

5. Access to equipment and digital technologies should be equitable and offered to all patients as appropriate.

Further information

NICE guidelines:

  • NG5 – Medicines optimisation: the safe and effective use of medicines to enable the best possible outcomes
  • NG197 – Shared decision making

Journal articles:

5.3 Medicine taking behaviour

The MDT should provide personal support to each patient to assist in finding the right medicine taking routine for them.

The routine should be reviewed if new medicines are started, or medicines are stopped.

1. A shared decision making approach should be adopted to support and encourage correct medicine taking behaviour.

Appropriate NICE guideline (NG197) should be followed.

2. The cystic fibrosis MDT should develop a consistent and co-ordinated approach to supporting adherence.

3. Educational materials must be available in different formats and languages as needed, and translators must be available as required.

4. When adding new treatment (or stopping treatment), this must be discussed and agreed with the patient.

A shared decision making approach should be used so that the patient understands the rationale, purpose and goals of the treatment, and how it will fit into their current medicine taking regimen.

5. Patients should be supported to develop good medication taking habits.

6. Discuss medicine taking habits and routines at routine clinic visits. One person from the MDT should lead on this for each patient.

7. Use data capture information where it is available to support discussions and decision making.

8. A member of the specialist pharmacy team should calculate the medication possession ratio (MPR) or proportion days covered (PDC) at annual review for medicines where delivery data is available.

Further information

NICE guidelines:

  • Shared decision making
  • NG197 – Shared decision making
  • CG76 – Medicines adherence: involving patients in decision about prescribed medicines and supporting adherence

Cystic Fibrosis Health Hub: CFHealthHub

5.4 Supply of medicines

Medicines supply can be complex as they can be received via several routes, including GPs, acute and community pharmacies and Homecare.

This can add unnecessary burden to patients and their families, causing delays and confusion which can impact on a patient’s ability to follow their medicine regimen.

1. Where possible, and in discussion with the pharmacist, patients should be given the choice of the best way for them to access and receive their medications.

Supply routes should be kept to a minimum to ease logistical and admin burden on patients.

2. Systems should have clearly defined medicines supply routes with communicated escalation processes for when there are issues, and emergency contact details to access support in a timely manner.

3. Communication between patients and care settings related to medicines and medicines changes should be robust and transparent (as discussed in NICE guideline NG5).

4. A dedicated contact method should be available (telephone, email, app etc) for health professionals to contact GP surgeries for requests to change and update prescriptions.

5. The process for capturing complaints and issues, and escalation of issues with Homecare should be clearly communicated with all services so they can report concerns and keep aware of outcomes.

Further information

NICE guideline:

  • NG5 – Medicines optimisation: the safe and effective use of medicines to enable the best possible outcomes

Royal Pharmaceutical Society:

Priority 6: Health inequalities

There is now a much clearer understanding of the role that social determinants of health and other social factors have on both health inequalities and health outcomes.

Social determinants of health are all the non-medical factors, such as the conditions in the places where a person lives, learns, works, and plays. These social conditions can impact on health risks and lead to health inequities and avoidable differences in health outcomes.

Those from more socially disadvantaged or deprived backgrounds have poorer physical and mental health outcomes than those from more advantaged backgrounds (The King’s Fund).

Social deprivation can be described as a lack of resources and opportunities, such as income, education, employment, and adequate housing, etc.

Several of these factors can co-exist and overlap, impacting negatively on the lives of individuals and communities, culminating in poverty. People with disability or chronic illness are more likely to:

  • live in poverty
  • have poorer education outcomes
  • work part-time or be unemployed
  • report a poorer quality of life compared with non-disabled people (House of Commons Library)

Nearly half of those in poverty in the UK in 2018 were from families in which someone had a disability, with some ethnic groups facing much higher rates of poverty than others (The Health Foundation).

Further information

There are a range of national strategies aiming to address some of these health social inequalities:

Areas of focus:

6.1 Social determinants of health

It is widely accepted that social determinants of health can be more important than healthcare or lifestyle choice in influencing health.

Also, having a systematic understanding of patients’ social risks to health may help guide both interventions to address these as well as decisions about medications and treatment.

However, despite this, data on patients’ social needs are not routinely documented in NHS settings (World Health Organization).

Poorer socioeconomic circumstances have been linked with worse outcomes in cystic fibrosis.

Compared with the least deprived areas, children with cystic fibrosis from the most deprived areas weighed less, were shorter, had a lower body mass index, were more likely to have chronic pseudomonas aeruginosa infection and have a lower FEV1 ratio (Taylor-Robinson, et al, 2018).

1. Workforce training and education should be implemented to understand how social factors can impact on health outcomes and their role in tackling these.

2. In the absence of a national consensus social risk screening tool, services should develop and implement their own social screening tools, and where need is indicated, refer to the specialist cystic fibrosis social worker for further support.

3. Specialist cystic fibrosis social workers are essential members of the cystic fibrosis MDT and have a crucial role to play in early identification of social risks and vulnerabilities.

3. They provide early interventions to mitigate these, such as liaising with education providers and employers to achieve better educational and career outcomes.

Both of which play key roles in improving social mobility, and connecting patients to information, advice and resources, including economic resources, for example, welfare benefits, charitable funds and specialist financial advice.

4. All individuals with cystic fibrosis and their families, where appropriate, should be made aware of the Cystic Fibrosis Trust’s grants scheme and other charitable grant schemes as well as local authority support.

5. Cystic fibrosis teams should consider indoor and outdoor environmental risks and triggers, and consider how to support the patient (such as signposting to council and housing services).

Further information

Office for Health Improvement and Disparities (OHID):

  • Health disparities and health inequalities: applying all our health guidance

Cystic Fibrosis Trust:

Department for International Development:

Journal articles:

6.2 Ethnicity

Ethnicity is important to consider, as people from an ethnic minority background are statistically more likely to live in socially deprived areas (Gov.uk).

Research suggests people with cystic fibrosis from Asian backgrounds in the UK also have worse health outcomes than white people with cystic fibrosis.

People from ethnic minority backgrounds also may have disproportionately decreased eligibility for CFTR modulators (Desai M, et al, 2022).

1. Cultural competency and awareness training should be available for staff.

2. Services should collect and monitor data about ethnicity in addition to social risk to help identify areas of inequality and improvement within the cystic fibrosis pathway.

3. Services should ensure that there is representation of patients from ethnic minority backgrounds in the development of cystic fibrosis pathways and services to ensure the requirements of patients are fully understood and met.

Further information

GOV.UK:

NHS Race & Health Observatory: website

Journal article:

6.3 Poverty

Disability and chronic illness leaves people financially vulnerable: half of all people who live in poverty live in a household where at least one person is living with a disability or long-term health condition (University of Bristol).

Recent research by the Cystic Fibrosis Trust showed a typical family with cystic fibrosis will lose £564 per month (or £6,786 per year) because of the condition.

This financial burden is made up of extra spending – including heating – essential but costly dietary needs, attending medical appointments, home medical equipment, and prescription charges, as well as loss of income.

The rise in the cost of living is placing additional financial pressures on disabled households where living costs are already typically higher.

The Cystic Fibrosis Trust’s research included a survey that found:

  • 85% of respondents were worried and stressed about money
  • in the last 12 months, 1 in 5 had run out of food before they had money to buy more
  • 1 in 3 borrowed money for basic essentials such as food, clothing, heating and internet
  • 1 in 5 people missed a hospital appointment due to cost in the last 12 months (Cystic Fibrosis Trust)

1. Ensure that services implement social risk screening to identify any issues that patients and their families have relating to food, fuel, digital and appliance insecurity, and access to transport; and where need has been identified, to refer to the specialist cystic fibrosis social worker for further support.

2. Individuals and their families should be signposted to the Cystic Fibrosis Trust for information on financial support, including welfare grants and benefits advice.

Further information

NICE:

  • NG214 – Integrated health and social care for people experiencing homelessness

Cystic Fibrosis Trust:

Journal articles:

The King’s Fund:

6.4 Digital poverty and exclusion

Digital poverty can affect people with cystic fibrosis, for example, not having a phone or device to access health apps or have virtual consultations with their health team.

Appliance poverty is a key issue too, for example, a working fridge is vital to keep and store medication at the correct temperature.

1. Services need to mitigate against digital exclusion; for example, by screening for challenges that technology may pose for those with limited electronic skills or low health literacy, those with limited English proficiency, and communities with no access to devices or poor internet connection.

Further information

NHS England:

6.5 Equity of access

COVID-19 has highlighted some of the health and wider inequalities that persist in our society, and how some groups and communities are more likely to experience difficulties accessing health care.

Some of the known barriers to accessing healthcare are geographical location, access to transportation and other circumstances, for example occupational flexibility, financial barriers, cultural and language barriers and poor levels of health literacy (a person’s ability to understand and use information to make decisions about their health).

1. Services should develop technology enabled care services (find out more on the NHS England website) to improve access to healthcare.

2. Workforce training and education on the role health literacy has had on health outcomes should be provided, and all patient information should be clear and accessible, for example, provided in a language and format that can be understood by those receiving it.

3. Services should ensure aspects of identity are not overlooked such as race, language spoken, gender, or sexual orientation, to ensure healthcare services are provided in line with cultural, social, communication and personal needs.

4. Services must screen for lack of resources that may impact on access to services, such as transport, digital poverty, or lack of childcare, and refer to the specialist social worker for support.

5. Services should identify those at greatest risk of poor health outcomes and those who are disengaged with services.

Targeted interventions and support to address and overcome barriers to care should be implemented to mitigate and reduce risks.

6. Remote monitoring technology for adherence monitoring can be used to optimise preventative care, make best use of financial resources and improve patient activation.

Patient activation has been identified as important by the Kings Fund.

Further information

King’s Fund:

National Institute for Health Research Evidence:

6.6 Health behaviours

The ability of individuals to lead healthy lives is influenced by the wider determinants of health and behaviours shaped by people’s opportunities and experiences.

Smoking, vaping, illicit drug use, poor diet, obesity, physical inactivity and higher alcohol consumption are all behaviours closely associated with markers of social and economic disadvantage.

1. Refer patients and their families (people they live with) to local stop smoking services if commissioned and available in your local area, for help to stop smoking or provide alternative methods of stop smoking support if no local services are available.

2. Ensure all staff involved in cystic fibrosis care are trained to give Very Brief Advice (VBA) on the risks of smoking and how to quit.

3. Ensure NICE guideline NG209 is embedded in systems where all pregnant, postnatal smokers and household members are referred to smoking cessation support.

4. Currently, there are no universally agreed upon screening tools to facilitate the assessment of adolescent health-compromising behaviours, including smoking and vaping, substance use, diet, physical activity, sleep, risky sexual activities, and healthcare seeking behaviours.

Services should consider creating and using their own screening tools locally to identify areas of concern and provide appropriate interventions, such as referral to educational resources or local substance misuse, weight management or physical activity services.

Information captured locally can be used to inform future service delivery.

5. Services should promote workforce education to understand the link between social determinants of health and health-compromising behaviours, and interventions to promote positive behaviour changes considering individuals’ social circumstances.

Further information

NICE:

6.7 Environmental risks

Indoor and outdoor environmental risks and factors can trigger and exacerbate symptoms for people with cystic fibrosis and impact wellbeing.

Environmental risks and factors include the presence of air pollution; poor living conditions and tobacco exposure that affect respiratory health.

1. Services to ensure they are aware of any environmental risks that could impact on health or wellbeing including but not limited to air pollution, poor living conditions and tobacco exposure, and to provide education to patients regarding these.

While it is impossible to fully negate these risks, teams should be able to advise patients on strategies to minimise these.

2. Services should consider indoor and outdoor environmental risks and triggers, and consider how to support the patient (such as signposting to council/housing services).

3. Where environmental risks relate to poor living conditions, the cystic fibrosis MDT should refer to the specialist cystic fibrosis social worker, who can liaise with the patient’s local community services; such as housing, environment health, social care and local childcare support services, where appropriate, to resolve issues.

4. Clinicians should ask about damp and mould during regular health checks, and direct patients to the Centre for Sustainable Energy (CSE).

5. Undertake the free e-learning module Helping People Living in Cold Homes, which supports health and social care professionals to put NICE Guideline NG6 (Excess winter deaths and illness and the health risks associated with cold homes) into practice.

6. Signpost patients who live in poor or cold housing to appropriate initiatives Help from your energy supplier: the Energy Company Obligation (via gov.uk website).

Further information

NICE:

  • NG6 – Excess winter deaths and illness and the health risks associated with cold homes

NHS England:

Cystic Fibrosis Trust:

World Health Organization:

GOV.UK:

University of Bristol, January 2023: The financial cost of cystic fibrosis

Asthma + Lung UK:

Centers for Disease Control and Prevention:

Medical News Today:

Centre for sustainable energy:

Key worker case studies

Clinical lead occupational therapist at Manchester Adult Cystic Fibrosis Centre (MACFC)

The role of a cystic fibrosis occupational therapist

The role of the cystic fibrosis occupational therapist at MACFC focuses on optimising functional wellbeing and self-management in adults with cystic fibrosis by supporting participation in everyday activities that are important to them.

Occupational therapy assessment uses a ‘whole person’ approach where physical, cognitive, mental health, social and environmental needs are considered to identify barriers and solutions to any difficulties people with cystic fibrosis encounter in everyday activities. Key areas of intervention include functional mobility and transfers, personal care, productivity (including education and employment), leisure and rest.

Key aims

The key aims of the occupational therapist is to provide advice and support to help people with cystic fibrosis adapt to everyday activities and environmental modifications at home, work, education or in the community; along with developing satisfying daily routines to support health and wellbeing.

They provide support to overcome cognitive difficulties impacting on daily activities and managing treatments and education and strategies for managing effects of fatigue. They encourage participation in virtual peer support groups, co-facilitated with the psychosocial team, on topics such as sleep and managing fatigue and provide advice on aids and equipment to facilitate participation.

They provide support during key life stages, for example transition to adult services, becoming a parent, and end of life planning and signposting and referral to local community services such as adaptation teams and housing. They also identify and address frailty in older patients.

Key benefits

The unique offer of occupational therapy within the cystic fibrosis team allows for more personalised care, focusing on all areas of patients’ lives and providing enhanced support to patients. Occupational therapists work successfully with multidisciplinary teams to improve patient outcomes and help them to ask better questions around function.

Health based youth worker (HBYW) at Southampton Children’s Hospital

The role of a health based youth worker

The aim of youth work is to enable young people to develop holistically, working with them to facilitate their personal, social and educational development, to enable them to develop their voice, influence and place in society and to reach their full potential.

Southampton currently have 7 HBYWs working across different health specialisms including cystic fibrosis, as well as offering access to a broad range of youth service activities to all young people living with a long-term health condition aged 11-25 years. This service is the PEEER Youth Service; reflecting patient empowerment, self-esteem, employability and resilience.

Youth workers work closely with the psychosocial team to bring the young person into focus and to ensure that their goals, holistic needs, and priorities are at the centre of their care.

Key aims

The key aims of the HBYWs is to provide support for young people living with cystic fibrosis, aged 11 to 25, to enable them to develop the cognitive, social, and emotional skills they need to thrive and reach their full potential.

The youth workers participate as a professional member of the healthcare team, contributing to the care of patients as part of the cystic fibrosis multidisciplinary team. They provide focused one-to-one youth support to young people identified as needing targeted intervention in line with care pathways. They also offer support during clinic appointments, hospital admissions and in the community in line with the national curriculum.

Acting as a social prescribing link worker they enable access to local community services for appropriate recreational, social, educational and advocacy support providing a varied programme of activities and opportunities to engage young people, delivered flexibly in partnership with the PEEER Youth Service team.

They contribute to the development of transition services, working alongside the adult team, ‘bridging the gap’ between paediatric and adult services, and acting as an advocate and source of support to young patients throughout their transition journey.

Key benefits

Young people are supported to grow their lives alongside their health needs, and empowered to explore ways to manage the impact of their cystic fibrosis when it becomes challenging. They are encouraged to think outside the box and find creative and individualised ways to absorb cystic fibrosis related routines into daily life so that they cause minimal impact on the wider priorities of being a young person.

Youth workers act as a non-biased keyworker to whom the young people, their parents and health care workers can refer to for guidance and advice and can provide information relating to psychosocial, educational/vocational issues and generic health issues such as substance misuse and sexual health.

A common outcome of this role is the improved engagement of young people within their clinics and overall health management. This supports the wider multidisciplinary team as an engaged young person who feels valued and respected is far more likely to be honest and share concerns and difficulties they are experiencing.

The role can provide improved screening and outcomes of functional related difficulties and offers exploration of areas such as motivation and enjoyment of life, leading to a positive impact for patients.

Acknowledgements

We would like to thank the following individuals and organisations for their support and input into the development of this RightCare toolkit:

  • Sally Basford, Paediatric Cystic Fibrosis Nurse Specialist, Royal United Hospital Bath NHS Foundation Trust and Vice Chair of the Cystic Fibrosis Nursing Association (CFNA)
  • Amanda Bevan, Divisional Pharmacist (Family Health), Southampton University Hospitals NHS Trust and Clinical Representative (CF) Specialised Respiratory CRG, NHS England
  • Kathy Blacker, National Programme of Care Senior Manager – Internal Medicine and Lead Commissioner – Specialised Respiratory CRG, NHS England
  • Marie Bolton, Specialist Paediatric Cystic Fibrosis Physiotherapist, Nottingham University Hospitals NHS Trust
  • Catherine Brown, Specialist Physiotherapist in Cystic Fibrosis, West Midlands Adult Cystic Fibrosis Centre and Chair of the Association of Chartered Physiotherapists in Cystic Fibrosis (ACPCF)
  • Fiona Dowdall, Cystic Fibrosis Social Worker, Manchester Adult Cystic Fibrosis Centre
  • Imogen Felton, Consultant in Respiratory Medicine and Adult Cystic Fibrosis, Royal Brompton Hospital
  • Professor Andrew M Jones, Consultant Physician, Centre Director Manchester Adult Cystic Fibrosis Centre and Chair of RightCare Cystic Fibrosis toolkit group, NHS England
  • Jacqueline Lowden, Clinical Specialist Paediatric Cystic Fibrosis Dietitian, Leeds Children’s Hospital
  • Angela McGowan, Consultant Respiratory Physician, University Hospitals of North Midlands NHS Trust
  • Helen Oxley, Consultant Clinical Psychologist, Manchester Adult Cystic Fibrosis Centre
  • Kevin W Southern, Professor of Child Health, University of Liverpool
  • Kerry-Lee Watson, Clinical Lead Cystic Fibrosis Specialist Dietitian, Kings College Hospital NHS Foundation Trust and Co-Chair of British Dietetic Association Cystic Fibrosis Specialist Group
  • Caroline Whitton, Lead Cystic Fibrosis Specialist Nurse, University Hospitals Plymouth NHS Trust and Chair of the Cystic Fibrosis Nursing Association (CFNA)
  • Thank you to members of Cystic Fibrosis Trust’s Community Involvement Group, who shared insights and expertise from their lived experience of cystic fibrosis.

Publication reference: PRN01325_i 

Date published: 28 August, 2024
Date last updated: 5 September, 2024