What parallels exist between haemophilia and cystic fibrosis services?

There are many parallels between haemophilia and cystic fibrosis and the NHS cystic fibrosis service offers a model from which much can be learned. Haemnet sat down with Su Madge, Director of one of Europe’s largest cystic fibrosis centres to learn about how the service developed and what the future may bring.

 

Dr Susan Madge, Consultant Nurse and Director of the Adult Cystic Fibrosis Service, and Clinical Lead for Infection and Immunity, Royal Brompton Hospital, London

 

 

 

How did the NHS cystic fibrosis service evolve?

The CF service developed with improving clinical care, multidisciplinary teamwork, internationally shared knowledge and research, patient/parent representation in national clinical and research working parties/committees and an overall shared vision. As recently as the 1960s and 1970s, children with CF were dying at a young age from respiratory infection and malnutrition. Early and aggressive treatment of respiratory bacterial infection and the introduction of enteric coated pancreatic enzyme therapy slowly started to improve survival into the teenage years and early adulthood. Multidisciplinary team working was written into standards of care and patients/parents were actively involved in the creation of new CF centres, planning management, contributing to national documents and lobbying government on various issues.

Cystic fibrosis care became centralised in specialist paediatric centres with recognition that holistic, home-based care would also be important as treatment is complex, daily and lifelong. In the absence of NHS leadership, clinicians and the Cystic Fibrosis Trust (CFT) the patient advocacy charity, unilaterally designated Cystic Fibrosis Centres (CFCs) throughout the country. Establishing centres for adults was a more challenging task as more children were surviving into adulthood and outgrowing the paediatric centres.  Many of the new adult centres around the UK were opened because of the hard work of adults with CF who wanted to move out of paediatrics and not have to travel across the country to the few adult centres available.

 

How did the CF community help to bring about change?

When service expansion began, there were fewer CFCs and the CF clinical community was smaller. Clinicians working in CF knew one another and formed strong collaborative groups (often hosted and financed by the CFT) to describe and document guidelines for all aspects of care which would become the national expectation. Initially there was no official involvement but, presented with a fully functioning service, NHS England recognised the importance of developing a service specification based on these guidelines. This was timely because the cost of new treatments and increasing demand for resources were beginning to have a significant impact on hospital budgets.

The involvement of people with CF and their families in all aspects of CF continues to be very active. The CF community throughout the UK has always driven change. The CFT was founded by parents and clinicians in the early 1960’s, the template of clinical and parent/patient partnership persists today.

 

What is the Cystic Fibrosis Registry?

The UK CF Patient Registry was started in the early 1990s and now aims to include every person in the UK diagnosed with CF. It is hosted, though not owned, by the CFT and is run as an independent research project. Applications to use the data are made to a Steering Committee that includes parents, patients and clinicians and must go through an ethics approval process. Availability of data depends on patient consent. Pharmaceutical companies pay for pharmacovigilance studies. Income from these activities support the day-to-day management of the Registry. In line with CFCs throughout Europe, all national registry data are annually merged with the European CF Registry.

 

How are Cystic Fibrosis Centres funded?

Cystic fibrosis is an NHS nationally commission service. All patients on the registry have an annual severity banding score calculated based on their clinical data. Each band attracts a certain cost which is paid to the patient’s CFC.  Worsening disease status costs more therefore increases the allocated severity band.

NHS England recently approved new medication for groups of people with certain genetic mutations, this has the potential to significantly improve health. These drugs are expensive and their use may have implications for the way the service is financed: morbidity and survival could potentially affect the banding level. However, the impact on the current service model is uncertain. Experience with the new treatment is limited and, while there is no doubt that most people with CF accessing the medication feel better, the future remains uncertain.

Improvement in diagnostic technology means that increasing numbers of people are being diagnosed with CF as adults. These new diagnoses are at the less severe end of the CF spectrum, but people are still seen regularly by the CFC because their quality if life can be maintained with minimal intervention and problems can be identified and managed at an early stage. The CF community has ensured that NHS England understands the value of investing in early intervention in preventing more expensive complications later.

 

How did management guidelines come about?

The informal collaborative nature of the CF community led to the first management guidelines. In partnership with the CFT, groups made up of the various disciplines involved in CF care were formed to write and share guidelines on different aspects of care. Significantly, every group included adult patients and parents. Guidelines include clinical care, cross infection, physiotherapy, nursing, dietetics, psychology, school and work etc.

Until 2015, CFCs conducted peer reviews to assess adherence to the management guidelines. The review team were able to hold Trusts to account for their spending on CF services as the number of patients each centre looked after was public knowledge. Peer reviews were hosted and funded by the CFT, which also published the reports on its website. Audit is now the responsibility of NHS England’s Quality Surveillance Programme https://www.qst.england.nhs.uk

The UK guidelines have contributed to models for management guidance developed in Europe, Australia and New Zealand. The current NICE guideline is based on the CF management guidelines. The CF community was initially uncertain about the merits of having a NICE guideline, but it was felt that it could offer greater leverage to ensure the recommended services are delivered.

 

How effective are multidisciplinary teams?

Cystic fibrosis is a multisystem and complex disease therefore multidisciplinary working is successful in CFCs. All centres have a Director often, but not always, a consultant. Some centres rotate the Director’s post within the team and larger centres have Leads for the different therapies. Staffing levels have evolved informally in line with the management guidelines. Everyone in the team has an equal say, and every discipline involved recognises the strength in shared knowledge. Children and adults with CF see every member of the team for their review and their assessment is not complete without the input of each team member.

 

Summary

The parallels between haemophilia and CF, both in their epidemiology and the way NHS care is organised, suggests there is scope for shared learning between the two services. Cystic fibrosis care has benefited from the very active participation of patients, parents and the Cystic Fibrosis Trust, which has strengthened the effectiveness of clinicians and multidisciplinary teams. The UK Cystic Fibrosis Registry is an important asset that generates income and confers authority on the CF community.