Stuart Elborn

Session three: Natural history, clinical outcome measures and standards of care in paediatric diseases

How care site accreditations can improve outcomes – experience from Cystic Fibrosis

J Stuart Elborn - President, European Cystic Fibrosis Society and Director of NI Adult CF Centre

The assessment of disease outcomes in rare diseases is challenging. To deliver the highest quality care possible for people with rare diseases engagement in the processes required for optimal delivery of care needs full engagement from patients, families, clinicians, managers, patient organisations and medical and scientific societies. To achieve this It is firstly important that there are appropriate and agreed standards of care that can be delivered and that both process and specific outcomes can be measured to assess there impact. This nurtures a culture of excellence and provides levers to drive change.

Care delivery for cystic fibrosis has been a challenge since the disease was properly identified in the early 1950's. In developed countries specialist care cystic fibrosis centres rapidly developed and has been the predominant model of care delivery since then. In most countries CF care is provided by specialist clinicians working either as a single site or with networked clinics which relate to a central specialist service. The network model has been primarily developed in paediatric care while the care of adults with CF is usually at a single specialist centre. Standards of care have been developed at country and continental levels and provide clear guidance for optimal service provision, management and research. In most of the Standards of Care documents a minimum of 50 patients is required for centre designation although in some countries 100 patients is the number required for this status. It is the policy of most patient organisations that care for people with cystic fibrosis should be delivered by specialist centres with trained multi-disciplinary teams who have the majority of their time dedicated to cystic fibrosis medicine.

Many centres and subsequently countries and continents have developed databases and registries of people with cystic fibrosis which allows the collection of specific information on outcomes such as survival and important surrogates of survival such as lung function, infection status and body mass index. In addition these registries facilitate the collection of treatment approaches and behaviours of the CF centres. The registries are used at various levels to improve outcomes. CF centres use their registry data to audit their own care and at a national level, comparisons can be made between centres and practices associated with good outcomes can be identified. International data can also be used for comparison. This has been particularly important in identifying the challenges of delivering care in Eastern Europe.

In some countries a third part of the process of improving outcomes involves site visits and accreditation. Combining Standards of Care registry outcomes and site visits allows for the triangulation of the key factors required to ensure optimal care is being delivered to patients. Various models have been applied in USA. European countries and using this approach these have already demonstrated that it is possible to significantly improve outcomes by helping centres apply Standards of Care in an optimal way in their individual site.

The importance of ongoing audit, site visitation and accreditation and the continuous development of up to date Standards of Care are critically important in improving clinical outcome measures in rare diseases such as cystic fibrosis.

Stuart Elborn

22 Feb 2012