Dr Clarissa Pilkington, Consultant in Adolescent and Paediatric Rheumatology at Great Ormond Street Hospital (GOSH) sheds light on paediatric rheumatology and how treatment has improved over the years
Paediatric rheumatology is a vibrant and relatively new speciality with active research pushing forward new therapies and drugs. It now needs to grow to ensure equitable access to good standards of care across the UK. Paediatric rheumatology centres have developed in a geographically haphazard way, though many are based in paediatric centres. However, there are fundamental problems – there are already too many patients for these centres, the centres are underfunded and the shared care networks that they rely on lack proper organisation, let alone funding.
Specialist hospitals and paediatric care are both expensive: paediatric care in specialist hospitals is doubly so. Why is specialist paediatric care needed? Why not provide this care locally?
Any treatment provider will need staff with up to date specialist training. So, the staff need to be trained and then that trained personnel need to see sufficient patients to maintain the skills gained in this training. Local teams may support their staff to undergo extra training at specialist centres, but it is becoming increasingly difficult for local teams to support the study leave needed to maintain the training. For local teams, the rheumatology patients will be only one part of their job, and if they look after too few patients, they become frustrated by the difficulties in maintaining their skills. Staff turnover is an expensive problem. Undoubtedly better care is achieved for patients if they are known to the local team and that team actively looks after their welfare. However, patient numbers for each paediatric speciality are too small within District General Hospitals to amass sufficient expertise for general paediatricians and the allied health professional staff to look after speciality patients without close co-operation and back-up from the expert centres. The local teams rely on the expert centres multidisciplinary team of specialist nurses, physiotherapists, occupational therapists, psychologists and doctors to confirm the diagnosis and to initiate or change treatment plans.
It is the expert centres that undertake research that improves patient care and outcomes. As a new and growing area of medicine, these improvements are hard-won against the background of underfunding, oversubscribed services and continual reorganisation. Treatments within paediatric rheumatology have improved out of all recognition. Twenty years ago, patients succumbed to childhood arthritis and many ended up with severely damaged joints, wheelchair-bound and unable to lead independent lives. Thanks to specialist centres this is now a rarity for those who are treated. The benefit to society of preventing lifelong disability and dependence is not simply measured in the reduction in the cost of surgery for joint replacements or in hospital stays. We should be able to offer this type of service across the country and not just inconvenient pockets geographically lucky enough to be near a specialist centre.
One solution is to embrace the lifelong benefits of proper early intervention for all patients and improve the networks for the shared care of patients between specialist centres and General District Hospitals. This will involve establishing protocols; robust clinical governance and excellent communication through enhanced IT. The countrywide maintenance of patient care can only be addressed at an economically efficient level. The benefit of covering all these patients only is achieved if local teams work well with the specialist centres.
Researchers working on the underlying mechanisms of disease have produced molecules that can target specific points in the inflammatory pathway that results in arthritis. These newer biologic treatments have been far more effective, but they are costly. The drug trials in children often lag behind the adult trials. These diseases are much less frequently seen in children compared to adults, making it difficult to recruit enough numbers. The trials in children often end up being more difficult to do and more expensive to fund. Yet children are not little adults: the drugs are often cleared faster in children than in adults, necessitating higher relative doses. The effects on a growing skeleton and a maturing immune system can also differ: it is imperative that the trials are undertaken in children too.
At the moment, some charities and funding bodies are focussing on phase 1 and 2 trials: these trials focus on side effects and the pharmacokinetics of drugs rather than their effectiveness at treating diseases. Many parents are unhappy to put their children in these trials unless the adult trials have shown the drug to be effective. Once a drug has been approved and is being used to treat patients, data needs to be collected to determine what the effects are once the children have matured and are in adulthood. This means 30-40 years of follow up fora cohort of patients. Yet funding for registries or cohort studies is seen as mundane and is difficult to obtain. Funding is more often successful when linked to scientific research projects, which are often only for a few years. There have been funding calls for cohort studies, but these have not been for the 30-40 years time span that is needed.
Specialist paediatric hospital services are often undertaking the research that underpins the “cutting edge” treatments. These treatments are only available within the specialist centres until they become considered “standard”. At this stage, the expert centre will work with local teams to provide these standard treatments closer to home. Local teams will need advice on all aspects of treatment, especially when unexpected events occur. This advice is time-consuming and needs to be available at all times. This imposes a significant workload on expert centres. Funding to set up good networks of care, with good communication and support between expert centres and local teams are essential to ensure children have the care that medical improvements have made possible.
Dr Clarissa Pilkington
Consultant in Adolescent and Paediatric Rheumatology
University College Hospital and Great Ormond Street
Hospital (GOSH)
Clarissa.Pilkington@gosh.nhs.uk
