Conduct of the inquiry
On 2 December 2021, the Standing Committee on Health, Aged Care and Sport adopted an inquiry into childhood rheumatic diseases referred by the Minister for Health, the Hon Greg Hunt MP. The Committee was asked to focus on childhood rheumatic diseases and juvenile arthritis (referred to collectively as ‘childhood rheumatic diseases.’ A copy of the terms of reference can be found at page xi.
The Committee announced its inquiry via media release on 3 December 2021 and called for written submissions. The Committee invited submissions from a range of people and groups with an interest in childhood rheumatic diseases including peak bodies, federal and state government departments and agencies, universities, medical professionals and medical research organisations.
The inquiry received 127 submissions, which are listed at Appendix A.
The Committee held two public hearings – the first in Canberra on Thursday, 17 February 2022 and the second in Sydney on Tuesday, 8 March 2022. A list of witnesses and organisations who attended these public hearings is in Appendix B. Transcripts for all public hearings can be found on the Committee’s website.
Timeframe and interim report
The Committee appreciates the time taken by the childhood rheumatic disease community in providing the information in these submissions and hearings, which constitute a wealth of high-quality evidence addressing the terms of reference.
The Committee was committed to produce a report on this evidence prior to the dissolution of the House of Representatives for the 2022 Federal Election. Due to the short period of time between the receipt of this evidence and the expected date of that election, regrettably, a full inquiry report addressing all the terms of reference in depth has not been able to be produced.
However, the Committee acknowledges the significance of this health issue for Australian children and their families and thus has produced this interim report to provide an overview of the evidence and to make some key recommendations to begin the process of improving the understanding and treatment of childhood rheumatic diseases in Australia.
The Committee urges its successor committee in the 47th Parliament to re-adopt the terms of reference for this inquiry to give this important topic the consideration it deserves, including gathering further evidence if required and to produce a full report on childhood rheumatic diseases.
Overview of childhood rheumatic diseases
Prevalence and causes
Childhood rheumatic diseases are diseases that ‘affect joints, tendons, ligaments, bones, and muscles’ in children. They include inflammatory rheumatic diseases such as juvenile idiopathic arthritis (JIA), systemic lupus erythematosus (SLE), juvenile dermatomyositis (JDM), scleroderma, vasculitis, uveitis, and auto-inflammatory syndromes; and noninflammatory rheumatic diseases such as chronic widespread musculoskeletal pain, joint hypermobility, genetic collagenopathies and growth-related musculoskeletal conditions. This inquiry focused on the inflammatory diseases.
JIA is the most common inflammatory disease, affecting one to two per thousand children below 16 years of age. It occurs when a child’s immune system attacks his or her own joints, and sometimes other organs, particularly the eyes. More than 80 per cent of patients suffer pain on a daily basis, 85 per cent have a reduced ability to participate in school and sport, 50 per cent will continue to have arthritis into adulthood, and 20 per cent will enter adulthood disabled.
The exact cause of JIA is not known. Ms Victoria Allen, a parent of a child with a childhood rheumatic disease and Chair of the Juvenile Arthritis Foundation Australia (JAFA), provided a lay explanation of current understanding, in reference to her daughter’s disease:
Her immune system will think her ankle is a foreign body, and it will go and start attacking that tissue. It will attack either within the joints or in the tendon sheaths around it. If it attacks within the joints, that's what will cause the permanent disability. It's her immune system responding to a number of factors.
…. The idea at the moment is that this is the after-effects of a virus, with a genetic predisposition towards this disease—so it is likely my daughter had a virus which impacted her immune system, and it has never calmed down again. Any factors that go to the operation of your immune system can be relevant, like diet…or if you're tired or sleep deprived …. Anything that impacts the operation of her immune system may cause a flare-up, and we won't know…
As mentioned above, JIA often also results in complications in a child’s eyes, through an associated disease known as uveitis. This occurs in approximately one in four children with JIA. Without treatment this can lead to vision impairment or blindness, and about 10 per cent of children with uveitis lose vision each year.
While other inflammatory rheumatic diseases are rarer than JIA, they can have even more serious consequences, including death. Childhood SLE can involve the immune system attacking ‘almost any organ in the body’ and often affects the brain and kidneys, resulting in 10-20 per cent of patients dying within 10 years. It is believed to affect Indigenous Australian children at a rate up to 18 times higher than non-Indigenous Australian children, and also affects Polynesian children at high rates. JDM causes muscle and skin inflammation and can lead to difficulties with breathing and swallowing. It causes death in up to three per cent of cases.
Department of Health initiatives
In its submission to the inquiry the Australian Government’s Department of Health described its initiatives in relation to childhood rheumatic diseases. It noted that under the 2019 National Strategic Action Plan for Arthritis, which it described as a ‘blueprint to guide national efforts to improve the health-related quality of life for people living with arthritis, including children living with juvenile arthritis’,it has provided:
$1 million to Arthritis Australia for information, education and support activities from January 2020 to December 2023. This includes activity to expand the reach and coverage of …JIA kids’ camps and programs run by arthritis organisations to cater for more children, different age groups including young adults, and children with other rheumatic conditions.
$1 million to support consumer awareness and education activities from April 2020 to December 2023. This…includes the development and dissemination of consumer-focused care guides for people with JIA and their carers and families. It is anticipated that final versions of the guide …will be completed by April 2023.
$2 million to support activities relating to education and awareness for health practitioners from April 2020 to December 2023. This…includes development of a living guideline for JIA …. expected to be completed in 2023.
The Department further noted that on 14 October 2021, $20 million in funding opportunities from the Medical Research Future Fund were announced targeting chronic musculoskeletal conditions in children and adolescents. One of the three streams of research these grants will cover is ‘chronic autoimmune diseases that affect the musculoskeletal system such as JIA, juvenile [SLE] and juvenile scleroderma’. The Department also commented that ‘since 2000, the National Health and Medical Research Council has expended $12,200,868.29 towards childhood rheumatic diseases research.’
On the question of information available to GPs and other health professionals the Department explained the availability of ‘HealthPathways’. It submitted that:
HealthPathways is a commercial platform which can be accessed by a clinician and provides region-specific advice on assessment, condition management and local referral opportunities for a large number of conditions …. Each pathway provides clear and concise guidance for assessing and managing a patient with a particular symptom or condition. …
There are HealthPathways that currently exist that relate to childhood rheumatic diseases.
Finally, the Department commented that:
The AIHW’s [Australian Institute for Health and Welfare’s] national centre for monitoring chronic conditions is funded by the Australian Government Department of Health to monitor chronic conditions in Australia including chronic musculoskeletal conditions. The monitoring of chronic musculoskeletal conditions includes the routine monitoring of JIA.
Childhood rheumatic diseases are included in AIHW publications including the National Health Survey and the Australian Burden of Disease Study.
Experiences of patients and families
The Committee received numerous submissions from children suffering from childhood rheumatic diseases, adults who developed such diseases in childhood and patients’ families. They described in harrowing detail a host of impacts on patients and their families: for the children themselves these can include frequent pain, difficulty with everyday physical activities as simple moving around the house or tying shoelaces, difficult participating in sport or games, prolonged absences from school due to the pain and need to attend medical appointments, feelings of isolation from peers and schoolmates, and mental health challenges; parents face heavy financial burdens, the inability for many (particularly mothers) to work fulltime due to time needed to care for their children, difficulty caring for other children and high levels of stress; and many brothers and sisters become distressed by their sibling’s condition and/or resentful that their sibling is receiving what they see as special treatment.
The challenge faced by children with rheumatic diseases and their families is highlighted by the statistics on the impact of those diseases on their mental health: one study found that 93 per cent of children with JIA suffer from dysthymic disorder (a milder, but longer lasting form of depression); there are higher rates of depression and anxiety in family members of children with rheumatic diseases; one third of siblings of children with JIA required psychological support; and 30-40 per cent adults who developed JIA in childhood have symptoms of anxiety or depression.
The Committee was fortunate to have two JIA patients, Daman Cassar age 14 and Ms Greta Hazel-Harrison age 19, along with Daman’s mother Mrs Joanne Cassar, appear at its Sydney hearing. All three were forthright in describing their experiences with this disease and ways these could be improved. Daman explained the current state of his illness and treatment:
…the arthritis has spread to most of my joints, including my ankles, elbows, jaws, toes, wrists, thumbs, fingers, back and neck. It is most prevalent, however, in my thumbs and wrists, neck and back, and I've had enthesitis in my elbows recently. This causes me severe pain, which is treated through a variety of methods. To combat severe joint and muscle pain from arthritis I go to the physiotherapist and exercise physio. I also have to take tablets every week and every night, and I have a fortnightly injection of tocilizumab. When I have a joint flare-up, I have to get a joint injection to settle things down. I'm usually very sore after these for about four days, but then I feel better for about a year. This helps to keep my joints moving and functioning properly.
Ms Hazel-Harrison told the Committee about the pain she experiences as a result of her condition:
Juvenile arthritis is an often unheard-of disease, met with confusion by people who do not believe that such a disease even exists. Childhood rheumatic disorders are real, and the pain experienced as a result is certainly real too. Arthritic pain, for me, is unpredictable and intrusive. It often feels like a bomb of burning hot shrapnel has erupted in my joints, and I must walk around pretending that I have not got burning hot shards of metal tearing my joints apart. The pain can reach points where it's painful to sit on a mattress and there's no relief. It's an overwhelming, trapping and upsetting sort of pain.
Mrs Cassar commented on her and her husband’s experience when Daman was diagnosed at age four:
Obviously it was very difficult. It was quite a shock to find out that our son had arthritis. We were hoping at that stage that he just had arthritis in one knee. Was he going to be that child? Obviously it didn't play out that way. It was very difficult in the early days. We were originally seen through the Sydney Children's Hospital public clinic. The difficulty with that was that we would wait for hours and we had a four-year-old who was in pain and didn't want to be there, so it was very difficult in the early days. We ended up seeing the paediatric rheumatology team privately. Because the waiting took a lot of hours, and because of the stress on Daman, it was easier to go private. Obviously it's costly.
The submissions to the inquiry from patients and families are full of similar descriptions of the terrible impacts of these diseases. The Committee was deeply impressed, not only by their resilience in the face of such adversity, but by the determination of those impacted, including older children, to work towards better outcomes for themselves and for future sufferers.
This was typified by a submission from a 59 year old woman who was diagnosed with JIA aged 16, well before the development of the effective treatments that are now available, and too old for even the treatments of the time to work as they could have. As a result of her condition she has had ‘six hip replacements, two neck fusions, two elbow operations, two wrist fusions, replaced knuckles, foot & toe fusions’, and has been unable to have children. She concluded her submission by writing:
I still believe life is truly beautiful and most of my healthy friends are too busy to stop and notice. Being forced to stop in life I have had some advantages. I am very lucky I was born in Australia where our hospital system and surgeons are second to none.
My hope is that children today won’t have to suffer the life I’ve suffered.
The Committee fully shares her faith in the Australian health system, and her hope for the future of Australian children with rheumatic diseases. It trusts that the initial findings and recommendations of this inquiry, set out below, will take the first steps to making that hope a reality.
The most pressing concern identified across the evidence was the shortage of staff in Australia’s health system specialising in treating childhood rheumatic diseases. Dr Rebecca James, Consultant Paediatric Rheumatologist, Children's Health Queensland Rheumatology Service and member of the Australian Paediatric Rheumatology Group (APRG) told the Committee that:
You will hear many challenges discussed today, but fundamental to almost all of them is workforce. If we had investment into an appropriately resourced medical, nursing and allied health workforce, the other issues would, for the most part, fall into place. We could conduct research. We could provide outreach clinics to rural and remote areas. We could upskill community health providers in the recognition of paediatric rheumatic disease. We could participate in clinical trials. We could appropriately attend to children's mental and physical health and help them to achieve the same educational and professional milestones as their healthy peers.
Evidence from health professionals in South Australia (SA) and Victoria both highlighted how far their services’ numbers of specialists fall short of international standards. Yet those two states have the most specialists in Australia, in per capita and absolute terms respectively, and the situation in other states and the territories is even worse. This was demonstrated by a table provided by the APRG that is reproduced at Table 1.1.
Table 1.1: Australia's paediatric rheumatologist workforce equivalent fulltime (EFT)
New South Wales
12.6 per cent
49.9 per cent
24.2 per cent
61.9 per cent
52.9 per cent
0.0 per cent
0.0 per cent
Australian Capital Territory
0.0 per cent
31.6 per cent
Source: Australian Paediatric Rheumatology Group, Submission 70, p. 15
The Committee heard that even the current paediatric rheumatologist workforce is not being fully utilised in the public system, due to lack of funding. It was suggested that the current workforce could provide 15 EFT in the public system if fully funded. A notable example of this problem is Victoria, where there are seven paediatric rheumatologists but only the trainee (who is charitably funded) works fulltime in the public system.
It was unanimously agreed that the solution to this problem is to train and fund more specialists. At present there are only three training positions nationwide – in Brisbane, Melbourne and Perth – and only the Brisbane position has consistent public funding, while the others rely on charitable donations. A submission from six trainee specialists commented that:
A keen consideration…is the inadequate number of specialist training centres across Australia, and the tenuous nature of funded training positions in paediatric rheumatology. This has not only allowed for dire delays in combating the workforce shortage but also acts as a deterrent for future trainees. Some current trainees have had to wait years prior to being able to commence specialist training due to lack of accredited positions. All the trainees who will complete their training within the next 18 months have had to move either interstate or overseas (often both) to meet training requirements.
The trainees recommended that publicly funded positions be established at all currently accredited sites.
There was discussion of the establishment of more training sites, especially in New South Wales (NSW), which lost training accreditation in 2009. The problem is that a certain level of specialist staffing is required before a training site can be accredited. Dr James of the APRG put forward a potential response:
One possible model… is block funding over a 10-year period. If you funded all six tertiary paediatric rheumatology services across Australia with block funding for 10 years to have a trainee in each of those services—not necessarily a paediatric rheumatologist—you could significantly upskill the workforce. It's a self-fulling prophecy: the more paediatric rheumatologists you have, the more capacity you have for training…
Nurses and Allied Health Professionals
The evidence made clear that effective treatment requires not only paediatric rheumatologists, but also ‘multidisciplinary care teams’. The key members of such a team, in addition to the rheumatologist, were identified as a physiotherapist, occupational therapist and advanced practice nurse; other allied health professionals who may have an important role to play include psychologists, social workers, podiatrists, dieticians, pharmacists, osteopaths and orthotists. Administrative staff are also vital to allowing health professionals, particularly nurses, to focus on their clinical work.
There is apparently no true multidisciplinary care currently available in Australia. A model was trialled several years ago at the Royal Children’s Hospital, Melbourne, but did not receive ongoing funding. The numbers of professionals with relevant expertise are well below international standards; Australia has just eight specialist nurses, compared to 84 in the United Kingdom (four times more per capita), and in 2020 there were just 1.4 EFT specialist physiotherapists. Those nurses explained that while formal nursing qualifications can be somewhat tailored to paediatric rheumatology, ultimately they are trained on the job, which requires exposure to the field and enough specialist doctors to train them. As with doctors, there is the problem that trained nurses presently have no guarantee of getting specialist roles.
The Committee received evidence from paediatric rheumatology physiotherapists, as well as bodies representing osteopaths, psychologists and hospital pharmacists. Regrettably, due to time constraints, the Committee was unable to scrutinize the role these professionals play, although the common theme across theirs and many other submissions was the need for more funding for allied health support for childhood rheumatic disease. Patients themselves identified psychological support as particularly important. The only dedicated psychology support in paediatric rheumatology teams nationwide is a fractional position in Perth.
Ophthalmologists and their staff also have an important role to play in treating children with JIA, due to uveitis affecting almost 30 per cent of patients with JIA, which can lead to vision loss or blindness. Uveitis is asymptomatic in children, so all JIA patients must be screened for it as regularly as every three months; those who do develop the condition must then be treated. Professor John Grigg, representing the Royal Australian and New Zealand College of Ophthalmologists (RANZCO), explained how children who develop uveitis are treated:
What we've learnt is that initial acute treatment would be steroid therapy—prednisone and things like that. But that has its own side effects, so we've moved to these steroid-sparing agents …. So we only have six weeks of steroid therapy, and then we go into methotrexate and other biologics. But this then raises the point that ophthalmologists aren't as well trained in these immunotherapy things, so it's a real partnership with the rheumatologist.
Currently patients can only access a true multidisciplinary clinic in Sydney, with a uveitis specialist, paediatric ophthalmologist, paediatric rheumatologist and other staff. This clinic is ‘overwhelmed’ with patients, and the care is provided by the doctors unpaid, with the hospital’s costs covered by Medicare. Of the situation in the rest of the country, Dr Sophie Zagora, Member, Special Interest Group Uveitis, RANZCO, told the Committee:
The clinic we're running is probably the only one in the country that runs the way it does. We spoke to all our colleagues, when we put our submission in…and whilst some of the other states have started to implement it it's not on the scale of this.
There was general agreement that what is needed to make this clinic more sustainable, and to provide similar services to children across the rest of the country, is more funding.
Regional and rural patients
The Committee heard that there are particularly severe challenges in providing appropriate care to children with rheumatic diseases in regional and rural areas, but that these challenges are not insurmountable. The primary solution proposed was outreach clinics, where the ‘team physically goes and provides care and provides education locally.’ It was emphasised that these should be multidisciplinary, just like the city clinics discussed above; for example the Paediatric Rheumatology Team, Perth Children’s Hospital, submitted that:
We advocate for funding for the development of multidisciplinary outreach services that include the capacity for visiting allied health staff, particularly nursing and physiotherapy, to provide education and upskilling to local allied health providers on the provision of care to children with rheumatological diseases. The monetary and environmental cost of travel for medical and allied health staff to conduct 3-4 rural or remote clinics a year in each rural area of need (e.g. Bunbury, Broome, Kalgoorlie or Albany) would cost less than the sum of funds paid for every patient and accompanying family member to travel to Perth.
A key feature of this proposed solution is the idea of a ‘hub and spoke’ model. The basic idea of such a model is ‘linking primary and secondary health care to specialist tertiary centres.’ That is, the specialist centres are the hub, and more local health providers are the spokes. Dr Ben Whitehead of Children's Health Queensland Rheumatology Service and Chair of the APRG described it as:
…having local—whether they be medical or other—clinicians who have some experience and can be our eyes and ears on the ground where it might not be possible to have a paediatric rheumatologist in a town of 2,000 people.
The current and proposed operation of such a model in NSW, for example, is illustrated by figure 1.1.
Figure 1.1: The hub and spoke model in New South Wales
Source: Name withheld, Submission 64, p. 9
It was said of this model that:
The hub and spoke model of care will improve local access to the specialist rheumatology multidisciplinary team and foster a local service model for rural and regional children, young people and their families. Utilising telehealth technologies to support local teams and families. This model will engage local health professionals as part of the [multidisciplinary team], allow for upskilling of local practitioners and services allowing improved interval care.
Existing clinics and services
The Committee heard that some outreach services are already operating, albeit in limited form. Dr Navid Adib conducts multidisciplinary clinics in northern Queensland (Cairns, Townsville, Mackay, Rockhampton and Bundaberg) and in Toowoomba and the Sunshine and Gold Coasts but these are all private (with bulk billing where needed) except for one day in Townsville every three months. In NSW, Professor Davinder Singh-Grewal provides clinics in Wagga Wagga and Orange, although only with Commonwealth funding through the Rural Doctors Network and without any allied health support. Unfortunately this arrangement is of no help to people living in the north and south of the state, and the Australian Capital Territory (ACT).
The sole outreach in Victoria is one private clinic in Gippsland every three months; Victorian paediatric rheumatologists also previously provided clinics in Tasmania, but these were stopped due to the COVID pandemic and as of March 2022 have resumed. There are no outreach services at all in SA, although that state’s Paediatric Rheumatology Team submitted that with adequate funding and staffing they would ideally conduct such clinics in Port Augusta, Mount Gambier, Alice Springs and Darwin. There is some private outreach in Western Australia, although the details of this were unclear.
In addition to these outreach clinics, more permanent paediatric rheumatology services are provided in two non-capital cities: the Gold Coast and Newcastle. The service at the Gold Coast University Hospital was established in March 2019 using funds redirected from the Hospital’s General Paediatrics team, and consists of a paediatric rheumatologist one day per fortnight supported by that team, with no allied health or nursing support. Less detailed evidence was available concerning the service at the John Hunter Children’s Hospital, although it consists of a paediatric rheumatologist one day per month, with no specialist nursing or allied health support.
The experience of regional and rural patients
The inadequacy of the current network of outreach clinics was made clear by the evidence the Committee received of the care regional and rural patients are currently receiving. The mother of a boy with JIA in Mount Isa explained:
We currently have no specialist care in Mount Isa and frequently have to travel to Brisbane to seek care. This means time away from my husband and daughter (8yrs) and time off work for the both of us. Although we have very understanding workplaces this often is with short notice and ends up being 2-3 days at a time as we have to travel so far.
Numerous other regional and rural families from across the country wrote to the Committee of similarly difficult experiences.
The Paediatrics Department, Royal Hobart Hospital, submitted that:
Currently in Tasmania there are no paediatric rheumatologists. This means that children with rheumatological conditions are reliant on being managed by general paediatricians with the support of adult trained rheumatologists, or interstate paediatric rheumatologists, who may visit infrequently (previously twice a year), tele health or families needing to travel interstate to be seen.
Dr Mark Friswell of the Women’s and Children’s Hospital, Adelaide, told the Committee:
I saw someone yesterday from Broken Hill. They made a five-hour drive each way for a 20-minute appointment, and they have to do the same again to come and see the ophthalmologist two weeks later. It's ridiculous.
On the situation in northern Queensland, Dr Adib commented:
Again, it's not just Cairns …. it's the communities beyond. I have patients from Atherton or Innisfail—smaller places—but I don't know many people from further, like Aboriginal communities in the far north of Queensland. I'm not confident, if we're actually capturing those who do have primary care needs. I think, yes, as it was alluded to before, occasionally you see these kids with very severe arthritis; eventually someone sees them and sends them to the centre.
Education and awareness
The need for better education and awareness of childhood rheumatic disease, and JIA in particular, was an almost universal theme among submitters to the inquiry. There were calls for the better education of staff in the medical profession, the school system and the general community when it comes to recognising and diagnosing symptoms of childhood rheumatic disease and increased understanding of and support for those children and their families.
Ms Louise Hardy, National Policy and Government Relations Manager at Arthritis Australia told the Committee that:
Juvenile arthritis is poorly recognised by the community and by health professionals, but it is as common as juvenile diabetes. Children live with many debilitating symptoms, such as severe pain, which is often not well treated; fatigue, brain fog; mental health impacts; and sometimes permanent damage to joints, eyes and other organs. These symptoms affect kids' ability to participate in school, sports and social activities, and kids can face a real lack of empathy and understanding from teachers and their peers. We've heard stories of children being punished, excluded and bullied and told they must be faking and they don't look sick. If juvenile arthritis is not diagnosed early and treated properly, there's a risk of major and preventable illness and disability.
Musculoskeletal Australia (MSK) told the Committee:
More broadly, increasing community awareness of childhood rheumatic disease will not only improve community understanding, but may also increase knowledge and awareness among parents and families thereby resulting in improved time to diagnosis.
General practitioners, paediatricians and the wider medical profession
The call for greater awareness and training for General Practitioners (GPs), paediatricians and the broader medical profession came from childhood rheumatic disease advocacy groups, patients and their families, and from within the medical profession itself. While acknowledging that a GP cannot be expected to diagnose and treat every disease, there appeared to be general acceptance that some basic awareness training is needed for this cohort of medical professionals.
MSK was one of many who suggested to the Committee that a focus on improved knowledge and awareness of childhood rheumatic diseases is required for many health professionals, but GPs in particular:
General practitioners (GPs) are often the first ‘port of call’, however, many of them do not have the knowledge, awareness and experience with children with rheumatic diseases. This can often lead to conditions being undiagnosed for lengthy periods with this delay resulting in further complications and deterioration.
However, MSK also pointed out that there are knowledge gaps in the wider health system. It submitted:
Even when attending hospital emergency departments, parents of children with rarer rheumatic diseases report that doctors have little awareness of their conditions. Too many parents commented that they have heard too many doctors and nurses say to their child, ‘You’re too young to have arthritis’ or ‘That’s an old person’s disease’. …
Health professionals need to adopt a wholistic, patient-centred approach to the management of children with rheumatic diseases given these diseases impact so many aspects of children’s lives and the lives of their families.’
MSK stated in its submission that the significance of underdiagnosing or misdiagnosing these diseases cannot be underestimated for it leads to too great a physical, social, economic and psychological cost for children and their families.
Professor Mark Arnold, President of the NSW and ACT Branch of the Australian Rheumatology Association (ARA), who represents over 405 adult and paediatric rheumatologists submitted to the Committee that when it surveyed NSW families affected by these diseases, they:
…voiced their frustration at the lack of understanding of rheumatic disease by health care professionals, describing long delays in diagnosis from symptom onset and multiple visits to multiple health care professionals before a diagnosis and definitive treatment could be offered. This places children at significant risk and my paediatric colleagues have many patients in whom preventable delayed diagnosis has resulted in permanent musculoskeletal disability or visual impairment. Research shows that allied health practitioners also show low levels of comfort in managing children with paediatric rheumatic diseases.
Ms Francesca Tondi explained in her written submission to the Committee that her family’s journey with childhood rheumatic disease began in January 2018, when their son, Ollie, then 3 years of age, had a swollen knee. Ms Tondi describes attending at least 20 different appointments, including seeing a GP, a paediatrician and Ollie undergoing a CT scan under general anaesthetic. Nearly two years later there was still no diagnosis for Ollie, despite his pain and limping. On return to their native Italy, Ollie was diagnosed with JIA after one appointment with a paediatrician. Ms Tondi stated:
The doctor clinically observed his joints and was immediately able to recognise the disease. He then wrote a letter to our GP and Orthopaedic team at the Sunshine Coast University Hospital and we were referred to the Rheumatoid clinic at the Children Hospital in Brisbane.
Ms Tondi also believes there needs to be greater awareness and knowledge of the disease, and more training given to GPs and health practitioners. She submitted:
Too many children slip through the cracks and are diagnosed too late, leading to permanent disability. I think Ollie would have been one of them if we hadn’t been to Italy on holiday. We need to talk about it. People need to know about the symptoms as often it’s dismissed as “growing pains”. There should be more training for GPs and health practitioners.
Ms Allen had a similar story to Ms Tondi, and many other submitters. Ms Allen spoke of her family’s journey with the disease:
My GP didn't know what juvenile arthritis was. The only reason that we were able to get a diagnosis within four months for my then 14-month-old daughter was that we had a family friend who happened to be a GP and who had done her diploma in paediatrics. Throughout the past decade, I have constantly had to explain to everyone who has come into contact with my daughter, whether at school, sporting activities or birthdays or whether they're health professionals, what juvenile arthritis is. Nobody knows. Given that this is one of the most common chronic diseases, there should be far more awareness.
Dr Georgina Tiller, Consultant Rheumatologist at the Rheumatology Unit, Royal Children's Hospital in Melbourne heralded the call from within the medical profession, highlighting the need to better educate her health practitioner colleagues. She told the Committee that it is not for lack of interest, passion or expertise that there is not better awareness of these diseases:
I think the question is: how do we educate the other doctors around us, and on what level should we educate them? I think that we work very hard to do that at every level already, and it comes back to the lack of funded time in the public system. That means, as I think has been pointed out a number of times, that our time is vastly taken up with clinical work, which is 100 per cent our priority as clinicians. We want children to be walking and doing all of the things they need to be doing. So the time that we spend in the education sphere is actually in our own time. We run education sessions on every level…there are a number of GP education sessions that happen during the year, but they are usually at night-time, via Zoom.
We hold education sessions with general paediatric colleagues on a regular basis, and that would be both interdepartmental and across states. Then there's education at the other levels, which is educating allied health professionals as well as our nursing colleagues, who are absolutely critical in our team. One of the terms of reference is certainly related to education. But it's not just education of doctors and education of other subspecialists, which include the allied health subspecialists; it includes education of our partners, who are general practitioners and general paediatricians. I think it comes back to a lack of time, which comes back to a lack of funding. We are all extremely passionate. We engage in education activities all the time, but it comes back to the fact that we are also quite overwhelmed clinically.
Schools and the broader school community
The Committee received significant evidence about a large gap in awareness in schools about childhood rheumatic disease.
Ms Hardy from Arthritis Australia explained to the Committee that in a recent survey of their consumers about any potential recommendations for this inquiry, Arthritis Australia was told in no uncertain terms that families and children want, amongst other high priorities, ‘better education of health professionals, especially around early diagnosis [and] better community awareness, especially in schools.’
MSK submitted that ‘school teachers also need access to professional development modules to better understand the needs of these children when they are working with them.’ MSK spoke of the need for teachers to better understand the challenges children with a rheumatic disease face during their life, inside and outside of school, along with more education of all school children about the issues faced by children with chronic health condition.
Some evidence spoke of children and parents having to inform teachers and other school staff at the beginning of every new school year about the student’s condition and needs. Daman Cassar told the Committee that ‘it's just been quite hard to let everyone know every single year, because the message doesn't get passed down. The teachers haven't experienced it before.’ Ms Allen described the ‘trauma’ of her daughter starting a new school:
My daughter has just transitioned to high school, and I must have spoken to over 15 people at her school to explain what juvenile arthritis is. She hasn't been able to use her hands and she hasn't been able to walk this year already. Transitioning into a completely new high school, where there is no understanding of juvenile arthritis, is frankly traumatic for her and traumatic for me.
Daman’s mother, Mrs Cassar, raised a particular issue faced by NSW school children who suffer with JIA, that is, the requirement that they hand write their examinations for the Higher School Certificate (HSC) unless disability provisions are granted. Mrs Cassar explained to the Committee that the NSW Education Standards Authority (NESA):
…suggest the use of a scribe if children cannot write for an exam; however, they do not consider children with juvenile arthritis, who may experience jaw pain, discomfort and slurred speech and not be able to physically dictate their thoughts for a three-hour exam. We urge the committee to inquire with NESA into the granting of disability provisions for the HSC for children with childhood rheumatic disease in the small joints and hands, as NESA's current policies do not allow for our suffering children to be granted typing provisions for the HSC, and this in turn reduces their chances of being anywhere near equal to their peers.
Mrs Cassar explained that her son’s school have engaged a scribe to assist him with his assessment tasks previously, but that the difficulty remans in trying to ‘verbalise what you want to say and making sure that somebody actually writes that—and, when you use a scribe, you're supposed to dictate 'full stop', 'capital letter' and so forth, so it's quite a process.’
Daman Cassar explained to the Committee that while his school has been supportive of finding alternative technology to assist him to complete his schoolwork, he still faces significant challenges. He recounted his firsthand experience for the Committee:
Yes, [his school] has come up with multiple ways, like voice to text—they let me use that. But in exams and assessments tasks it's quite hard to use writing. Because of my wrists and my thumbs, I can't physically write as fast as everyone else does, and it causes a lot of pain .… a scribe is quite hard on my jaw, and also laptops are a bit hard to get.
When Dr Tiller of the Royal Children’s Hospital was asked about what more can be done for schools, she told the Committee:
I think there are good examples of models of care, of education, that happen at the grassroots level through school. That would particularly be in models of care of similar chronic disease prevalence, such as type 1 diabetes in childhood and allergy and anaphylaxis in childhood, where there are very well-established, systematic education processes that occur at the level of school. The lack of funding we have to produce educational resources has always been a limiting factor. It's not that we don't have the passion to be able to do it or that we don't have the expertise to do it.
Mrs Buffy Squires, Community Programs Coordinator, MSK told the Committee that over the recent years MSK has developed a suite of primary school resources and they are now calling for more funding to further develop resources for schools, and in particular, secondary schools. Mrs Squires stated:
… you've got to remember that kids spend most of their waking hours at school—or ideally they do when they're well enough to go to school—so we want the school to be on board. We need to develop resources that help increase community awareness for kids when they're out in the real world.
The Committee acknowledges the commitment of many GPs and the wider medical profession and the staff who work tirelessly in their school communities across the country who do have a good understanding of childhood rheumatic diseases. Despite the multiple pressures they face in their respective professions, the Committee is of the view that it is important to provide more support to enable medical professionals and school staff to have up-to-date resources they need to fill this knowledge gap and give them the confidence to know what to do when a patient or student presents with a childhood rheumatic disease.
Access to medication and treatments
The management of childhood rheumatic diseases includes both pharmacological and non-pharmacological treatments supervised and delivered through paediatric rheumatology specialists.
Modern treatment of most rheumatic diseases aims for complete remission of all symptoms, prevention of long-term damage to joints and internal organs and normal participation in school and sporting activities. To achieve this, children with rheumatic diseases need to have ‘the right drug, at the right time, with minimum delay’.
While Australia usually prides itself on having one of the greatest healthcare systems in the world, treatment for childhood rheumatic diseases is an exception.
Dr James of the APRG explained to the Committee:
Children with rheumatic disease in Australia do not have access to the same standard of care as their overseas peers. They do not have access to the same standard of care as other paediatric chronic illnesses in Australia such as diabetes or cystic fibrosis. They do not have access to medications that are international standard for treating conditions like arthritis, lupus and preventable forms of blindness. For children from rural, regional and, particularly, Indigenous backgrounds, the situation is even more dire.
Medications include non-steroidal anti-inflammatory drugs (NSAIDs), corticosteroids, disease-modifying anti-rheumatic drugs (DMARDs) - either synthetic oral DMARDs (usually methotrexate) and newer biologic DMARDs (bDMARDs) which are administered by injection or by intravenous infusion. Short-term simple analgesics can be used for pain relief as well as oral and intravenous antibiotics as required.
Recent advances in the treatment for childhood rheumatic diseases, particularly DMARDs and bDMARDs, have resulted in major improvements to short, medium and long-term outcomes. Early and aggressive treatment is essential to increase the likelihood of disease remission, reduce the risk of complications and long-term disability and improve quality of life.
The Committee received evidence from the Society of Hospital Pharmacists of Australia (SHPA) who raised the significance of hospital pharmacist led medication management. SHPA highlighted the complexity and importance of dispensing rheumatoid treatments accurately:
This is especially important for Disease Modifying Anti-Rheumatoid Drugs (DMARDs) such as methotrexate, where incorrect dose frequency can lead to hospitalisation and potentially fatal consequences. Over the last decade, there have been seven tragic and preventable deaths due to methotrexate overdose in Australia, with another 28 notifications to regulators on incidents involving methotrexate.
In addition, SHPA highlighted the important role that some hospitals and hospital pharmacy departments have in relation to early access to childhood rheumatic diseases treatment:
Public hospitals and hospital pharmacy departments play a crucial role in access to novel, usually high-cost and/or off-label medicines to treat complex and uncommon diseases before these medicines are registered on the Australian Register of Therapeutic Goods (ARTG) and well before they are listed on the PBS. They are also integral to patient access to clinical trials.
Physiotherapists and occupational therapists are essential in the treatment of childhood rheumatic diseases. In Australia, access to physiotherapists with training in paediatric rheumatology is limited especially in rural and regional areas. In the private sector, waiting times can be long and visits are often costly. Since physiotherapy and occupational therapists are an integral component of the multidisciplinary team, there is an urgent need to address this skill shortage.
Diagnosis of childhood rheumatic diseases has significant psychological effects on the child/adolescent and their family and can lead to emotional difficulties. Standards of care include initial and ongoing psychosocial support for children/adolescents with childhood rheumatic diseases and their families. An Australian study reported that parents and adolescents believed that psychological services were vital in addressing chronic pain, depression and family functioning.
Uveitis is a vision threatening inflammatory eye condition that affects some 20 per cent of children with oligoarticular arthritis and is the most common cause of blindness in children in the Western world. Eye drops, bDMARDs and frequent eye examinations are required to preserve vision and prevent blindness. Other potential complications include growth abnormalities and flexion contractures; infections as a result of immunosuppressive treatments; and other organ damage including heart and lungs.
Pharmaceutical Benefits Scheme medications
The APRG informed the Committee that for childhood rheumatic diseases ‘Pharmaceutical Benefits Scheme (PBS) restrictions on the prescribing of medications are a barrier to international best practice care.’
The APRG commented that:
… access to appropriate medications to treat paediatric rheumatic disease in Australia is a fundamental and major problem. The very limited number of PBS-listed drugs available in Australia or the treatment of paediatric rheumatic disease disregards many evidence-based treatment guidelines internationally, and puts Australia out of step with international best practice care.
Access to PBS-funded biologic drugs is also limited by regulatory hurdles. There is an enormous bureaucratic process, and considerable cost, to obtaining PBS-listing for a drug. Because the Australian market is in international terms very small, and the costs associated with the PBS approval process substantial, drug manufacturers are not incentivised to apply for PBS approval when a drug is not likely to be prescribed in large numbers across the Australian population. This means that a number of biologic drugs which are considered international standard of care are not available to children in Australia.
The RANZCO Special Interest Paediatric, Glaucoma and Uveitis Specialists recommended that ‘Adalimumab is PBS-listed for uveitis without joint involvement under the cotreatment of a paediatric rheumatologist.’
There are many examples in paediatric rheumatology where access to medications that are evidence based, and in many cases considered standard-of-care, are not easily accessible to patients as a result of restrictions placed on their supply through the PBS.
The APRG informed the Committee of the following important examples:
Low cost medications commonly used in paediatric rheumatic disease
Intra-articular steroid injections (‘cortisone injections’ or ‘joint injections’) are the first line of therapy for many children with arthritis. The most effective and long-acting steroid used for this purpose is Triamcinolone hexacetonide which is neither TGA-registered nor PBS listed.
Low dose methotrexate is a first-line treatment for most rheumatic diseases. However it causes distressing nausea in up to two thirds of patients. Evidence suggests subcutaneous (i.e. injectable) administration is more effective and causes less nausea, but this form is only PBS-listed for Rheumatoid Arthritis, a disease of adults. It is not listed for arthritis in children.
Ondansetron is a proven treatment for methotrexate-induced nausea, but is only PBS-funded when it is used to treat nausea from methotrexate use in cancer treatment, not nausea from methotrexate use in the treatment of rheumatic disease.
Biologics - High cost medications used increasingly in paediatric rheumatic disease
Biologic drugs have revolutionised the treatment of paediatric rheumatic disease over the past two decades. In JIA they are generally used when methotrexate or a similar drug is not successful in controlling disease.
Access to PBS-funded biologic drugs for patients with JIA is limited by criteria which were formulated almost two decades ago. They no longer reflect modern best practice and require urgent revision. Many of the criteria also make artificial and non-scientific distinctions between adult and paediatric disease processes, particularly in childhood uveitis and ankylosing spondylitis. This denies Australian children access to proven effective treatments. Without access to appropriate biologics, Australian children are forced to depend further on steroid medications which have profound and long term side effects such as cataracts, dramatic weight gain, diabetes, osteoporosis, growth retardation, cardiovascular disease and mood disorders.
Inefficiencies prescribing biologics
In addition, the APRG noted that the process to access biologics via the PBS is laborious and inefficient. It suggested that PBS processes are revised so that pharmaceutical companies applying for PBS listing for a biologic agent are mandated to submit appropriate applications for both adult and paediatric indications, and to revisit the application over time as new data in children emerges. The ARPG stated:
In order to prescribe a biologic, the doctor requires written approval from the Complex Drugs Programme (part of Human Services Medicare). The application forms are complicated, reflect a poor understanding of the disease processes and patterns and are time-consuming to complete and submit to Medicare. Clinicians await a response from Medicare (usually some 3-7 days afterwards) with an individual authority number which is manually entered onto the prescription. This original prescription is then physically forwarded by mail or picked up by the patient who then presents it to their pharmacy for supply to be obtained. This unnecessarily bureaucratic process is inefficient and wastes not only clinician time, but also patient time - and often leaves patients suffering for extended periods before they can commence or continue their therapy. This process is repeated every 3-6 months.
Many other PBS-funded medications can be approved immediately by phone or through a well established ‘streamlined authority’ process. The streamlined authority system requires a clinician to confirm that the patient meets defined criteria, thus maintaining governance over the prescribing of high-cost medications, without creating unnecessary inefficiencies, delays and wastage.
Dr James raised this concern regarding inefficiencies and commented ‘the administrative processes to prescribe current medications are laborious and inefficient. Precious time that could be used for care for patients is instead consumed by paperwork.’
JAFA raised some barriers to equitable access to some treatments. For example, eligibility criteria related to the arbitrary number of joints required to be affected before bDMARDS can be prescribed and the need to first fail methotrexate treatment. PBS rules restrict prescribing and access to bDMARDs to children with an active joint count of at least 20 swollen and tender joints or at least four major active joints. JAFA submitted that:
These restrictions directly impact children/adolescents with sacroiliitis as their main arthritis manifestation. Although bDMARDs are a proven and effective treatment for sacroiliitis, they cannot be PBS reimbursed if only two joints are affected.
The restriction on prescribing bDMARDS for uveitis even though their efficacy has been clearly documented, especially for Adalimumab which has revolutionised the treatment of arthritis-related uveitis which occurs most commonly when less than 4 joints are affected, excluding the very individuals who would benefit the most.
Dr James was concerned that some conditions such as ankylosing spondylitis, a condition in adults, have access to medication on the PBS called adalimumab. However, sacroiliitis, an analogous condition that children with childhood rheumatic disease present with, can't access adalimumab, even though it is in all international guidelines for treating that condition, as it is only available in Australia for over 18 years of age.
Dr James suggested one of the major challenges for Paediatric Rheumatologists is to get access to medications:
Processes exist within the TGA and the PBAC to get drug approval but those processes are almost, by definition, excluding of conditions like the ones that we treat. This is because, for financial or other barriers, it's not worth the drug company's while or there isn't the research available to the extent that is required in paediatrics.
Dr Whitehead of the APRG commented on the high cost of medications for childhood rheumatic diseases:
The common medications that we use, the biologic medications, are in the ballpark of $10,000 to $30,000 a year. One of the medications is in the ballpark of a couple of hundred thousand dollars a year, and that's a medication that's given once every two to three months as an injection. The medication that the patients use if they can't get access to that one is a painful injection every day, so we can do it every day or we can do it every three months. It's just money that makes the difference.
Professor Catherine Hill, President, ARA, emphasised that the issue of getting the right medication onto the PBS is not due to PBAC rejecting applications. Professor Hill stated that:
The issue of access to medicines is not because the PBAC is rejecting those; it's because the applications are not coming through—we have a fairly small market, obviously. There's also the administrative burden, and no paediatric rheumatologist, given the workforce shortages, should be having extra admin burden related to that.
Mrs Joanne Cassar, mother of Damon who has JIA made the following comment in relation to the impact of the lack of access to medication:
Having not met the PBS criteria for access to biologics early in Daman's disease journey, he unfortunately has painfully deformed thumbs, wrists and permanent damage shown in MRI scans. But we note that, interestingly, in three years and one month, when Daman turns 18 and is deemed an adult, he will suddenly and literally overnight become eligible for approximately a dozen more biologic medications on the PBS than he is currently eligible for.
The APRG discussed the following emerging treatments:
Janus Kinase Inhibitors (JAK inhibitors) are an oral DMARD approved for use in adults with rheumatoid arthritis and psoriatic arthritis. In Australia the JAK inhibitor Tofacitinib is PBS listed for treatment of adults with severe rheumatoid arthritis. In September 2020, the FDA approved Tofacitinib as the first JAK inhibitor for the treatment of polyarticular JA in children aged 2 years and older based on clinical trial benefits.
In August, 2021, the European Commission approved it for patients with JA and juvenile psoriatic arthritis. JAK inhibitors have the added appeal for children of being administered orally, obviating the need for painful injections. A recent large pivotal study confirmed the efficacy and safety of Tofacitinib in JIA patients aged 2-18 years recruited across 14 countries, including Australia (42). JAK inhibitors also have demonstrated benefits in children with JA related uveitis which does not respond to other therapies.
JAK inhibitors represent a further advance in the treatment of debilitating JA and should be fast tracked for approval for use in Australian children with JA. JAFA is concerned that the current protracted TGA/PBAC/PBS approval process will unduly deny access to Australian children for years.
Families of children with rheumatic disease experience a number of ‘out-of-pocket’ costs. These are cumulative and include loss of income due to regular attendance at medical appointments, increased childcare costs for siblings whilst the affected child is attending those appointments, allied health therapy (physiotherapy, psychology, occupational therapy and others), equipment and costs associated with pharmaceuticals that are not covered by the PBS.
For example, bDMARDs are very costly to families for a child requiring them, but they are ineligible for PBS reimbursement. Families are often left ‘hoping to obtain treatment on compassionate grounds or via a private prescription at a cost of $25,000 to $30,000 per year which is clearly unaffordable for the vast majority.’
Medication costs, even if accessed through the PBS, can impose a substantial additional burden on families. The out-of-pocket costs listed above are the same for most rheumatic diseases requiring immunosuppressive therapy, yet only children with JIA on immunosuppressive medication are currently eligible for a health care card.
The APRG informed the committee that ‘families are being forced to pay large out-of-pocket costs to see non-specialist therapists in the community, some of whom have no training or experience in paediatric disease whatsoever.’
The stress of caring for an unwell child also has economic and workforce implications: Russo and colleagues found that 75 per cent of working mothers of children with JIA changed their job or left it altogether because of their child’s disease. Out-of-pocket healthcare costs are substantial for families of children with rheumatic disease, and are compounded by inequities in health care card eligibility.
Ms Victoria Allen, Chair, JAFA, has cared for her young daughter diagnosed with JIA for over 10 years. The Committee asked her to comment on the cost of treatment for JIA. Ms Allen responded:
If you factor in lost income and the inability to work, particularly full time, given the job that I had, then I think the costs to our family have been extraordinary.
Ms Julie Jones, Paediatric Rheumatology Advanced Practice Nurse, Royal Children’s Hospital, Melbourne, told the Committee that part of her job involves helping patients and families to complete medical forms for Centrelink to try to access carers allowance.
National Disability Insurance Scheme
Access to the National Disability Insurance Scheme (NDIS) for children with rheumatic diseases is often unpredictable, inconsistent and sometimes vastly out of alignment with the child’s needs. Early intervention leads to better care outcomes and lower overall healthcare and societal costs.
With access to modern treatments - including physiotherapy and occupational therapy - many children with rheumatic diseases at high risk of poor outcomes should be able to avoid permanent disability. Most children with JIA and other rheumatic diseases are excluded from receiving therapy through the NDIS because they have not yet developed ‘permanent and significant disability’. Paradoxically, being unable to access appropriate therapies, they may go on to develop such disability - and thus are able to access the NDIS to treat the disability that timely therapy could have prevented.
As a result on not receiving appropriate treatment some children many children with rheumatic disease struggle to stay fit and active, struggle to keep up at school and struggle to access the therapy they need. This means that over the long term, they fall further and further behind their healthy peers.
Ms Hardy of Arthritis Australia, echoed this sentiment and called for ‘easier access to the NDIS’.
Mrs Squires from MSK made the following comment when asked about accessing the NDIS for her son, Sam:
Sam was diagnosed at 13. From what I understand, if kids are below seven it is easier. It is something to do with an early start program that they like to get kids on to. After seven it is more difficult; … because it isn't a guaranteed permanent disability…
Ms Allen commented that:
I know that there have been significant issues with people trying to apply for the NDIS, but I have not applied for the NDIS. I know that there are issues about whether or not it's a permanent disability and whether or not you can grow out of it… I certainly know that there have been real and significant issues with people trying to access care. It is clearly a disability that may only be through the juvenile period, but I can tell you that when my daughter has a flare she is disabled.
Professor Singh-Grewal, NSW Branch, ARA, raised concerns about workforce shortages for appropriately trained staff to support children with childhood rheumatic diseases. He stated:
My difficulty with the NDIS is finding allied health professionals that have adequate skills to feel comfortable treating children with rheumatic diseases. Again, this is published research that we did that surveyed allied health professionals and nurses nationally. It found that a vast majority—even those who considered themselves to practise predominantly in paediatrics—were uncomfortable with treating this patient group.
The Committee was interested to hear whether experts thought there should be Medicare item numbers for childhood rheumatic disease.
Professor Singh-Grewal suggested that no-one would say no to that and commented that ‘I think it's sensible to think of packages for chronic diseases with specific diagnoses, and juvenile arthritis is clearly one of those, yes.’
Ms Jones, a paediatric rheumatology advanced practice nurse, commented that ‘[w]e tend to go more down the path of seeing the GP and getting a physio care plan or a mental healthcare plan which gives them access to Medicare billed services. We do have many people who have applied for NDIS and have not been successful.’
Transition services from paediatric rheumatology to adult rheumatology
In 50 per cent of affected children, arthritis continues into adulthood accounting for tens of thousands of adults with severe disability. Adults whose arthritis began in childhood suffer an increased burden of both physical and mental health impacts compared with counterparts whose arthritis started in adulthood. They are therefore more dependent on health and social services and less likely to be in full employment thus adding to long-term macro-economic impacts through lost productivity.
Children are not just small adults nor are adolescents just large children. Each has their own physiological and emotional requirements dictated by their constantly developing bodies and brains which must be considered and balanced with all treatment(s) at all times. Thus, it is essential that providers of treatment and care of childhood rheumatic diseases have specific training in paediatrics as well as rheumatology.
Transitions services are generally considered an integral component of specialist rheumatological care providing structured and co-ordinated transition from paediatric to adult health services to ensure ongoing clinical monitoring and care. Currently in Australia, there is limited formal transition planning resulting in adolescents with childhood rheumatic diseases being more likely to stop their medication and drop out of the health care system, increasing the risk of disease flares and complications.
ARA drew the Committee’s attention to the absence of a well established transition process for paediatric patients moving into adult care - those over 18 years of age - despite this being acknowledged as global best practice. ARA stated ‘[t]his results in high rates of drop out of care and poorer outcomes for patients.’
The Paediatric Rheumatology team from SA stated that ’50 per cent of children with JIA continue to have arthritis as adults, 20 per cent with significant disability.
In addition, associated disability, employment difficulties and psychosocial issues need to be actively addressed in adults. This could be addressed by improved management of the transition from paediatric to adult health care services. There are agreed principles and models for transition of young people from paediatric to adult care using clinics staffed by paediatric and adult rheumatologists and rheumatology specialist nurses supported by transition services.
Associate Professor Ruth Colagiuri, Founder and Director, JAFA, informed the Committee that for many children with childhood rheumatic disease it progresses into adulthood. The outcome for this cohort is unfortunate as they are very badly off even compared with other adults with rheumatoid arthritis ‘in terms of disabilities, inability to work, mental health problems, dependence on pensions and all those very sad things—hip replacement, joint replacements. It is a very sad state of affairs.’
Mrs Squires from MSK commented that they ‘would like to see more support for adolescents at the time when they transition from paediatric to adult care.’
Professor Hill of ARA, highlighted the transition period from childhood to adulthood for people with rheumatic diseases as high risk:
Kids today have the opportunity to have much better treatments. We see adults who were diagnosed 15 or more years ago having some terrible deformities from not having access to care. This transition period is a really at-risk time for adolescents, when they're transitioning between paediatric and adult care. That's something we could do better, but, at the moment, because we have this paediatric workforce shortage, that takes extra time.
Professor John Griggs, Head of Specialty of Ophthalmology, Save Sight Institute, University of Sydney, suggested setting up a transition clinic for 18 to 25 year olds who may think they don’t need to see anyone:
I suppose we're invested in this cohort because we've known them for 10 or 12 years, so we will find somewhere to go to fit them in. That's a lot of phone calls and letter writing and things just to get them into an already overcrowded adult clinic, and that's the other challenge. That's why we're trying to set up a transition clinic for 18- to 25-year-olds who may think they don't need to go and see anybody; we all know what that age group's like. But they still need to do this. Otherwise they will lose vision at this time. If we had something that would cater for their psychosocial needs, that would really help that cohort as well. The only adult services where there's rheumatology or immunology and ophthalmologists who treat uveitis are treating an older group with that, rather than this transition group.
Access to treatments post 18 years of age
Access to treatment for conditions like uveitis is critically important. Paediatric Rheumatologists informed the Committee that since access for paediatrics is required but not available they must:
Patch things together using compassionate supply, patient funding and hospital funding, but that tends to be quite specific to paediatrics. But they hit a ceiling when they're 16, 17 or 18, and they go to adult hospitals where the bucket for compassionate funding of drugs is much smaller. So, often, patients fall off a cliff. They have paediatric hospitals fund their care, they transition to adult services and the adult hospitals refuse to pay for the medications that the child needs. There's no alternative, which is why it's so important to have medications available through standardised pathways like the PBS rather than relying on these ad hoc patch-overs that we've come up with.
Dr Zagora, RANZCO, also highlighted an unmet need for patients moving between paediatric and adult treatment for rheumatic diseases:
Obviously we have this paediatric group that we see, but, once they reach the age of 16 or 18, then we need somewhere for them to go. At the moment they're getting lost because the surrounding hospitals are all full and they often can't accept referrals. They're also that in-between age group, the 16- to 25- or 30-year-olds. They get misplaced, but they still have these complications that keep occurring and need, for example, the adalimumab, or Humira, that was being accessed via the hospital. They've lost their area of being able to have that access. They often get lost to treatment and follow-up, and then can turn up later with further complications of the disease that we'd managed so carefully through that period. There is this unmet need there.
The APRG submitted that:
Australian statistics for incidence and prevalence of paediatric rheumatic diseases are lacking, outdated or inaccurate. There is a particular paucity of data pertaining to these diseases in Indigenous Australians despite evidence suggesting a higher prevalence of certain rheumatic diseases, particularly SLE, in this population.
This lack of basic data is the result of inadequate research infrastructure including, but not limited to, disease registries. This limits Australian participation in international trials and access to cutting-edge treatments for Australian children. Disease registries also allow monitoring of long term side-effects of immunosuppressive agents, an important aspect of medication governance.
Without disease registries it is impossible to have a true understanding of how many children suffer from paediatric rheumatic disease in Australia, how their outcomes compare to children overseas, and whether or not the medications we use are leading to long term side effects such as cancer or infertility.
These views were echoed by many other submitters, who also called for one or more registries to enable better data collection. JAFA proposed that such a registry be funded by the Commonwealth Government and managed by itself and the Australian Paediatric Rheumatology Group.
Recognising the limited number of paediatric rheumatology specialists currently working in Australia, the Committee recommends that the Australian Government urgently establish an eight-year program of secure funding to provide fellowships or training programs with the aim of tripling the number of paediatric rheumatology specialists by 2030.
The Committee recommends that the Australian Government fund positions in the largest states of Australia, and in particular New South Wales, so that several accredited sites are available for training and there is less need to obtain training overseas.
The Committee recommends that state by state funding is better coordinated for paediatric rheumatologists in the public hospital system and to support the expansion of allied health for paediatric rheumatology including specialist nursing services.
The Committee recognises that a multi-disciplinary approach to treatment of childhood rheumatic disease is internationally regarded as best practice and therefore the Committee recommends the establishment of multi-disciplinary centres in major capitals through joint state and federal collaboration.
The Committee recommends that the Australian Government directs its funding mainly toward the public system so the centres of excellence can develop training of rheumatologists, ophthalmologists, physiotherapists, occupational therapists, psychologists and registrars in work as a multidisciplinary team.
The Committee recommends the Australian Government fund a ‘hub and spoke’ model with outreach clinics in outer metropolitan, rural and regional areas so that equitable care can be provided to all Australian children.
The Committee recommends that education in childhood rheumatic diseases of healthcare professionals should be provided as a priority so that diagnosis is not delayed but it is also important that public awareness is increased. Greater education about childhood rheumatic disease for General Practitioners, the wider medical profession, schools and the broader community to encourage early diagnosis and greater support for patients and their families in all aspects of their health journey. Such education programs should be delivered in partnership with experienced patient organisations like the Juvenile Arthritis Foundation Australia.
The Committee recommends that state education systems should be provided with support and information about best practice management of children with arthritis to facilitate optimal support services and IT support for children and adolescents living with rheumatic diseases.
The Committee recommends increased family support should be provided including National Disability Insurance Scheme and social security for those families who have a child diagnosed with childhood rheumatic disease.
The Committee recommends that as a priority state and/or federal funding be provided for:
outreach nurses as they are vital for best management of childhood rheumatic diseases across the country.
dedicated psychology services in all Australian paediatric rheumatology units.
establishing transition centres in public hospitals in all states for transitioning adolescents with arthritis/rheumatic diseases to adult rheumatology services. Further training for adult rheumatologists working in this area is required.
Recognising that new medications that are disease altering are increasingly available in other countries, the Committee recommends that the Pharmaceutical Benefits Scheme needs to proactively develop pathways to make sure rheumatic disease treatments become available to paediatric patients in Australia as a matter of priority.
The Committee recommends a national registry should be supported with adequate data collection and the ability to participate in clinical trials and research should be supported.
The Committee recommends state education departments approve the use of alternatives to handwritten and/or dictation methods for student patients who cannot use these methods, to support them to complete their end of school examinations based on their individual needs.
The Committee recommends the Australian Government’s Department of Health in partnership with the Pharmaceutical Benefits Advisory Committee, experts, patient groups and sponsoring companies conduct an urgent review into:
access to treatments for juvenile arthritis including access to drugs not currently available in Australia, and
limitations on access to existing listed medications that prevent patients receiving the most effective medications.
The Committee suggests that the successive Standing Committee on Health, Aged Care and Sport (or equivalent) in the 47th Parliament consider completing a full inquiry report into Childhood Rheumatic Diseases.