As part of my current role, supported by the generous scholarship from Arthritis Australia, I have been working on a number of research projects in paediatric rheumatology, aimed at both advancing our knowledge of diseases affecting paediatric patients and at improving patient care.
These projects include
- Patient satisfaction with a switch to a biosimilar medication. Biosimilar medications are usually cheaper than the original brand-name drug although they have slightly different chemical structures, due to the complexity of the large molecules involved, so we have to ensure they work as well as the original. This has been documented in adults, but to a much lesser extent in paediatric patients. My sponsorship by Arthritis Australia has allowed me to observe first-hand the switch by the UK’s National Health Service to treating uveitis (an eye disease that can cause blindness) with a biosimilar, making treatment cheaper. Our project, which is ongoing, is aiming to confirm that this drug works just as well as the original medication, and also to get patients’ feedback on this switch. We are specifically asking patients and their parents about any side effects, to make sure that they are not more of a problem in the case of the biosimilar drug.
- Working with the Global Musculoskeletal Taskforce on a submission to the World Health Organisation (WHO): Many children with arthritis and uveitis in the developing world have little access to treatments considered standard in Australia. This leaves them at increased risk of pain, deformity, growth issues, and blindness. As part of an international collaboration, I have helped with an application to get tocilizumab, a potent immunosuppressant used in the treatment of systemic Juvenile Idiopathic Arthritis (JIA) added to the WHO’s Essential Medicines List for Children
- Review of forthcoming treatments of JIA: my paper, recently accepted for publication by the Lancet Rheumatology journal, summarises worldwide research into the most important recent advances in care, and the likely advances in the next few years.
- Chronic Non-Infectious Osteomyelitis of the Mandible: this is a rare and painful condition where the jaw bones become inflamed. Due to its rarity it can be missed. I have recently had an article accepted to Lancet Rheumatology showing how to better recognize the condition to raise awareness among other doctors, leading to earlier diagnosis and better outcomes for patients.
- I have also helped recruit and assess patients for the following drug studies: ADJUST (Adalimumab in JIA Associated Uveitis Stopping Trial) and the trials for Baricitinib (JAK inhibitor) in uveitis and polyarticular JIA, a form of JIA which affects multiple joints.
As part of this scholarship, I wanted to improve our knowledge of the treatment of uveitis. I chose to use the scholarship working at Bristol Royal Hospital for Children for its expertise in this disease. Attending joint uveitis clinics with their consultant rheumatologist and ophthalmologist, I have learnt how to optimise joined-up care for these patients. In discussion with my supervisor, I chose to research how well biosimilars work and how the patients find them. I had suspected that Australian funders such as the Pharmaceutical Benefits Scheme would soon require patients to switch to the cheaper biosimilar drugs, and this is now happening, so my witnessing the UK experience will really help doctors, patients, and their parents as the changeover rolls out in Australia.
In working with the WHO taskforce, I aimed to improve the standards of care for children in developing countries who have JIA. Several medications used for children living in Australia, the UK, and other developed countries are expensive and so have limited availability worldwide contributing to childhood disability and pain. I have therefore searched the literature and done a cost analysis, together with members of a global taskforce, and submitted this to the WHO for consideration. If a medicine can be added to the WHO’s Model List of Essential Medicines for Children, it makes it easier for developing countries to prioritise cost-effective treatments. Increasing awareness of how many children suffer without these medications is also important to ensure that they will be added to the Essentials Medicines List. This document is currently under review with WHO with a response expected in mid 2021.
Although some of the research has been delayed due to the COVID-19 pandemic, to date it appears that the cheaper medication is just as effective at controlling the patient’s disease, as I had expected. However, several patients have reported that the new medication (which is administered by an injection once per fortnight, as diabetics do for insulin), is more painful to administer than the medication they were on before and so they want to switch back to the original medication. This is a big concern for parents who are administering injections to their children, often for many years. As this idea of biosimilar switching is very new to paediatrics, there is little information published about patients’ views on it, so I hope that completing this project in the coming months will improve our knowledge and awareness of the potential difficulties.
In my audit of Chronic Non-Infectious Osteomyelitis of the jaw, I described several novel factors and considerations in the diagnosis and management of this condition. For example, the disease is more likely to be isolated to a single bone, the mandible (jaw), and is more likely to be resistant to first-line treatments.
Although some of my projects are not fully completed or published yet, there are some early benefits for patients with musculoskeletal diseases. I presented my audit of chronic non-infectious osteomyelitis of the jaw to our local team meeting, which helped to raise awareness and discussion among team members of this unusual condition. My editorial on scoring systems for patients with systemic JIA has also been published in Rheumatology, Oxford Press and will hopefully be widely read.
As I am now analysing the data for the uveitis patients about the switch to biosimilars, I cannot comment in detail yet, but I intend to publish my recommendations to both the medical community and to patient organisations, so that both groups are ready for this upcoming change.
The WHO taskforce project, has the potential to have significant impacts on sufferers of musculoskeletal disease worldwide. In developing countries this could significantly change a child’s life, particularly if they can avoid significant disability such as limb deformities and chronic pain. It will enable them to lead a normal life, with the ability to pursue their chosen career and integrate normally in society. It will also have significant effects for the wider family and community in not having a disabled child to care for.
My review of current treatments available aims to support doctors worldwide in keeping up with advancing knowledge in JIA. With so many potential new medications it is important that staff know all the options for patients and avoid delay in giving the best treatment.
I have not yet completed my project on biosimilar switching in uveitis, and therefore I will aim to complete and publish this in the coming months. I intend to remain involved with clinical research through my links with the Bristol team, and to work collaboratively with them from Australia to improve knowledge and outcomes for patients in both countries.
I plan to continue my involvement with the ADJUST study in particular, which continues in Bristol and will be running at RCH Melbourne while I am in post there from February 2021.