Patient Partnership in Juvenile idiopathic Arthritis (JIA) research

Juvenile idiopathic Arthritis (JIA) is a childhood autoimmune condition that causes pain, stiffness and swelling of the joints and other supporting structures of the body. It is thought to arise from a combination of genetic and environmental factors and affects about 3 in 1000 Canadian children.

One of the challenges in treating childhood arthritis is providing the right treatment at the right time. Although biologic therapies are frequently used and can be highly effective in reducing joint inflammation and pain, we are currently unable to accurately predict which children should start biologic therapies and which can discontinue treatment without having disease flares.

UCAN CAN-DU and UCAN CURE

Dr. Deborah Marshall

CIHR and Genome Canada funded research programs UCAN CAN-DU: Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Diseases and UCAN CURE: Precision Decisions for Childhood Arthritis, led by Drs. Susa Benseler and Rae Yeung, were created to address gaps in treatment approaches and support translational research for all children with juvenile arthritis.

As part of UCAN CAN-DU and CURE, Dr. Deborah Marshall leads the health economic research activity. This includes developing key performance indicators to:

• inform standardized measurement of the process of care,
• measure physician and patient and family preferences related to tapering of biologic medications, and
• model the cost-effectiveness of treatment strategies informed by individualized biological profiles.

This work was done in collaboration with a patient advisory committee that includes parents from Canada and the Netherlands, and Cassie+Friends (a Canadian patient advocacy organization dedicated to the pediatric rheumatic disease community) and led to advances in knowledge about the quality of life^{Footnote 1} and costs^{Footnote 2} in JIA, the complexity of disease-modifying anti-rheumatic drug treatment patterns in JIA in Canada, and hospital care^{Footnote 3} and medication^{Footnote 4} costs in the Dutch Health Care System.

JIA Patient and Community Engagement Research (PaCER)

With the support of UCAN and partnership with Cassie+Friends, Dr. Marshall received funding from the Alberta Children's Hospital Foundation to fund the JIA Patient and Community Engagement Research (PaCER) cohort to train a group of youth with lived experience of JIA in patient-engaged research.

The JIA-PaCER patient partners brought their lived experience to conducting research on the transition from pediatric to adult care in rheumatology. This patient-led study^{Footnote 5} was published to guide patients, caregivers, pediatric and adult rheumatologists and other members of the multi-disciplinary care team and provide support to JIA patients during this process. Cassie+Friends is implementing programs and initiatives to address and adapt recommendations that further support youth transitioning from pediatric to adult rheumatology care.

Producing an Arthritis Value-Framework with Economic Evidence – Paving the Way for Rare Childhood Diseases (PAVE)

The critical partnerships with Cassie and Friends in UCAN and the JIA PaCERs were also instrumental in the successful application for the European Joint Programme for Rare Diseases (EJP RD) grant, which funded her project entitled Producing an Arthritis Value-Framework with Economic Evidence – Paving the Way for Rare Childhood Diseases (PAVE).

The PAVE team is a strong international partnership of 6 countries including clinical leaders, researchers, social scientists and patient organizations. This project will build a value framework to investigate the socioeconomic impact of living with a rare disease, with data-informed calls to action to measure and ultimately reduce the cost and impact of childhood arthritis.

The analysis will be conducted by bringing together the voices of children and families, including the JIA PaCER, INBAR Israeli Arthritis Foundation, Cassie and Friends Society, and the European Network for Children with Arthritis and Autoinflammatory Diseases (ENCA) patient researchers. Patient and family research partners will co-design and help define the value framework and identify strategies to improve the lives of people living with JIA.

One Child Every Child initiative

Dr. Marshall is part of the team (Value, Impact and Knowledge Mobilization Accelerator lead) spearheaded by Dr. Susa Benseler and her team based at the Alberta Children's Hospital Research Institute (ACHRI). In April 2023, they received the Canada First Research Excellence Fund to support the One Child Every Child initiative to promote equitable improvement of children's lives worldwide.

Precision health and wellness is one of the initiative's strategic areas, focusing on improving diagnosis and treatment for children with chronic and complex medical conditions. Patient partnership is a component of this initiative, included at multiple levels levels from membership at the highest level of governance to partnership in each of the research themes and accelerators, to building research capacity of our patient partners through training in our Patient and Community Engagement Research Programme at the University of Calgary.

This successful achievement was a result of the international and national collaboration of various teams, including Genome Canada/Alberta, CIHR, Large Scale Applied Research Project (LSARP) programs (UCAN CAN-DU/CURE and Care4Rare), and Genomic Applications Partnership Program (GAPP) (TIGeR: Translational Implementation of Genomics for Rare diseases).

Summary

Collectively, these activities are improving capacity building in patient research, which contribute to patient research partners' involvement in research by sharing critical experiences of living with a health condition with other experts in JIA, ethics, law, and data sharing.

Date modified:

2023-09-26