In 2018 novel therapies became available for use in older children, initially in the United States and then followed by a number of European countries. The introduction of novel therapies is important to assess and to inform continuing improvement in the standard of care and achievement of improved joint health and associated quality of life for paediatric haemophilia patients. Consequently, HCD Economics is working with academic, patient group and research community partners to launch CHESS PAEDS II. In particular, the implication of access as potentially restricted by the age of the paediatric haemophilia patient needs to be further explored.
Writing in Blood (American Society of Haematology) in 2020, Paul McLaughlin et al confirmed that while chronic joint damage is most often associated with severe haemophilia more recent research has illustrated that people with moderate haemophilia also experience haemophilic arthropathy and functional impairment. The Joint Outcome Continuation Study, reported findings that Factor VIII prophylaxis was insufficient to protect joints from damage, from childhood through adolescence. Novel therapies, act as bypassing agents, replacing factor VII prophylaxis. The ultimate goal of paediatric bypassing treatment is to preserve the child’s joints, otherwise destroyed by recurrent bleeds.
Consequently, the study of burden in the paediatric population that has had access to novel therapies is compelling to understand alongside the burden of illness studied in a paediatric population that has not had access to novel therapies. Data from CHESS PAEDS I has been instrumental in contributing to this understanding and as a population of paediatric patients who have been prescribed novel therapies is now available for observation, there is a research obligation to launch CHESS PAEDS II.
CHESS PAEDS II will recruit a broad and representative group of paediatric haemophilia patients aged from 1-17. The recruitment will extend to parents and caregivers of these patients (CHESS PAEDS I identified statistically significant impact on parents who care for their children with haemophilia, causing them to miss work and forgo financial and social opportunities). The focus will be on moderate and severe haemophilia in the US and EU5, including patients with no previous factor treatment and older children who have started to be prescribed novel treatments.
Recognising the findings from CHESS PAEDS I (a bibliography of publication citations is available on request) CHESS PAEDS II will take the perspective of children and their parents to evaluate joint health, quality of life impact prior to lifestyle adaption and activity levels in the study population. As with CHESS PAEDS I, the study methodology will adopt the University of Chester/HCD Economics Burden of Illness (BOI) design, which is rooted in the principle of patient and public involvement and engagement, and draws upon physician submitted case report forms, along with a pool of patients that are physician recruited or directly recruited via Patient Advocacy Groups (PAGS). These PAGS are pivotal to the study implementation as members of the study Expert Review Group.
For more information on the CHESS PAEDS II research programme, including opportunities to join the research community investors partnering in the study, please contact Alison Rose. alison.rose@hcdeconomics.com.