OBJECTIVE: The use of qualitative and real-world data (RWD) are important complementary methods for disease and treatment outcomes research. However, neuronal ceroid lipofuscinosis (CLN2) research has been restricted to clinical study due to practical and ethical concerns regarding the affected children. To our knowledge, this is the first study undertaken to explore the utility of non-clinical RWD (i.e. school reports) for the qualitative study of CLN2 experiences and enzyme replacement therapy.
METHODS: Annual school reports and surveys were collected from a purposive sampling of 9 children with CLN2. A retrospective, reflexive thematic analysis was conducted to explore how these reports represent children and CLN2, and how this compares to guardians' perceptions of their child's life out with school.
RESULTS: Findings revealed that each examined site has used statutory reporting frameworks in inconsistent ways to report on curricular attainment and future-focused targets. School reports represented children with CLN2 through 1) subjective observations of their behaviours and (inter)personal, physical, and intellectual development; and 2) the interests of educators and guardians. Sparse reporting was found regarding the atypical development or regression experienced by children. Dissonance was also found between report content and guardians' perceptions and experiences.
CONCLUSIONS: For the purposes of disease and treatment outcomes research, school reports offer inconsistent RWD unable to clearly represent atypical development or regression. Their utilisation would therefore require the support of education systems to develop more relevant and consistent reporting methods. However, school reports do offer an unobtrusive method of data collection that can help describe positive treatment outcomes for children living with paediatric neurodegenerative diseases in support of their right to the highest attainable standard of health.
