Rare lysosomal disease registries: lessons learned over three decades of real-world evidence

Table 1 Observational registries versus clinical trials

Characteristics	Registries	Clinical studies
Purpose	Real-world observations	Controlled experiments
Duration	Indefinite	Finite
Inclusion criteria	General	Specific
Data collection	Voluntary	Required
Visits	Per medical practice	Per protocol
Analytical methods	Epidemiology	Biostatistics
Disease characteristics	Cross-sectional, longitudinal	Per protocol
Treatment outcomes	Long-term effectiveness	Efficacy and safety
Applicability	Broad patient populations	Per protocol

ISSN: 1750-1172