RWD233 CHARACTERISTICS OF AUSTRALIAN PBAC SUBMISSIONS USING RWE

a broad spectrum of hospital services, including pharmacy, outpatient, inpatient, emergency, and other treatments.Indirect costs from lost workdays were significant, with parents of pediatric patients losing 81 days ($2,914.29)annually, while adult patients and caregivers lost 33 and 31 days, costing $1,119.28 and $1,191.50,respectively.In addition, NF1 affects patients quality of life by experiencing physical, psychiatric, psychological, emotional, and social challenges.These include visible disabilities, cognitive and functional impairments, dependency, and difficulties in school and daily activities.Compared to the general population, individuals with NF1 face markedly higher levels of worry and compromised social and emotional well-being.Conclusions: Although rare, NF1 imposes a substantial burden on patients and their families.Notably, its economic impact surpasses that of many common chronic diseases, highlighting its significant burden.Implementing timely and effective treatment strategies is crucial to mitigating the profound financial and societal challenges.