Furthermore, 75% (n = 3) of included economic evidence concluded a positive pharmacoeconomic profile of ciltacabtagene autoleucel in multiple myeloma (MM) treatment [55,56,57], while 60% (n = 3) of economic evidence demonstrated voretigene neparvovec as a cost-effective option in RPE65-associated inherited retinal disease (RPE65-IRD treatment) [58,59,60]. Here, RPE65 is linked to Miyoshi myopathy.