Efficacy and safety of compassionate use for rare diseases: a scoping review from 1991 to 2022

Table 1 Characteristics of CU case reports/case series

	Items	Number of studies	%
Disease classification	01 Certain infectious or parasitic diseases	5	10.9
	02 Neoplasms	23	50.0
	03 Diseases of the blood or blood-forming organs	3	6.5
	04 Diseases of the immune system	2	4.3
	05 Endocrine, nutritional or metabolic diseases	4	8.7
	08 Diseases of the nervous system	3	6.5
	13 Diseases of the digestive system	2	4.3
	15 Diseases of the musculoskeletal system or connective tissue	1	2.2
	19 Certain conditions originating in the perinatal period	1	2.2
	20 Developmental anomalies	2	4.3
Age, years	0–8 years	6	13.0
	> 8–18 years	8	17.4
	> 18–60 years	22	47.8
	> 60 years	10	21.7
Treatment effect	Cured	4	8.7
	Symptoms improved	35	76.1
	No significant effect	4	8.7
	Worsened	1	2.2
	No description	2	4.3
TEAE	Yes	31	67.4
	None	13	28.3
	No description	2	4.3

ISSN: 1750-1172