Evaluating the national system for rare diseases in China from the point of drug access: progress and challenges

Table 1 Median availability of orphan drugs at hospital level in China from 2017 to 2020 (%)

Year (numbers of marketed orphan drugs)	Nationwide		Eastern area		Middle area		Western area		p value^c
Year (numbers of marketed orphan drugs)	Availability (IQR)^a	Median of hospital-specific change (IQR)^b	Availability (IQR)	Median of hospital-specific change (IQR)	Availability (IQR)	Median of hospital-specific change (IQR)	Availability (IQR)	Median of hospital-specific change (IQR)	p value^c
2017	27.2 (10.4)		31.0 (8.9)		24.7 (27.8)		27.2 (7.9)		< 0.001
2018	32.9 (10.1)	1.9 (5.1)**	34.8 (18.7)	1.9 (3.1)*	25.3 (32.3)	1.3 (4.7)	32.9 (10.1)	3.2 (6.6)*	< 0.001
2019	34.8 (11.4)	1.9 (3.5)	36.1 (19.3)	2.5 (5.4)	30.4 (29.1)	3.2 (2.8)	34.2 (10.1)	1.3 (4.7)	< 0.001
2020	41.1 (12.3)	5.1 (7.6)*	43.0 (20.3)	3.2 (10.8)	36.7 (27.8)	5.1 (9.2)	36.7 (14.6)	8.9 (9.2)*	< 0.001

^aIQR: Interquartile range, equals to the difference between 75 and 25th percentiles
^bMedian of hospital-specific change is defined as the median of orphan drug availability annual change in each hospital. The p value of Wilcoxon rank-sum test for the difference of median of hospital-specifific change each year compared with the previous year: *for p < 0.05, **for p < 0.01
^cp value of Wilcoxon rank-sum test for the difference of orphan drugs' average availability at hospital level between eastern, western, and middle areas each year

ISSN: 1750-1172