Orphanet Journal of Rare Diseases

Table 1 Number of trials and trial characteristics for all data

From: Estimating the clinical cost of drug development for orphan versus non-orphan drugs

	Non-Orphan					Orphan
	Phase					Phase
	Missing	1	2	3	Total	Missing	1	2	3	Total
Number of trials	57	95	94	315	561	123	122	204	153	602
Proportion (%)	10.16	16.93	16.76	56.15	100.00	20.43	20.27	33.89	25.42	100.00
Sex
Missing	56	38	37	126	257	111	37	57	24	229
Both	1	40	53	175	269	11	81	134	123	349
Female	0	3	3	13	19	0	2	11	6	19
Male	0	14	1	1	16	1	2	2	0	5
Age
Missing	56	38	37	126	257	111	37	57	24	229
Adult	0	44	15	26	85	2	8	8	0	18
Adult \| Senior	0	11	37	136	184	4	63	93	91	251
Child	0	1	2	4	7	1	1	2	6	10
Child \| Adult	0	0	0	1	1	0	5	8	4	17
Child \| Adult \| Senior	1	1	3	22	27	5	8	36	28	77
Funder Type
Missing	56	38	37	126	257	111	37	57	24	229
Industry	0	52	55	181	288	3	53	87	106	249
Industry \| NIH	0	0	0	0	0	0	0	1	1	2
Industry \| NIH \| Other	0	1	0	0	1	0	1	3	0	4
Industry \| Other	0	3	0	2	5	0	4	18	4	26
NIH	0	1	0	4	5	0	10	14	3	27
NIH \|Other	0	0	0	0	0	4	11	8	3	26
NIH \| U.S.Fed	0	0	0	0	0	0	0	0	1	1
Other	1	0	2	2	5	5	6	16	11	38
Study Start
Missing	55	40	42	123	260	40	40	42	28	150
1981 – 1985	0	0	1	0	1	3	0	0	0	3
1986 – 1990	0	0	0	3	3	7	0	3	1	11
1991 - 1995	0	0	1	1	2	30	3	5	9	47
1996 - 2000	1	1	4	25	31	21	8	26	15	70
2001 - 2005	0	24	17	73	114	18	27	77	60	182
2006 - 2010	1	21	15	55	92	1	28	28	26	83
2011 - 2015	0	9	14	35	58	3	16	23	14	56

Note: All values reflect number of trials unless otherwise specified. NIH = National Institutes of Health

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ISSN: 1750-1172

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