Outcome selection for tissue-agnostic drug trials for immune-mediated inflammatory diseases: a systematic review of core outcome sets and regulatory guidance

Table 1 Characteristics of included publications

Characteristics	Inflammatory conditions
Characteristics	ALL	RA*	JIA	AS	PsA	SS	CD**	UC	Uv	SLE*** (jSLE)
Total number included	44	10	2	4	8	2	7	2	1	6 (2)
Type of publication:
Collaborative reports	34	8	1	3	7	2	6	0	1	4 (2)
Regulatory guidance	10	2^X	1^e	1^e	1^e	0	1^e	2^X	0	2^X (0)
Patient population:
Adults	37^ap	10^ap	–	4	8	2	5^ap	2^ap	0	6^ap
Paediatric specific	7	0	2	0	0	0	2	0	1	2
Purpose:
Trials and LOS	43	10	2	3	8	2	7	2	1	6 (2)
Routine practice	8	2	0	2	2	0	1	0	0	0 (0)
Registries	4	0	0	3	1	0	0	0	0	0 (0)
Methods:
Initial literature or systematic review/database search	22	6	0	3	3	1	5	0	1	3 (0)
Surveys	9	2	1	1	2	1	0	0	1	0 (1)
Interviews/focus groups	4	0	0	1	2	0	1	0	0	0 (0)
Delphi/ NGT/other consensus group meeting	32	8	1	2	6	2	6	0	1	4 (2)
Patient involvement	14	5	0	2	4	0	3	0	0	0 (0)
HCP involvement	34	8	1	3	7	2	6	0	1	4 (2)

AS ankylosing spondylitis, CD Crohn’s disease, EMA European Medicines Agency, FDA Food and Drug Administration, HCP health care professionals, ICHOM International Consortium for Health Outcomes Measurement, JIA juvenile idiopathic arthritis, LOS longitudinal observation studies, NGT nominal group technique, PsA psoriatic arthritis, RA rheumatoid arthritis, SLE systemic lupus erythematosus, jSLE juvenile SLE, SS Sjogren’s syndrome, UC ulcerative colitis, uV uveitis
*ICHOM 2018 document for inflammatory arthritis covers RA, PsA, AS and JIA
**Kim 2018 and Ruemmele 2014 referred to both CD and UC (inflammatory bowel disease). To avoid confusion, these have been recorded under CD
***Values in brackets represent the subsets relating to jSLE (under SLE)
^eDenotes an EMA publication whilst ^f represents an FDA document
^xSignifies that both EMA and FDA produced documents for the condition
^apSignifies the inclusion of publications with mixed patient populations

ISSN: 1745-6215