Patient Participation in Patient Reported Outcome Instrument Development in Systemic Sclerosis

John Pauling^1,2, Tracy M. Frech^3,4, Robyn T. Domsic⁵ and Marie Hudson^6,7, ¹Department of Pharmacy and Pharmacology, University of Bath, Bath, United Kingdom, ²Department of Rheumatology, Royal National Hospital for Rheumatic Diseases, Bath, United Kingdom, ³Internal Medicine, Salt Lake City VAMC, Salt Lake, UT, ⁴Internal Medicine-Division of Rheumatology, University of Utah School of Medicine, SLC, UT, ⁵Medicine - Rheumatology, Univ of Pittsburgh Med Ctr, Pittsburgh, PA, ⁶Medicine, McGill University, Montreal, QC, Canada, ⁷Medicine/Rheumatology, Jewish General Hospital, Lady Davis Research Institute, Montreal, QC, Canada

Meeting: 2016 ACR/ARHP Annual Meeting

Date of first publication: September 28, 2016

Keywords: FDA, Outcome measures, Participation, patient engagement and systemic sclerosis

Session Information

Date: Tuesday, November 15, 2016

Title: Systemic Sclerosis, Fibrosing Syndromes, and Raynaud's – Clinical Aspects and Therapeutics - Poster III

Session Type: ACR Poster Session C

Session Time: 9:00AM-11:00AM

Background/Purpose: The patient perspective captured using Patient-Reported Outcome (PRO) instruments provide valuable insight into the patient condition not always captured by physician-derived assessment tools. Target patient population involvement is considered an essential component of PRO instrument design and is assessed by regulatory bodies when considering labelling claims in medical product development. We have reviewed the level of patient involvement in the development of PRO instruments used in the assessment of systemic sclerosis (SSc).

Methods: A comprehensive literature review was undertaken to identify studies reporting PRO instruments in SSc. Studies were assessed to establish whether the PRO instruments had been developed specifically for SSc or adopted from other disease areas. Studies reporting PRO instruments specific for SSc were scrutinised for evidence of target patient population involvement in the development of the instrument.

Results: A total of 58 PRO instruments that have been used in SSc research were identified. Twelve (21%) of these were developed specifically for outcome assessment within SSc populations (Table). Of these, 5 (42%) had not reported any patient involvement in the development phase of the instrument. Five SSc PRO instruments (42%) involved target patient population in the domain/item generation stage. Four (33%) of SSc PRO instruments had undertaken cognitive interviewing/linguistic evaluation to ensure item wording adequately captured the intended conceptual framework. The SCTC GIT questionnaires and the Systemic Sclerosis Questionnaire had each involved SSc patients in both domain/item generation and cognitive interviewing/linguistic evaluation stages of instrument development.

Conclusion: PRO instruments are particularly valuable in SSc due to the multi-faceted nature of the disease and the paucity of effective objective methods for assessing disease status. The majority of existing PRO instruments used in SSc have not involved significant target patient involvement in their development. By involving patients in the development and design phase of novel PRO instruments in SSc, we can ensure that PRO instruments used in the clinical and research settings adequately capture experiences most relevant to our patients. Table. Patient involvement in development of SSc-specifc PRO instruments * Authors report patient involvement but no details supplied.

Organ system	Conceptual Framework	Author, Year	PRO	Patient involvement in:
Organ system	Conceptual Framework	Author, Year	PRO	Conceptual Framework	Domain generation	Item generation	Cognitive interviewing	Linguistic Evaluation	Respondent Burden
Disability & function	Disability & Function in SSc	Steen and Medsger, 1997	Scleroderma HAQ subscales	No	No	No	No	Yes	No
	Disability and Function in SSc	Silman et al., 1998	UK Scleroderma Functional Score	No	No *	No *	No	No	No
	Disability and Function in SSc	Guillevin and Ortonne, 1983	Scleroderma Functional Index	No	No	No	No	No	No
Global Assessment of Health Status	Global disease assessment	Suarez-Almazor, et al., 2007 & Kallen et al. 2010	Symptom Burden Index	No	Yes	Yes	No	No	No
	Global disease assessment in SSc	Ruof et al., 1999	Systemic Sclerosis Questionnaire	No	Yes	Yes	Yes	Yes	No
	Global disease assessment in SSc	Ostojic and Damjanov, 2006	Scleroderma Assessment Questionnaire	No	Yes	Yes	No	No	No
Skin	Skin thickening, tethering and thinness in SSc	Nagy et al. 2009	Patient Skin Self Assessment Questionnaire	No	No	No	No	Yes	No
Body Image	Body Image in SSc	Jewett, 2015	BCSS	No	No	No	No	No	No
	Body Image in SSc	Jewett, 2010	Brief-SWAP	No	No	No	No	No	No
Peripheral Vascular	Raynaud’s phenomenon in SSc	Wigley et al., 1998 & Black et al. 1998	Raynaud’s Condition Score Diary	No	No	No	No	No	No
Gastrointestinal	GI symptoms in SSc	Khanna et al. 2007 & Khanna et al. 2009	SCTC GIT 1.0 and 2.0	No	Yes	Yes	Yes	Yes	No
	Mouth Handicap in SSc	Mouthon et al., 2007	MHISS	Yes Postal survey	Yes Postal survey	Yes Postal survey	No	No	No

Disclosure: J. Pauling, None; T. M. Frech, None; R. T. Domsic, None; M. Hudson, None.

To cite this abstract in AMA style:

Pauling J, Frech TM, Domsic RT, Hudson M. Patient Participation in Patient Reported Outcome Instrument Development in Systemic Sclerosis [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). https://acrabstracts.org/abstract/patient-participation-in-patient-reported-outcome-instrument-development-in-systemic-sclerosis/. Accessed .

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