Reconciling healthcare professional and patient perspectives in the development of disease activity and response criteria in connective tissue disease-related interstitial lung diseases

Interstitial lung diseases (ILD), including those related to connective tissue disease (CTD), and idiopathic pulmonary fibrosis (IPF) carry high morbidity and mortality. Great efforts are under way to develop and investigate meaningful treatments in the context of clinical trials. However, efforts have been challenged by a lack of validated outcome measures and by inconsistent use of measures in clinical trials. Lack of consensus has fragmented effective use of strategies in CTD-ILD and IPF, with a history of resultant difficulties in obtaining agency approval of treatment interventions. Until recently, the patient perspective to determine domains and outcome measures in CTD-ILD and IPF had never been applied. Efforts described here demonstrate unequivocally the value and influence of patient involvement on core set development. Regarding CTD-ILD, this is the first OMERACT working group to directly address a manifestation/comorbidity of a rheumatic disease (ILD) as well as a disease not considered rheumatic (IPF). The OMERACT 11 proceedings of the CTD-ILD Working Group describe the forward and lateral process to include both the medical and patient perspectives in the urgently needed identification of a core set of preliminary domains and outcome measures in CTD-ILD and IPF. Copyright © 2014. All rights reserved.

Aim

To identify domains and instruments perceived as important outcomes in the context of a 1-year multicenterrandomized controlled trial (RCT) of a promising treatment for idiopathic pulmonary fibrosis (IPF) and/or connective tissue disease associated interstitial lung disease (CTD-ILD).

Contributors

Saketkoo, L. A. Mittoo, S. Frankel, S. LeSage, D. Sarver, C. Phillips, K. Strand, V. Matteson, E. L.

Publication

Journal: Journal of Rheumatology
Volume: 41
Issue: 4
Pages: 792 - 798
Year: 2014
DOI: 10.3899/jrheum.131251

Further Study Information

Current Stage: Not Applicable
Date:
Funding source(s): Not reported


Health Area

Disease Category: Lungs & airways

Disease Name: Interstitial Lung Diseases

Target Population

Age Range: Unknown

Sex: Either

Nature of Intervention: Drug

Stakeholders Involved

- Clinical experts
- Patient/ support group representatives

Study Type

- COS for clinical trials or clinical research

Method(s)

- Delphi process
- Focus group(s)

(1) Structured 3-tiered Internet-based healthcare professionals Delphi process that featured links to original publications and subsequent articles. An “Inter-Expert Educational Component” allowed participants to upload commentary, articles, and links for review, as well as challenge or defend inclusion of a domain or instrument. (2) Patient-centered investigation involving data from 6 patient focus groups.