Reconciling Healthcare Professional and Patient Perspectives in the Development of Disease Activity and Response Criteria in Connective Tissue Disease–related Interstitial Lung Diseases

Abstract

Interstitial lung diseases (ILD), including connective tissue disease (CTD) related and idiopathic pulmonary fibrosis (IPF), carry a high morbidity and mortality. Great efforts are underway to develop and investigate meaningful treatments in the context of clinical trials. However, these efforts have been challenged by the lack of validated outcome measures and inconsistent use of measures in the context of clinical trials. This lack of consensus has fragmented effective use of investigative in CTD-ILD and IPF with a history of resultant difficulties in agency approval of treatment interventions. Patient perspective in determination of domains and outcome measures in CTD-ILD and IPF, prior to this effort, has never occurred. These efforts demonstrate unequivocally the value and impact of patient involvement on core set development. Regarding CTD-ILD, this is the first OMERACT working group to directly address a manifestation/co-morbidity 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.

Design of the PF-ILD trial: a double-blind, randomised, placebo-controlled phase III trial of nintedanib in patients with progressive fibrosing interstitial lung disease

“…Selection of patient-reported outcome measures was based on two documents: the US Food and Drug Administration (FDA) reports 'The Voice of the Patient,' 31 and a manuscript from the Outcome Measures in Rheumatology working group. 32 Both documents conclude that the most relevant aspects of the disease for patients with IPF, and with ILDs in general, are HRQoL, dyspnoea and cough. Accordingly, the absolute change from baseline to week 52 in the K-BILD total score has been included as one of the main secondary endpoints.…”

Outcome Measures for Clinical Trials in Interstitial Lung Diseases

“…A recent effort attempted to address this dilemma via consensus of an international community of IIP experts [12,13] and provide preliminary recommendations for the minimum set of outcome measures to be incorporated into future clinical trials.…”

Monitoring of Lung Involvement in Rheumatologic Disease

“…Semi-quantitative dyspnoea scales may add value in clinical trials [8,13,14] but offer no practical advantages in routine monitoring. Thus, symptomatic change should not be interpreted in isolation but should be integrated with objective measures, especially LFTs and imaging.…”