Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?

Patient-reported outcome measures (PROMs), tools to assess patient self-report of health status, are now increasingly used in research, care and policymaking. While there are two well-developed disease-specific PROMs for interstitial lung diseases (ILD) and idiopathic pulmonary fibrosis (IPF), many unmet and urgent needs remain. In December 2019, 64 international ILD experts convened in Erice, Italy to deliberate on many topics, including PROMs in ILD. This review summarises the history of PROMs in ILD, shortcomings of the existing tools, challenges of development, validation and implementation of their use in clinical trials, and the discussion held during the meeting. Development of disease-specific PROMs for ILD including IPF with robust methodology and validation in concordance with guidance from regulatory authorities have increased user confidence in PROMs. Minimal clinically important difference for bidirectional changes may need to be developed. Cross-cultural validation and linguistic adaptations are necessary in addition to robust psychometric properties for effective PROM use in multinational clinical trials. PROM burden of use should be reduced through appropriate use of digital technologies and computerised adaptive testing. Active patient engagement in all stages from development, testing, choosing and implementation of PROMs can help improve probability of success and further growth.

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Conflict of interest: M. Kalluri reports grants and personal fees from Boehringer Ingelheim, and Roche, outside the submitted work. Conflict of interest: F. Luppi reports grants and lecture fees from Roche and lecture fees from Boehringer Ingelheim. Conflict of interest: A. Vancheri has nothing to disclose. Conflict of interest: C. Vancheri reports grants and personal fees from Roche and Boehringer Ingelheim, outside the submitted work. Conflict of interest: E. Balestro reports personal fees from Roche and Boehringer Ingelheim, outside the submitted work. Conflict of interest: F. Varone reports unrestricted grants, consultancy and lecture fees from Roche and Boehringer Ingelheim. Conflict of interest: N. Mogulkoc has nothing to disclose. Conflict of interest: G. Cacopardo has nothing to disclose. Conflict of interest: E. Bargagli has nothing to disclose. Conflict of interest: E. Renzoni reports grants from Boeringher Ingelheim, and lecture fees from Boeringher Ingelheim and Roche, outside the submitted work. Conflict of interest: S. Torrisi reports personal fees from Hoffman La-Roche and Boehringer Ingelheim, outside the submitted work. Conflict of interest: M. Calvello has nothing to disclose. Conflict of interest: A. Libra has nothing to disclose. Conflict of interest: M. Pavone has nothing to disclose. Conflict of interest: F. Bonella reports grants and personal fees from Boehringer Ingelheim and Roche, and consultancy fees from BMS, Galapagos, GSK and Savara, outside the submitted work. Conflict of interest: V. Cottin reports personal fees and non-financial support from Actelion and Roche/Promedior; grants, personal fees and non-financial support from Boehringer Ingelheim; and personal fees from Bayer/MSD, Novartis, Sanofi, Celgene/BMS, Galapagos, Galecto, Shionogi, AstraZeneca and Fibrogen, outside the submitted work. Conflict of interest: C. Valenzuela reports personal fees from Boehringer Ingelheim, F. Hoffmann-La Roche, Galapagos and BMS, outside the submitted work. Conflict of interest: M. Wijsenbeek reports grants and other funding from Boehringer Ingelheim and Hoffman-La Roche, and other funding from Respivant, Galapagos, Novartis and Savara, outside the submitted work. All fees and grants were paid to her institution. Conflict of interest: E. Bendstrup reports grants and personal fees from Boehringer Ingelheim and Hoffmann-La Roche, and personal fees from Galapagos and AstraZeneca, outside the submitted work.