Unmet needs in the treatment of idiopathic pulmonary fibrosis-insights from patient chart review in five European countries
Open accessAbstract
BACKGROUND: Two antifibrotic drugs, pirfenidone and nintedanib, are approved by the European Medicines Agency and the US Food and Drug Administration for the treatment of idiopathic pulmonary fibrosis (IPF). In this analysis, treatment patterns of European patients with IPF were investigated to understand antifibrotic prescribing and identify unmet needs in IPF treatment practice. METHODS: Between February and March 2016, respiratory physicians from France, Germany, Italy, Spain, and the UK participated in an online questionnaire designed to collect information on IPF treatment patterns in patients under their care. Patients were categorized as treated (received approved antifibrotics) or untreated (did not receive approved antifibrotics, but may have received other unapproved therapies). Classification of IPF diagnosis (confirmed/suspected) and severity ('mild'/'moderate'/'severe') for each patient was based on the individual physician's report. Patients' perspectives were not recorded in this study. RESULTS: In total, 290 physicians responded to the questionnaire. Overall, 54% of patients with IPF did not receive treatment with an approved antifibrotic. More patients had a confirmed IPF diagnosis in the treated (84%) versus the untreated (51%) population. Of patients with a confirmed diagnosis, 40% did not receive treatment. The treated population was younger than the untreated population (67 vs 70 years, respectively; p ≤ 0.01), with more frequent multidisciplinary team evaluation (83% vs 57%, respectively; p ≤ 0.01). A higher proportion of untreated patients had forced vital capacity > 80% at diagnosis versus treated patients. Of patients with 'mild' IPF, 71% did not receive an approved antifibrotic versus 41% and 60% of patients with 'moderate' and 'severe' IPF, respectively. CONCLUSIONS: Despite the availability of antifibrotic therapies, many European patients with confirmed IPF do not receive approved antifibrotic treatment. Importantly, there appears to be a reluctance to treat patients with 'mild' or 'stable' disease, and instead adopt a 'watch and wait' approach. More education is required to address diagnostic uncertainty, poor understanding of IPF and its treatments, and issues of treatment access. There is a need to increase physician awareness of the benefits associated with antifibrotic treatment across the spectrum of IPF severity.
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The limited efficacy and tolerability challenges of pirfenidone and nintedanib contribute to low treatment uptake, frequent discontinuation, and impaired quality of life.
“Their limited efficacy, combined with tolerability challenges, contributes to low treatment uptake, frequent discontinuation of both treatments, and impaired quality of life.6-8”
From Deupirfenidone Compared with Pirfenidone and Placebo in Idiopathic Pulmonary Fibrosis (ELEVATE-IPF): a Phase 2b Randomized Placebo-Controlled Trial (2026) · cites this paper as [8]
The Discussion (page unavailable) says adverse events from pirfenidone and nintedanib may limit tolerability or lead to discontinuation, and suggests that concerns about side effects may affect treatment decisions. The Results report low uptake, but only 1.2% of patients discontinued a medication at their last consultation—not frequent discontinuation. The Discussion describes impaired quality of life as a possible physician perception, not an established treatment effect, and does not establish limited efficacy as the cause of low uptake. (LLM, conf=0.88)
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References 45 · Cited by 108
- Authors
- Maher, T. M., Molina-Molina, M., Russell, A.-M., Bonella, F., Jouneau, S., Ripamonti, E., Axmann, J., & Vancheri, C.
- Affiliations
- National Institute for Health and Care Research (GB)
- Ruhrlandklinik (DE)
- Institut de Recherche en Santé, Environnement et Travail (FR)
- F. Hoffmann-La Roche AG (CH)
- University of Catania (IT)
- Corresponding author
- Maher, Toby M.
- Venue
- BMC Pulmonary Medicine
- Published
- 2017-09-15
- Volume
- 17
- Issue
- 1
- Pages
- 124-124
- ISSN
- 1471-2466 (ISSN-L)
- PMC
- PMC5602932
- OpenAlex
- W2754910468
- Cited by
- 108
- References
- 45
- Citations by year
- 2026: 9 · 2025: 4 · 2024: 10 · 2023: 19 · 2022: 13 · 2021: 21 · 2020: 10 · 2019: 12 · 2018: 9 · 2017: 1
- FWCI
- 7.24
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- cc-by
- Locations
- BMC Pulmonary Medicine (publishedVersion, cc-by)
- Europe PMC (PubMed Central) (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Dipòsit Digital de la Universitat de Barcelona (Universitat de Barcelona) (publishedVersion, cc-by)
- HAL (submittedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- RECERCAT (Consorci de Serveis Universitaris de Catalunya) (submittedVersion, cc-by)
- Spiral (Imperial College London) (submittedVersion, other-oa)
- Funders
- F. Hoffmann-La Roche (CH)
- Topics
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Interstitial Lung Diseases and Idiopathic Pulmonary Fibrosis
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Chronic Obstructive Pulmonary Disease (COPD) Research
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Pulmonary Hypertension Research and Treatments
- Keywords
- idiopathic pulmonary fibrosis, antifibrotic therapy, pirfenidone, nintedanib, treatment patterns, unmet medical needs, undertreatment, physician prescribing, disease severity, multidisciplinary care, watch-and-wait approach, treatment access, physician awareness, diagnostic uncertainty, European patients, patient chart review, physician survey, forced vital capacity
- MeSH terms
- Aged, Aged, 80 and over, Anti-Inflammatory Agents, Non-Steroidal, Europe, Female, Health Services Needs and Demand, Humans, Indoles, Male, Middle Aged, Pyridones, Surveys and Questionnaires, Severity of Illness Index, Vital Capacity, Idiopathic Pulmonary Fibrosis
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