Related Experiment Video
Updated: May 19, 2026
Refined Murine Model of Idiopathic Pulmonary Fibrosis
Published on: June 17, 2025
A progression-free end-point for idiopathic pulmonary fibrosis trials: lessons from cancer
Carlo Vancheri1, Roland M du Bois
1Dept of Clinical and Molecular Biomedicine, University of Catania, Catania, Italy. vancheri@unict.it
Abstract:
Idiopathic pulmonary fibrosis (IPF) is a progressive fibroproliferative disease that results in increasing morbidity. To date there is only one licensed therapy for this condition and other agents are needed for this attritional disease. Efforts to study other agents have been obstructed by an increasing division of opinion about the most clinically meaningful end-point of phase III clinical trials to demonstrate efficacy. Many clinicians believe that an agent that impedes progression of the disease is more than acceptable and will encourage the pharmaceutical industry to further develop their IPF programmes. We have been impressed by the behavioural and biological similarities of cancer and IPF, and wondered if lessons could be learned about clinical trial design from lung cancer studies. Here, we set out our arguments that the similarities with cancer justify comparing the magnitude of therapeutic effects in clinical trials in nonsmall cell lung cancer with those in successful trials in IPF. We demonstrate that efficacy is of a similar magnitude in the two chronic lung diseases. We recommend that the demonstration of similar magnitudes of progression-free disease effect in IPF, using appropriate indices, should be considered as clinically meaningful benefit in future phase III clinical trials of novel therapies.
Insights
New research suggests comparing clinical trial results from idiopathic pulmonary fibrosis (IPF) and non-small cell lung cancer. This approach could establish meaningful endpoints for developing new IPF therapies.
Area of Science:
- Pulmonary Medicine
- Oncology
- Clinical Trial Design
Background:
- Idiopathic pulmonary fibrosis (IPF) is a progressive lung disease with limited treatment options.
- Current drug development for IPF faces challenges due to disagreements on clinically meaningful trial endpoints.
- There is a need for novel therapeutic agents to manage IPF progression.
Purpose of the Study:
- To explore the potential of using clinical trial designs from non-small cell lung cancer (NSCLC) studies for IPF drug development.
- To establish a benchmark for therapeutic efficacy in IPF trials based on comparisons with NSCLC.
- To advocate for a revised definition of clinically meaningful benefit in IPF trials.
Main Methods:
- Comparative analysis of therapeutic effect magnitudes in phase III clinical trials for IPF and NSCLC.
- Examination of behavioral and biological similarities between IPF and cancer.
- Evaluation of progression-free disease as a potential endpoint.
Main Results:
- The magnitude of therapeutic effects in successful IPF trials is comparable to those in NSCLC trials.
- Similarities in disease progression and response suggest shared therapeutic principles.
- Progression-free disease demonstrates a similar magnitude of effect in both chronic lung diseases.
Conclusions:
- Therapeutic effects in IPF and NSCLC trials show comparable magnitudes.
- Demonstrating similar progression-free disease benefits in IPF trials should be considered clinically meaningful.
- This approach can encourage pharmaceutical investment in IPF drug development programs.
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