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Pirfenidone for Treating Idiopathic Pulmonary Fibrosis: An Evidence Review Group Perspective of a NICE Single Technology Appraisal

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Abstract

The National Institute for Health and Care Excellence (NICE) published guidance on the use of pirfenidone (Esbriet®, Roche) for the treatment of mild to moderate idiopathic pulmonary fibrosis (IPF) in 2013. NICE decided to review existing guidance following publication of an additional clinical trial, and invited the manufacturer of pirfenidone to submit evidence of its clinical and cost effectiveness for the treatment of mild to moderate IPF when compared with best supportive care (BSC) or nintedanib; nintedanib was a comparator only for moderate IPF. An independent Evidence Review Group (ERG) critiqued the company submission and this paper summarises their report and subsequent NICE guidance. The key clinical effectiveness evidence was based on three randomised controlled trials (RCTs) and an open-label extension study. Supportive data were provided from two additional RCTs conducted in Japan, while one additional open-label study was included for safety outcomes. Meta-analysis of the three key RCTs found pirfenidone to be effective at reducing disease progression compared with placebo, but statistically significant differences were not identified in all of the RCTs. A statistically significant reduction in all-cause mortality was only demonstrated when pooling data across studies. The treatment effects of pirfenidone and nintedanib were broadly similar, based on an indirect comparison using network meta-analysis, although they have slightly different adverse event profiles. There remains considerable uncertainty in the cost-effectiveness estimates for pirfenidone versus BSC, particularly due to uncertainty regarding the duration of treatment effect and the method used to implement the stopping rule within the economic model.

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Acknowledgements

The authors wish to thank Dr. Stephen Bianchi and Professor David Thickett for providing clinical advice and commenting on draft materials during the project.

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Authors and Affiliations

  1. ScHARR, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK

    Sarah Davis, Rachid Rafia, Christopher Carroll, Jean Hamilton & Munira Essat

Authors
  1. Sarah Davis
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  2. Rachid Rafia
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  3. Christopher Carroll
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  4. Jean Hamilton
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  5. Munira Essat
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Contributions

SD, CC, JH (formerly named Jean Sanderson), ME and RR drafted the manuscript and take responsibility as guarantors of the content. All authors have given their approval for the final version to be published. This summary has not been externally reviewed by PharmacoEconomics

Corresponding author

Correspondence to Sarah Davis.

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Funding

This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment Programme (project number 142/06/02 STA); see the HTA programme website for further project information (http://www.hta.ac.uk). This summary of the ERG report was compiled after NICE issued guidance. The views and opinions expressed herein are those of the authors and do not necessarily reflect those of NICE or the Department of Health.

Conflict of Interest

SD, RR, CC, JH and ME have no potential conflicts of interest.

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Davis, S., Rafia, R., Carroll, C. et al. Pirfenidone for Treating Idiopathic Pulmonary Fibrosis: An Evidence Review Group Perspective of a NICE Single Technology Appraisal. PharmacoEconomics 37, 763–775 (2019). https://doi.org/10.1007/s40273-018-0727-1

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