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Pirfenidone for Treating Idiopathic Pulmonary Fibrosis (Review of TA282): a Single Technology Appraisal

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Pirfenidone for Treating Idiopathic Pulmonary Fibrosis (Review of TA282): a Single Technology Appraisal Confidential until published Pirfenidone for treating idiopathic pulmonary fibrosis (review of TA282): A Single Technology Appraisal Produced by School of Health and Related Research (ScHARR), The University of Sheffield Authors Sarah Davis, Senior Lecturer in Health Economics, ScHARR, University of Sheffield, Sheffield, UK Rachid Rafia, Research Fellow, ScHARR, University of Sheffield, Sheffield, UK Christopher Carroll, Reader in Systematic Review and Evidence Synthesis, ScHARR, University of Sheffield, Sheffield, UK Munira Essat, Research Fellow, ScHARR, University of Sheffield, Sheffield, UK Jean Sanderson, Research Associate in Statistics, ScHARR, University of Sheffield, Sheffield, UK Naila Dracup, Information Specialist, ScHARR, University of Sheffield, Sheffield, UK Dr Stephen Bianchi, Consultant in Respiratory Medicine, Sheffield Teaching Hospitals NHS Foundation Professor David Thickett, Consultant in Respiratory Medicine, University Hospitals of Birmingham NHS Foundation Trust and Chair in Respiratory Medicine, University of Birmingham Correspondence Author Sarah Davis, Senior Lecturer in Health Economics, ScHARR, University of Sheffield, Sheffield, UK Date completed Date completed (05/03/2016) Source of funding: This report was commissioned by the NIHR HTA Programme as project number 142/06/02. 1 Copyright 2017 Queen's Printer and Controller of HMSO. All rights reserved. Confidential until published Declared competing interests of the authors None of the authors have any conflicts of interest to declare. Acknowledgements We would like to thank Paul Tappenden, ScHARR, for providing comments on the draft report and Andrea Shippam, Programme Manager, ScHARR, for providing administrative support and in preparing and formatting the report. Rider on responsibility for report The views expressed in this report are those of the authors and not necessarily those of the NIHR HTA Programme. Any errors are the responsibility of the authors. This report should be referenced as follows: Davis S, Rafia R, Carroll C, Essat M, Sanderson J, Dracup N, Bianchi S, Thickett D. Pirfenidone for treating idiopathic pulmonary fibrosis: A Single Technology Appraisal. School of Health and Related Research (ScHARR), 2016. Contributions of authors Christopher Carroll and Munira Essat summarised and critiqued the clinical effectiveness data reported within the company’s submission. Sarah Davis and Rachid Rafia critiqued the health economic analysis submitted by the company. Jean Sanderson critiqued the statistical analyses undertaken by the company. Naila Dracup critiqued the company’s search strategy. Dr Stephen Bianchi and Professor David Thickett acted as clinical advisors to the ERG. All authors were involved in drafting and commenting on the final report. 2 Copyright 2017 Queen's Printer and Controller of HMSO. All rights reserved. Confidential until published Abbreviations AEs Adverse events BSC Best supportive care CADTH Canadian Agency for Drugs and Technologies in Health COPD Chronic obstructive pulmonary disease CrI Credible interval CRD Centre for Reviews and Dissemination CS Company’s submission CSR Clinical study review DLco Diffusing capacity for carbon monoxide ERG Evidence Review Group EQ-5D EuroQoL five dimensions questionnaire FVC Forced vital capacity HR Hazard ratios HRCT High resolution computed tomography HRQoL Health-related quality of life HTA Health Technology Appraisal ICER Incremental cost-effectiveness ratio IPD Individual patient data IPF Idiopathic pulmonary fibrosis ITT Intention to treat KM Kaplan-Meier LOCF Last observation carried forward NAC N-acetylcysteine NICE National Institute for Health and Care Excellence NHS National Health Service NMA Network meta-analysis PBO Placebo PBS Pharmaceutical Benefits Scheme PEY Person exposure years PFN Pirfenidone PFS Progression-free survival PRISMA Preferred Reporting Items for Systematic Reviews and Meta-Analyses PSS Personal Social Services QALY Quality-adjusted life year RCT Randomised controlled trial 3 Copyright 2017 Queen's Printer and Controller of HMSO. All rights reserved. Confidential until published SD Standard deviation 6MWD Six minute walking distance SGRQ St George’s Respiratory Questionnaire STA Single Technology Appraisal TA Technology Appraisal VC Vital capacity UCSD SOBQ University of California San Diego Shortness of Breath Questionnaire 4 Copyright 2017 Queen's Printer and Controller of HMSO. All rights reserved. Confidential until published CONTENTS Abbreviations...................................................................................................................................... 3 1 SUMMARY ................................................................................................................................. 11 1.1 Critique of the decision problem in the company’s submission ........................................... 11 1.2 Summary of clinical effectiveness evidence submitted by the company .............................. 12 1.3 Summary of the ERG’s critique of clinical effectiveness evidence submitted ..................... 16 1.4 Summary of cost effectiveness evidence submitted by the company ................................... 19 1.5 Summary of the ERG’s critique of cost effectiveness evidence submitted .......................... 20 1.6 ERG commentary on the robustness of evidence submitted by the company ...................... 21 1.7 Summary of exploratory and sensitivity analyses undertaken by the ERG .......................... 22 2 BACKGROUND .......................................................................................................................... 24 2.1 Critique of company’s description of underlying health problem ........................................ 24 2.2 Critique of company’s overview of current service provision .............................................. 25 3 CRITIQUE OF COMPANY’S DEFINITION OF DECISION PROBLEM ............................... 27 3.1 Population ............................................................................................................................. 31 3.2 Intervention ........................................................................................................................... 32 3.3 Comparators .......................................................................................................................... 32 3.4 Outcomes .............................................................................................................................. 33 3.5 Other relevant factors ............................................................................................................ 39 4 CLINICAL EFFECTIVENESS ................................................................................................... 40 4.1 Critique of the methods of review(s) .................................................................................... 40 4.2 Critique of trials of the technology of interest, their analysis and interpretation (and any standard meta-analyses of these) ........................................................................................... 51 4.3 Subgroup analyses ................................................................................................................ 93 4.4 Non-randomised and non-controlled evidence ..................................................................... 97 4.5 Safety evidence ................................................................................................................... 106 4.6 Critique of trials identified and included in the indirect comparison and/or multiple treatment comparison .......................................................................................................... 115 4.7 Critique of the NMA ........................................................................................................... 134 4.8 Additional work on clinical effectiveness undertaken by the ERG .................................... 145 4.9 Conclusions of the clinical efficacy section ........................................................................ 149 5 COST EFFECTIVENESS .......................................................................................................... 158 5.1 ERG comment on company’s review of cost-effectiveness evidence ................................ 158 5.2 Summary and critique of company’s submitted economic evaluation by the ERG ............ 162 5.3 Exploratory and sensitivity analyses undertaken by the ERG ............................................ 220 5.4 Conclusions of the cost-effectiveness section ..................................................................... 222 6. IMPACT ON THE ICER OF EXPLORATORY ANALYSES UNDERTAKEN BY THE ERG .. .................................................................................................................................................... 225 7 END OF LIFE ............................................................................................................................ 233 8 OVERALL CONCLUSIONS .................................................................................................... 234 8.1 Implications for research ..................................................................................................... 237 9 REFERENCES ........................................................................................................................... 238 10. APPENDICES .......................................................................................................................
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