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Idelalisib for Treating Refractory Follicular Lymphoma

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Idelalisib for Treating Refractory Follicular Lymphoma CONFIDENTIAL UNTIL PUBLISHED in collaboration with: Idelalisib for treating refractory follicular lymphoma Produced by Kleijnen Systematic Reviews Ltd. in collaboration with Erasmus University Rotterdam (EUR) and Maastricht University Authors Rob Riemsma, Reviews Manager, Kleijnen Systematic Reviews Ltd, UK Nasuh Büyükkaramikli, Health Economics Researcher, Erasmus School of Health Policy & Management, EUR, the Netherlands Isaac Corro Ramos, Health Economics Researcher, EUR Stephanie Swift, Systematic Reviewer, KSR Ltd Steve Ryder, Health Economist, KSR Ltd Nigel Armstrong, Health Economist, KSR Ltd Marscha Holleman, Health Economics Researcher, EUR Gimon de Graaf, Health Economics Researcher, EUR Gill Worthy, Statistician, KSR Ltd Caro Noake, Information Specialist, KSR Ltd Maiwenn Al, Health Economics Researcher, EUR Jos Kleijnen, Director, KSR Ltd, Professor of Systematic Reviews in Health Care, Maastricht University Correspondence to Rob Riemsma, Kleijnen Systematic Reviews Unit 6, Escrick Business Park Riccall Road, Escrick York, UK YO19 6FD Date completed 07/08/2018 1 Copyright 2018 Queen's Printer and Controller of HMSO. All rights reserved. CONFIDENTIAL UNTIL PUBLISHED Source of funding: This report was commissioned by the NIHR HTA Programme as project number STA 14/12/01. Declared competing interests of the authors None. Acknowledgements None. Commercial in confidence (CiC) data are redacted throughout the report. Academic in confidence (AiC) data are redacted throughout the report. Copyright belongs to Kleijnen Systematic Reviews Ltd. 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: Riemsma R, Büyükkaramikli N, Corro Ramos I, Swift SL, Ryder S, Armstrong N, Holleman M, De Graaf G, Worthy G, Noake C, Al M, Kleijnen J. Idelalisib for treating refractory follicular lymphoma: a Single Technology Assessment. York: Kleijnen Systematic Reviews Ltd, 2018. Contributions of authors Rob Riemsma acted as project lead and systematic reviewer on this assessment, critiqued the clinical effectiveness methods and evidence and contributed to the writing of the report. Nasuh Büyükkaramikli acted as health economic project lead, critiqued the company’s economic evaluation and contributed to the writing of the report. Isaac Corro Ramos, Steve Ryder, Nigel Armstrong, Marscha Holleman and Gimon de Graaf acted as health economists on this assessment, critiqued the company’s economic evaluation and contributed to the writing of the report. Stephanie Swift acted as systematic reviewer, critiqued the clinical effectiveness methods and evidence and contributed to the writing of the report. Gill Worthy acted as statistician, critiqued the analyses in the company’s submission and contributed to the writing of the report. Caro Noake critiqued the search methods in the submission and contributed to the writing of the report. Maiwenn Al acted as health economist on this assessment, critiqued the company’s economic evaluation, contributed to the writing of the report and provided general guidance. Jos Kleijnen critiqued the company’s definition of the decision problem and their description of the underlying health problem and current service provision, contributed to the writing of the report and supervised the project. 2 Copyright 2018 Queen's Printer and Controller of HMSO. All rights reserved. CONFIDENTIAL UNTIL PUBLISHED Abbreviations AE Adverse events AIC Akaike information criterion ALT Alanine aminotransferase ANC Absolute neutrophil count ASCO American Society of Clinical Oncology ASCT Autologous stem cell transplantation AST Aspartate aminotransferase AWMSG All Wales Medicines Strategy Group bd/b.i.d Twice daily BI budget impact BIC Bayesian information criterion BSA body surface area BSC Best supportive care CDF Cancer Drugs Fund CE Cost effectiveness CEA Cost effectiveness analysis CEAC Cost effectiveness acceptability curve CHMP Committee for Medicinal Products for Human Use CI Confidence interval CLL Chronic lymphocytic leukaemia CMV Cytomegalovirus CR Complete response CRD Centre for Reviews and Dissemination CrI Credible interval CS Company’s submission CSR Clinical study report CHMP Committee for Medicinal Products for Human Use CTCAE Common Terminology Criteria for Adverse Events (National Cancer Institute) CUP Compassionate use programme DBL Database lock DHAP Dexamethasone, cytarabine, cisplatin DOR Duration of response DSU Decision Support Unit EAP Early access programme ECOG Eastern Cooperative Oncology Group EMA European Medicines Agency EPAR European public assessment report EQ-5D European quality of life-5 dimensions ERG Evidence Review Group EUR Erasmus University Rotterdam FACT-Lym Functional assessment of cancer therapy: lymphoma FDA Food and Drug Administration FL Follicular lymphoma FLIPI Follicular lymphoma international prognostic index HMRN Haematological Malignancy Research Network HR Hazard ratio HRQL Health-related quality of life HTA Health technology assessment ICER Incremental cost effectiveness ratio iNHL Indolent non-Hodgkin’s lymphoma IPD Individual patient data IRC Independent review committee ITC Indirect treatment comparison 3 Copyright 2018 Queen's Printer and Controller of HMSO. All rights reserved. CONFIDENTIAL UNTIL PUBLISHED ITT Intention to treat KM Kaplan–Meier KSR Kleijnen Systematic Reviews LDH Lactate dehydrogenase MAIC Matching-adjusted indirect comparison MeSH Medical subject headings MHRA Medicines and Healthcare Products Regulatory Agency NA Not applicable NCPE National Centre for Pharmacoeconomics NHS National Health Services NICE National Institute for Health and Care Excellence NIHR National Institute for Health Research ORR Overall response rate OS Overall survival PCR Polymerase chain reaction PFS Progression-free survival PJP Pneumocystis jirovecii pneumonia PPS Post-progression survival PR Partial response PRESS Peer review of electronic search strategies PSA Probabilistic sensitivity analyses QALY(s) Quality-adjusted life year(s) QoL Quality of life R-CHOP Rituximab, cyclophosphamide, doxorubicin, vincristine, prednisolone R-CVP Rituximab, cyclophosphamide, vincristine, prednisone RCT Randomised controlled trial RS Relative survival RWE Real world evidence SAE Serious adverse events ScHARR School of Health and Related Research SCT Stem cell transplantation SHTAC Southampton Health Technology Assessments Centre SIGN Scottish Intercollegiate Guidelines Network SMC Scottish Medicines Consortium SmPC Summary of product characteristics SOC Standard of care STA Single technology appraisal ToT Time on treatment TSD Technical support document TTNT Time to next treatment TTP Time to progression TTR Time to response UK United Kingdom WHO World Health Organisation 4 Copyright 2018 Queen's Printer and Controller of HMSO. All rights reserved. CONFIDENTIAL UNTIL PUBLISHED Table of Contents Abbreviations .................................................................................................................................... 3 Table of Tables .................................................................................................................................. 7 Table of Figures ................................................................................................................................ 9 1. SUMMARY ................................................................................................................................ 10 1.1 Critique of the decision problem in the company’s submission .......................................... 10 1.2 Summary of clinical effectiveness evidence submitted by the company ............................ 10 1.3 Summary of the ERG’s critique of clinical effectiveness evidence submitted .................... 11 1.4 Summary of cost effectiveness evidence submitted by the company .................................. 12 1.5 Summary of the ERG’s critique of cost effectiveness evidence submitted ......................... 14 1.6 ERG commentary on the robustness of evidence submitted by the company ..................... 16 1.6.1 Strengths ....................................................................................................................... 16 1.6.2 Weaknesses and areas of uncertainty ............................................................................ 16 1.7 Summary of exploratory and sensitivity analyses undertaken by the ERG ......................... 17 2. BACKGROUND......................................................................................................................... 19 2.1 Critique of company’s description of underlying health problem. ...................................... 19 2.2 Critique of company’s overview of current service provision ............................................ 20 3. CRITIQUE OF COMPANY’S DEFINITION OF DECISION PROBLEM ........................ 22 3.1 Population ............................................................................................................................ 24 3.2 Intervention .......................................................................................................................... 24 3.3 Comparators ........................................................................................................................ 25 3.4 Outcomes ............................................................................................................................
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