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Topotecan, pegylated liposomal doxorubicin hydrochloride and paclitaxel for second-line or subsequent treatment of advanced ovarian cancer (report contains no commercial in confidence data) Produced by Centre for Reviews and Dissemination, University of York Authors Ms Caroline Main, Research Fellow, Systematic Reviews, Centre for Reviews and Dissemination, University of York, YO10 5DD Ms Laura Ginnelly, Research Fellow, Health Economics, Centre for Health Economics, University of York, YO10 5DD Ms Susan Griffin, Research Fellow, Health Economics, Centre for Health Economics, University of York, YO10 5DD Dr Gill Norman, Research Fellow, Systematic Reviews, Centre for Reviews and Dissemination, University of York, YO10 5DD Mr Marco Barbieri, Research Fellow, Health Economics, The Economic and Health Research Centre, Universitat Pompeu Fabra, Barcelona, Spain Ms Lisa Mather, Information Officer, Centre for Reviews and Dissemination, University of York, YO10 5DD Dr Dan Stark, Senior Lecturer in Oncology and Honorary Consultant in Medical Oncology, Department of Oncology, Bradford Royal Infirmary Mr Stephen Palmer, Senior Research Fellow, Health Economics, Centre for Health Economics, University of York, YO10 5DD Dr Rob Riemsma, Reviews Manager, Systematic Reviews, Centre for Reviews and Dissemination, University of York, YO10 5DD Correspondence to Caroline Main, Centre for Reviews and Dissemination, University of York, YO10 5DD, Tel: (01904) 321055, Fax: (01904) 321041, E-mail: [email protected] Date completed September 2004 Expiry date September 2006 Contributions of authors Caroline Main Lead reviewer responsible for writing the protocol, study selection, data extraction, validity assessment and writing the final report. Laura Ginnelly Involved in the cost-effectiveness section, writing the protocol, study selection, data extraction, development of the economic model and report writing. Susan Griffin Involved in the cost-effectiveness section, writing the protocol, study selection, data extraction, development of the economic model and report writing. Gill Norman Second reviewer involved in the clinical-effectiveness section, involved in study selection, data extraction, validity assessment and writing the final report. Marco Barbieri Involved in the cost-effectiveness section, responsible for the review and re-analysis of the company submissions. Lisa Mather Devised the search strategy and carried out the literature searches; wrote the search methodology sections of the report. Dan Stark Provided input at all stages, commented on various drafts of the report and contributed to the discussion section of the report. Stephen Provided input at all stages, commented on various drafts of the Palmer report; overall responsibility for the cost-effectiveness section of the report. Rob Riemsma Provided input at all stages, commented on various drafts of the report; overall responsibility for the clinical-effectiveness section of the report. Conflicts of interest: Colleagues at the Centre for Health Economics (not involved in this review) have received research funding from GlaxoSmithKline and Schering-Plough. Source of funding: This report was commissioned by the NHS R&D HTA Programme. Relationship of reviewer(s) with sponsor: None Acknowledgements We thank the expert advisory panel for their useful advice and constructive comments on the report. The views expressed in this report are those of the authors and not necessarily those of the NHS R&D Programme. We also wish to thank Dr Tony Ades of the MRC Health Services Research Collobaration at the University of Bristol for his help and advice on the use of the mixed comparison model applied in the economic analysis. Figure 7: Reproduced from W. ten Bokkel Huiink, S.R. Lane & G.A. Ross on behalf of the International Topotecan Study Group. Long term survival in a phase III, randomised study of topotecan versus paclitaxel in advance epithelial ovarian carcinoma. Annals of Oncology 15: 100-102, 2004, page 101 with permission of Oxford University Press. 2 TABLE OF CONTENTS EXECUTIVE SUMMARY. 9 1. AIM OF THE REVIEW 27 2. BACKGROUND 27 2.1. Description of underlying health problem 27 2.2. Current service provision 29 2.3. Description of new intervention 30 3. METHODS 36 3.1. Search strategy 36 3.2. Inclusion and exclusion criteria 38 3.3. Data extraction strategy 40 3.4. Quality assessment strategy 41 3.5. Methods of analysis/synthesis 41 3.5.1. Clinical effectiveness 41 3.5.2. Cost Effectiveness 42 3.5.3. Handling the company submissions 42 4. RESULTS 43 4.1.1. Quantity and quality of research available 43 4.1.2 Description of included studies 49 4.2 Assessment of clinical effectiveness 67 4.2.1. Effectiveness of PLDH versus Topotecan 67 4.2.2. Effectiveness of topotecan versus paclitaxel 87 4.2.3. Effectiveness of PLDH versus paclitaxel 100 4.2.4. Effectiveness of single agent paclitaxel compared to a combination of cyclophosphamide, doxorubicin and cisplatin 107 4.2.5. Effectiveness of paclitaxel in combination with platinum-based chemotherapy versus platinum-based therapy alone 113 4.2.6. Effectiveness of single-agent paclitaxel versus oxaliplatin 120 4.2.7. Effectiveness of paclitaxel given weekly versus every three weeks 124 4.2.8. Effectiveness of paclitaxel 175 mg/m2 compared to 250 mg/ m2 128 4.2.9. Effectiveness of oral versus intravenous topotecan 133 4.3. Other Related Systematic Reviews 138 4.4. Assessment of Economic Evaluations 139 4.4.1. Introduction 139 4.4.2. Review of Smith 139 4.4.3. Review of Ojeda 146 4.4.4. Review of Capri 149 4.4.5. Review of Prasad 152 4.4.6. Review of GlaxoSmithKline submission 156 4.4.7. Review of Schering-Plough Ltd submission 163 4.4.8. Review of Bristol-Myers Squibb Ltd submission 169 4.5. Summary of cost-effectiveness 172 4.6. Summary of findings from the cost-effectiveness review 176 5. ECONOMIC MODEL 178 5.1. Introduction 178 5.2. Methods 178 5.2.1. Analysis 1: Base case model 185 3 5.2.2. Analysis 2: Platinum-sensitive model 190 5.3. Model inputs 191 5.4. Analytic methods 204 5.5. Results 205 5.5.1. Analysis 1: Base case 206 5.5.2. Analysis 2: 213 5.6. Budget impact analysis 216 5.7. Economic model conclusions 218 6. DISCUSSION 221 6.1 Clinical effectiveness 221 6.2 Cost-effectiveness 234 7. CONCLUSIONS 237 7.1 Clinical effectiveness 237 7.2 Cost-effectiveness 239 8. REFERENCES 241 9. APPPENDICES 252 Appendix 1. Details about FIGO cancer staging 252 Appendix 2 Summary of current manufacturers information provided for health professionals 253 Appendix 3. Search strategy 258 Appendix 4. Excluded studies 265 Appendix 5. Details of quality assessment 269 Appendix 6. Data extraction tables 272 Appendix 7. Trials only available in abstract form 350 Appendix 8. Additional tables for the review of cost-effectiveness 354 Appendix 9. Quality of life searches 380 Appendix 10 WinBUGS code 389 Appendix 11. Additional tables for the economic model 408 TABLES Table 1. Summary of included RCTs 46 Table 2. Summary of the comparators included in the review of clinical effectiveness 48 Table 3. Quality checklist for included trials 66 Table 4. Summary of overall survival data based on long-term follow-up for PLDH versus topotecan. 67 Table 5. Summary of overall survival data based on long-term follow-up for PLDH versus topotecan – platinum-sensitive disease subgroup 69 Table 6. Summary of overall survival data based on long-term follow-up for PLDH versus topotecan – platinum-refractory disease subgroup 70 4 Table 7. Overall survival for sub-groups according to baseline disease characteristics (multivariate Cox regression analysis) 71 Table 8 Summary of progression-free survival data based on long-term follow-up for PLDH versus topotecan 72 Table 9. Summary of objective response rates based on long-term follow-up for PLDH versus topotecan 73 Table 10. Percentage of patients with a maintained or improved QoL score at 12 weeks follow-up for PLDH versus topotecan. 76 Table 11. Time (in months) spend in the three health states and their differences 78 Table 12. Treatment emergent adverse events in a least 10% of participants by preferred term: All grades, Grade 3, and Grade 4. 81 Table 13. Response rate by treatment group for topotecan versus paclitaxel 89 Table 14. Response in relation to platinum sensitivity for topotecan versus paclitaxel 90 Table 15. Response in relation to baseline disease status for topotecan versus paclitaxel 90 Table 16 Response duration for participants treated with topotecan or paclitaxel 91 Table 17. Time to response data for participants treated with topotecan or paclitaxel 91 Table 18. Number and percentage of patients and courses with haematological toxicity by worst CTC Grade. 93 Table 19. Related or possibly related non-haematological toxicities occurring in more than 10% of patients treated with topotecan or paclitaxel 94 Table 20. Response to Topotecan (third-line) treatment according to response to paclitaxel (second-line) 96 Table 21. Response to Paclitaxel (third-line) treatment according to response to topotecan (second-line) treatment 96 Table 22. Haematological toxicity during cross-over (third-line) treatment with topotecan versus paclitaxel 97 Table 23. Non-haematologic Toxicity to Cross-over (third-line) treatment for topotecan versus paclitaxel 98 Table 24. Summary of overall survival data for all participants for PLDH versus paclitaxel 100 Table 25. Summary of overall survival data based on PLDH versus paclitaxel – platinum-sensitive disease subgroup 101 Table 26. Summary of overall survival data based on PLDH versus paclitaxel – platinum-resistant disease subgroup 101 Table 27. Treatment emergent adverse events in a least 10% of participants by preferred term: All grades, Grade 3, and Grade 4. 103 Table 28. Response to CAP compared to paclitaxel treatment. 108 Table 29. The maximum grade toxicities during treatment with paclitaxel compared with CAP 111 5 Table 30. The proportion of patients in each treatment group who experienced moderate or severe toxic effects during treatment along with the RR and 95% CI 118 Table 31.
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  • Ep 2569287 B1

    Ep 2569287 B1

    (19) TZZ _T (11) EP 2 569 287 B1 (12) EUROPEAN PATENT SPECIFICATION (45) Date of publication and mention (51) Int Cl.: of the grant of the patent: C07D 413/04 (2006.01) C07D 239/46 (2006.01) 09.07.2014 Bulletin 2014/28 (86) International application number: (21) Application number: 11731562.2 PCT/US2011/036245 (22) Date of filing: 12.05.2011 (87) International publication number: WO 2011/143425 (17.11.2011 Gazette 2011/46) (54) COMPOUNDS USEFUL AS INHIBITORS OF ATR KINASE VERBINDUNGEN ALS HEMMER DER ATR-KINASE COMPOSÉS UTILISABLES EN TANT QU’INHIBITEURS DE LA KINASE ATR (84) Designated Contracting States: • VIRANI, Aniza, Nizarali AL AT BE BG CH CY CZ DE DK EE ES FI FR GB Abingdon GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO Oxfordshire OX144RY (GB) PL PT RO RS SE SI SK SM TR • REAPER, Philip, Michael Abingdon (30) Priority: 12.05.2010 US 333869 P Oxfordshire OX144RY (GB) (43) Date of publication of application: (74) Representative: Coles, Andrea Birgit et al 20.03.2013 Bulletin 2013/12 Kilburn & Strode LLP 20 Red Lion Street (73) Proprietor: Vertex Pharmaceuticals Inc. London WC1R 4PJ (GB) Boston, MA 02210 (US) (56) References cited: (72) Inventors: WO-A1-2010/054398 WO-A1-2010/071837 • CHARRIER, Jean-Damien Abingdon • C. A. HALL-JACKSON: "ATR is a caffeine- Oxfordshire OX144RY (GB) sensitive, DNA-activated protein kinase with a • DURRANT, Steven, John substrate specificity distinct from DNA-PK", Abingdon ONCOGENE, vol. 18, 1999, pages 6707-6713, Oxfordshire OX144RY (GB) XP002665425, cited in the application • KNEGTEL, Ronald, Marcellus Alphonsus Abingdon Oxfordshire OX144RY (GB) Note: Within nine months of the publication of the mention of the grant of the European patent in the European Patent Bulletin, any person may give notice to the European Patent Office of opposition to that patent, in accordance with the Implementing Regulations.