Benefits and costs of innovative oncology drugs in Belgium (2004-2017) [printed text] / Mattias Neyt  , Author ; Carl Devos , Author ; Nancy Thiry, Author ; Cindy De Gendt, Author ; Nancy Van Damme, Author ; Diego Castanares-Zapatero , Author ; Nicolas Fairon , Author ; Frank Hulstaert, Author ; Leen Verleye, Author ; Geert Silversmit, Author . - Brussels [Belgium] : KCE = Federaal Kenniscentrum voor de Gezondheidszorg = Centre Fédéral d'Expertise des Soins de Santé = Belgian Health Care Knowledge Centre, 2021 . - 346 p. : ill., ; A4. - ( KCE Reports. Health Technology Assessment (HTA); 343) . ISSN : D/2021/10.273/23 : € 0,00 Languages : English ( eng)
| Descriptors: |
Indexation
2018-02 ; Antineoplastic Agents ; Neoplasms ; R343 ; Randomized Controlled Trials ; Review ; Technology Assessment, Biomedical
Classification
QV 269 Antineoplastic agents. Antineoplastic antibiotics
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| Contents note: |
SCIENTIFIC REPORT 22 -- 1 INTRODUCTION 22 -- 1.1 SETTING THE SCENE 22 -- 1.1.1 Increasing expenditures for oncology drugs 22 -- 1.1.2 The increasing cancer drug prices 23 -- 1.1.3 Patient benefit, no longer a hard requirement for marketing authorisation 24 -- 1.2 ARE THE EXTRA EXPENDITURES ASSOCIATED WITH PATIENT BENEFITS? 26 -- 1.2.1 Cancer drugs as one of many contributors to cancer survival 26 -- 1.2.2 Small and uncertain patient benefit of new cancer medicines 27 -- 2 OBJECTIVES 28 -- 2.1 OPPORTUNITY COST OR DISPLACEMENT EFFECT 28 -- 2.2 RESEARCH QUESTIONS 28 -- 3 METHODOLOGY 29 -- 3.1 PART 1: DATABASES, STATISTICAL ANALYSES, AND PRESENTATION OF RESULTS 29 -- 3.1.1 Belgian Cancer Registry (BCR) data selection and linkage with Intermutualistic Agency (IMA) data and vital status 29 -- 3.1.1.1 Selection of the study population in the Belgian Cancer Registry database 29 -- 3.1.1.2 Linkage with health insurance data 32 -- 3.1.1.3 Vital status 32 -- 3.1.2 Statistical analyses 33 -- 3.1.2.1 Descriptive analyses 33 -- 3.1.2.2 Survival analysis 33 -- 3.1.2.3 Statistical software 34 -- 3.1.3 Graphics and Tables 34 -- 3.2 PART 2: SELECTION OF DRUGS 35 -- 3.2.1 Selection criteria and process 35 -- 3.2.2 Selected cancer types and drugs 37 -- 3.3 PART 3: LITERATURE SEARCHES 37 -- 3.3.1 Search medical literature 37 -- 3.3.2 Search economic literature 38 -- 3.4 PART 4: RIZIV REIMBURSEMENT CRITERIA 39 -- 4 RESULTS PER CANCER TYPE 40 -- 4.1 BREAST CANCER 40 -- 4.1.1 Introduction 40 -- 4.1.2 Observational data 42 -- 4.1.2.1 Patient characteristics, survival and expenditures 42 -- 4.1.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 47 -- 4.1.3 Medical literature: efficacy 48 -- 4.1.3.1 HER2(+) Breast cancer: neo-adjuvant and adjuvant setting (pertuzumab) 48 -- 4.1.3.2 HER2(+) Breast cancer: metastatic disease or locally advanced disease – first line (pertuzumab) 49 -- 4.1.3.3 HER2(+) Breast cancer: metastatic disease or locally advanced disease – second line (pertuzumab, trastuzumab emtansine) 49 -- 4.1.3.4 HER2(-) Breast cancer: metastatic disease or locally advanced disease 50 -- 4.1.3.5 Discussion 50 -- 4.1.4 Economic literature: cost-effectiveness 51 -- 4.1.5 Summary 57 -- 4.2 CHRONIC MYELOID LEUKAEMIA 59 -- 4.2.1 Introduction 59 -- 4.2.2 Observational data 60 -- 4.2.2.1 Patient characteristics, survival and expenditures 60 -- 4.2.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 67 -- 4.2.3 Medical literature: efficacy 67 -- 4.2.3.1 First-line treatment 68 -- 4.2.3.2 Second- and third-line treatment 70 -- 4.2.3.3 Discussion 70 -- 4.2.4 Economic literature: cost-effectiveness 71 -- 4.2.5 Summary 76 -- 4.3 COLORECTAL CANCER 78 -- 4.3.1 Introduction 78 -- 4.3.2 Observational data 79 -- 4.3.2.1 Patient characteristics, survival and expenditures 79 -- 4.3.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 84 -- 4.3.3 Medical literature: efficacy 84 -- 4.3.3.1 First-line treatment 85 -- 4.3.3.2 Second-line (or later) treatment 86 -- 4.3.3.3 Discussion 87 -- 4.3.4 Economic literature: cost-effectiveness 87 -- 4.3.5 Summary 91 -- 4.4 HEAD AND NECK CANCER 93 -- 4.4.1 Introduction 93 -- 4.4.2 Observational data 94 -- 4.4.2.1 Patient characteristics, survival and expenditures 94 -- 4.4.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 100 -- 4.4.3 Medical literature: efficacy 100 -- 4.4.3.1 Discussion 102 -- 4.4.4 Economic literature: cost-effectiveness 103 -- 4.4.5 Summary 106 -- 4.5 MELANOMA 107 -- 4.5.1 Introduction 107 -- 4.5.2 Observational data 108 -- 4.5.2.1 Patient characteristics, survival and expenditures 108 -- 4.5.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 119 -- 4.5.3 Medical literature: efficacy 120 -- 4.5.3.1 BRAF and MEK inhibitors melanoma 121 -- 4.5.3.2 Immunotherapy in melanoma 122 -- 4.5.3.3 Discussion 123 -- 4.5.4 Economic literature: cost-effectiveness 124 -- 4.5.5 Summary 129 -- 4.6 MESOTHELIOMA 132 -- 4.6.1 Introduction 132 -- 4.6.2 Observational data 132 -- 4.6.2.1 Patient characteristics, survival and expenditures 132 -- 4.6.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 139 -- 4.6.3 Medical literature: efficacy 139 -- 4.6.3.1 Discussion 140 -- 4.6.4 Economic literature: cost-effectiveness 141 -- 4.6.5 Summary 142 -- 4.7 MULTIPLE MYELOMA 143 -- 4.7.1 Introduction 143 -- 4.7.2 Observational data 145 -- 4.7.2.1 Patient characteristics, survival and expenditures 145 -- 4.7.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 150 -- 4.7.3 Medical literature: efficacy 150 -- 4.7.3.1 Multiple Myeloma: First-line treatment in adult patients ineligible for stem-cell -- transplantation 151 -- 4.7.3.2 Multiple myeloma: first-line treatment in adult patients eligible for stem-cell -- transplant 152 -- 4.7.3.3 Multiple myeloma: treatment of relapsed or refractory disease 152 -- 4.7.3.4 Discussion 153 -- 4.7.4 Economic literature: cost-effectiveness 154 -- 4.7.5 Summary 158 -- 4.8 NON-HODGKIN LYMPHOMA 160 -- 4.8.1 Introduction 160 -- 4.8.2 Observational data 161 -- 4.8.2.1 Patient characteristics, survival and expenditures 161 -- 4.8.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 175 -- 4.8.3 Medical literature: efficacy 176 -- 4.8.3.1 CLL/SLL 176 -- 4.8.3.2 DLBCL 178 -- 4.8.3.3 Mantle cell lymphoma 179 -- 4.8.3.4 Discussion 181 -- 4.8.4 Economic literature: cost-effectiveness 183 -- 4.8.4.1 Chronic lymphocytic leukaemia 183 -- 4.8.4.2 Mantel-cell lymphoma 188 -- 4.8.5 Summary 190 -- 4.8.5.1 CLL/SLL 190 -- 4.8.5.2 DLBCL 191 -- 4.8.5.3 Mantle cell lymphoma 192 -- 4.9 NON-SMALL CELL LUNG CANCER 193 -- 4.9.1 Introduction 193 -- 4.9.2 Observational data 194 -- 4.9.2.1 Patient characteristics, survival and expenditures 194 -- 4.9.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 199 -- 4.9.3 Medical literature: efficacy 199 -- 4.9.3.1 Anti-EGFR therapy in NSCLC 200 -- 4.9.3.2 ALK inhibitors for non-small cell lung cancer 203 -- 4.9.3.3 Immunotherapy 203 -- 4.9.3.4 Discussion 205 -- 4.9.4 Economic literature: cost-effectiveness 205 -- 4.9.5 Summary 209 -- 4.10 OVARIAN CANCER 211 -- 4.10.1 Introduction 211 -- 4.10.2 Observational data 211 -- 4.10.2.1 Patient characteristics, survival and expenditures 211 -- 4.10.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 220 -- 4.10.3 Medical literature: efficacy 220 -- 4.10.3.1 Bevacizumab in ovarian cancer 221 -- 4.10.3.2 Discussion 222 -- 4.10.4 Economic literature: cost-effectiveness 222 -- 4.10.5 Summary 226 -- 4.11 PROSTATE CANCER 227 -- 4.11.1 Introduction 227 -- 4.11.2 Observational data 227 -- 4.11.2.1 Patient characteristics, survival and expenditures 227 -- 4.11.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 233 -- 4.11.3 Medical literature: efficacy 233 -- 4.11.3.1 Discussion 235 -- 4.11.4 Economic literature: cost-effectiveness 235 -- 4.11.5 Summary 238 -- 4.12 RENAL CELL CARCINOMA (RCC) 239 -- 4.12.1 Introduction 239 -- 4.12.2 Observational data 241 -- 4.12.2.1 Patient characteristics, survival and expenditures 241 -- 4.12.2.2 Drug uptake for a selection of drugs during the first 5 years after diagnosis 247 -- 4.12.3 Medical literature: efficacy 249 -- 4.12.3.1 Targeted therapy – first line treatment 250 -- 4.12.3.2 Targeted therapy – second line treatment 250 -- 4.12.3.3 Immunotherapy 251 -- 4.12.3.4 Discussion 252 -- 4.12.4 Economic literature: cost-effectiveness 253 -- 4.12.5 Summary 259 -- 5 USE OF ANTICANCER DRUGS AT THE END OF LIFE 261 -- 6 SUMMARY 266 -- 7 DISCUSSION 276 -- 7.1 REAL-WORLD OBSERVATIONAL DATA 276 -- 7.1.1 Data from the Belgian Cancer Registry (BCR) and Intermutualistic Agency (IMA) – strengths/limitations and nuance needed 276 -- 7.1.2 Limitations inherent to the use of observational data – no good estimate of treatment effect 280 -- 7.2 LIMITED BENEFITS IN OVERALL SURVIVAL 284 -- 7.3 QOL AS AN IMPORTANT ENDPOINT 287 -- 7.3.1 A patient-relevant endpoint in clinical trials 287 -- 7.3.2 Measuring QoL with a generic utility instrument 287 -- 7.3.3 Improvement needed in measuring and reporting QoL, and using the results in economic evaluations 288 -- 7.3.4 An example to show (the importance of) transparent reporting of QoL data 292 -- 7.4 THE USE OF PROGRESSION-FREE SURVIVAL AS A SURROGATE ENDPOINT 295 -- 7.4.1 Is PFS a valid surrogate for OS?. 295 -- 7.4.2 Can PFS be considered as a surrogate for quality of life? 296 -- 7.4.3 The problem of PFS in economic evaluations 297 -- 7.4.4 Do we try hard enough? Searching for a solution that fits both goals 298 -- 7.5 SUBSTANTIAL INCREASE IN GROSS EXPENDITURES, LIMITED BY REFUNDS AND TAXES 299 -- 7.5.1 Managed entry agreement refunds 299 -- 7.5.2 Taxes and clawback 300 -- 7.6 MANAGED ENTRY AGREEMENT 301 -- 7.6.1 Managed entry agreement – when the exception becomes the rule 301 -- 7.6.2 Incentives to demonstrate survival or QoL benefits for the patients 302 -- 7.6.2.1 Before marketing approval 302 -- 7.6.2.2 After marketing approval 304 -- 7.6.3 Confidential prices 307 -- 7.6.3.1 A short-term advantage creating long-term problems and making the system intransparent 307 -- 7.6.3.2 Confidential prices making economic evaluations and policy decisions intransparent 308 -- 7.7 WHY WE MAINLY RELIED ON UK DOSSIERS FOR INFORMATION ON ECONOMIC EVALUATIONS IN THIS REPORT 309 -- 7.8 THE IMPORTANCE OF THE (DISCOUNT FOR THE) COMPARATOR 311 -- 7.9 DIFFERENT ARGUMENTS USED FROM DIFFERENT PERSPECTIVES 313 -- REFERENCES 317 |
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https://doi.org/10.57598/R343C |
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PDF (11 Mb) |
| Record link: |
https://kce.docressources.info/index.php?lvl=notice_display&id=4747 |
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Authors' reply to Ward in BMJ (International), 347(2013) ([09/01/2013])
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