Addressing Challenges in Access to Oncology Medicines Analytical Report ADDRESSING CHALLENGES IN ACCESS TO ONCOLOGY MEDICINES Analytical Report 2 Acknowledgements This analytical report was prepared by the Health Division, OECD Directorate for Employment, Labour and Social Affairs. The OECD Health Division would like to acknowledge the valuable contributions from the OECD Expert Group on Pharmaceuticals and Medical Devices. Writing this report would not have been possible without the contribution of experts from the OECD and EU countries. The authors are also grateful to various members of the Expert Group for input to the research for this paper as well as review of prior drafts. This paper benefited from review and feedback by Ruth Lopert and Francesca Colombo at the OECD Directorate for Employment, Labour and Social Affairs. Prior versions of this paper were reviewed by colleagues at the European Commission. This document, as well as any data and map included herein, are without prejudice to the status of or sovereignty over any territory, to the delimitation of international frontiers and boundaries and to the name of any territory, city or area. The statistical data for Israel are supplied by and under the responsibility of the relevant Israeli authorities. The use of such data by the OECD is without prejudice to the status of the Golan Heights, East Jerusalem and Israeli settlements in the West Bank under the terms of international law. This document was produced with the financial assistance of the European Union under The Third Health Programme 2014-2020. The contents of this report are the sole responsibility of the OECD and can in no way be taken to reflect the views of the European Union. The opinions expressed and arguments employed herein do not necessarily reflect the official views of the OECD member countries or EU Member States. ADDRESSING CHALLENGES IN ACCESS TO ONCOLOGY MEDICINES © OECD 2020 3 Table of contents Acknowledgements 2 List of acronyms and abbreviations 5 Country abbreviations 7 Executive summary 8 1 Introduction 11 2 Country approaches to challenges in oncology medicines 14 2.1 Mitigating increasing uncertainty in the assessment of clinical benefit 14 2.2 Managing the ‘cascade’ of indications 21 2.3 Managing place in therapy and pricing combination regimens 25 2.4 Managing increasing treatment costs within budget constraints 30 3 Access to oncology medicines in OECD/EU countries 40 3.1 Access is multi-dimensional 40 3.2 Measuring access to oncology medicines 41 3.3 Access to oncology medicines remains unequal across OECD/EU countries 46 3.4 Time to access for new oncology products/indications varies across OECD/EU countries 50 3.5 Countries report inequities in access and difficulties in meeting patient expectations 58 3.6 Cost-sharing and medicine prices affect affordability 61 4 Conclusions 67 References 70 Annex A. OECD data collection 78 Expert interviews and survey 78 Workshop 82 Annex B. OECD access survey – limitations and country-specific caveats 83 Annex C. OECD access survey – indication-specific results 86 Annex D. OECD survey – cost sharing requirements 93 Tables Table 1.1. Summary of challenges identified in managing oncology medicines 12 Table 2.1. Pricing products with multiple indications in OECD/EU countries 22 Table 2.2. Price change likely to occur following approval and coverage of a new indication of an existing product in OECD/EU countries 23 ADDRESSING CHALLENGES IN ACCESS TO ONCOLOGY MEDICINES © OECD 2020 4 Table 2.3. Management of off-label use of oncology medicines in OECD/EU countries 25 Table 2.4. Approaches to attributing value to constituents of combination regimens in OECD/EU countries 29 Table 3.1. ESMO-MCBS Treatment Scores 43 Table 3.2. Time between first marketing authorisation and marketing authorisation in subsequent countries/regions for product/indications approved since 2014 (n=31 pairs) 51 Table 3.3. Time between first marketing authorisation and granting of coverage in subsequent OECD/EU countries for product/indications approved since 2014 (n=31 pairs) 53 Table 3.4. Level of coverage decision making across OECD/EU countries 59 Table A A.1. Responses to OECD survey 78 Table A A.2. Final product/indication sample for OECD access survey 80 Table A B.1. Country-specific caveats 83 Table A C.1. Regulatory approval and coverage status of medicines for metastatic breast cancer 87 Table A C.2. Regulatory approval and coverage status of medicines for non-small cell lung cancer 88 Table A C.3. Regulatory approval and coverage status of medicines for colorectal cancer 89 Table A C.4. Regulatory approval and coverage status of medicines for melanoma 90 Table A C.5. Regulatory approval and coverage status of medicines for multiple myeloma 91 Table A C.6. Regulatory approval and coverage status of medicines used as supportive care 92 Table A D.1. Patient cost-sharing requirements for oncology medicines and medical services potentially including the administration of an oncology medicine 93 Figures Figure 2.1. Policies and approaches to address uncertainty in coverage and/or pricing decisions across OECD/EU countries 20 Figure 2.2. Approaches to determining place in therapy of new drugs across OECD/EU countries 26 Figure 2.3. Setting prices of products used in combination regimens 28 Figure 2.4. Trends in the number of oncology medicines approved and their prices 31 Figure 2.5. Comparisons of sales and expenditures per capita for oncology medicines in Italy 33 Figure 2.6. Trends in pharmaceutical expenditure 34 Figure 2.7. The role of the Cancer Drugs Fund in the English National Health System 36 Figure 2.8. Percentage of products reported as no longer protected from competition, by number of generics/biosimilars available across OECD/EU countries 38 Figure 3.1. Time to access as measured in this study 46 Figure 3.2. Percentage of sample product/indication pairs by approval and coverage status across OECD/EU countries 47 Figure 3.3. Time between approval and coverage dates across OECD/EU countries 57 Figure 3.4. Cost-sharing for oncology pharmaceuticals dispensed to patients for self-administration in OECD/EU countries 63 Figure 3.5. Cost-sharing for outpatient specialist cancer care services including the administration of a medicine in OECD/EU countries 64 Figure 3.6. Cost-sharing for hospital inpatient care for cancer including the administration of a medicine in OECD/EU countries 64 Boxes Box 2.1. Medicines approved with pan-tumour indications 17 Box 2.2. Existing international collaboration initiatives 21 Box 2.3. Criteria used to determine eligibility for funding through Italy’s Fund for Innovative Medicines in Oncology 36 Box 3.1. ESMO – Magnitude of Clinical Benefit Scale (ESMO-MCBS) 43 Box 3.2. Types of user charges or cost-sharing requirements - definitions 62 ADDRESSING CHALLENGES IN ACCESS TO ONCOLOGY MEDICINES © OECD 2020 5 List of acronyms and abbreviations ABPI Association of the British Pharmaceutical Industry AIFA Italian Medicines Agency ASCO American Society of Clinical Oncology ASP Average sales price ATC Anatomical Therapeutic Chemical classification ATMP Advanced therapy medicinal products CADTH Canadian Agency for Drugs and Technologies in Health CAR-T Chimeric antigen receptor T-cell therapy CDF Cancer Drugs Fund (England) CHMP Committee for Medicinal Products for Human Use CMS Centers for Medicare & Medicaid Services (United States) CTLA-4 Cytotoxic T-lymphocyte-associated protein 4 DDD Defined daily dose DHSC Department of Health & Social Care (England) dMMR Deficient mismatch repair DRG Diagnosis related group EBP Evidence Building Program (Canada) ECL European Cancer Leagues EEA European Economic Area EFPIA European Federation of Pharmaceutical Industries and Associations EGFR Epidermal growth factor receptor EMA European Medicines Agency EML Essential Medicines List (i.e. 21st WHO Model List of Essential Medicines) EOPYY National Organization for the Provision of Health Services (Greece) ESMO European Society for Medical Oncology ESMO-MCBS European Society for Medical Oncology Magnitude of Clinical Benefit Scale EU European Union EUnetHTA European Network for Health Technology Assessment Euripid European Integrated Price Information Database FDA Food and Drug Administration (United States) GDP Gross domestic product GP General practitioner ADDRESSING CHALLENGES IN ACCESS TO ONCOLOGY MEDICINES © OECD 2020 6 GRADE Grading of Recommendations Assessment, Development and Evaluation HAS Haute Autorité de Santé (France) HER-2 Human epidermal growth factor receptor 2 HIV Human immunodeficiency virus HTA Health Technology Assessment HTAi Health Technology Assessment International IBP Indication-based pricing INCa French National Cancer Institute MEA Managed entry agreement MfE Medicines for Europe MOH Ministry of Health MSI-H Microsatellite instability-high NHS National Health Service NHSE-I National Health Service England & Improvement NICE National Institute for Health and Care Excellence NoMA Norwegian Medicines Agency NTRK Neurotrophic Tyrosine Receptor Kinase OOP Out-of-pocket payments PBAC Pharmaceutical Benefits Advisory Committee (Australia) PBS Pharmaceutical Benefits Scheme (Australia) pCODR pan-Canadian Oncology Drug Review PD-1 Programmed cell death protein 1 PD-L1 Programmed cell death ligand 1 PMPRB Patented Medicine Prices Review Board PPRI Pharmaceutical Pricing and Reimbursement Information network QALY Quality-adjusted life year RCT Randomised controlled trial RWD Real world data TKI Tyrosine kinase inhibitor TLV Dental and Pharmaceutical Benefits Agency (Sweden) WHO World Health Organization WHOCC WHO Collaborating Centre for Drug Statistics Methodology WTP Willingness to pay ADDRESSING CHALLENGES IN ACCESS TO ONCOLOGY MEDICINES © OECD 2020 7 Country abbreviations AUS Australia BEL Belgium CAN Canada CHE Switzerland CHL Chile CYP Cyprus1 CZE Czech Republic DEU Germany DNK Denmark EST Estonia FRA France GBR United Kingdom (England only) GRC Greece HUN Hungary IRL Ireland ISR Israel ITA Italy JPN Japan KOR Korea LTU Lithuania LVA Latvia MLT Malta NOR Norway SWE Sweden USA United States 1 Note by Turkey: The information in this document with reference to “Cyprus” relates to the southern part of the Island.