Background Expanding the indication of already approved immuno-oncology drugs presents treatment opportunities for patients but also strains healthcare systems. Cost-based pricing models are discussed as a possibility for cost containment. This study focuses on two drugs, pembrolizumab (Keytruda) and daratumumab (Darzalex), to explore the potential effect of indication broadening on the estimated price when using the cost-based pricing (CBP) model proposed by Uyl-de Groot and Löwenberg (2018). Methods The model was used to calculate cumulative yearly prices, cumulative prices per indication, and non-cumulative indication-based prices using inputs such as research and development (R&D) costs, manufacturing costs, eligible patient population, and a profit margin. A deterministic stepwise analysis and scenario analysis were conducted to examine how sensitive the estimated price is to the different input assumptions. Results The yearly cumulative cost-based prices (CBPs) ranged from €52 to €885 for pembrolizumab per vial and €823 to €31,941 for daratumumab per vial. Prices were higher in initial years or indications due to smaller patient populations, decreased over time or after additional indications. Sensitivity analysis showed that the number of eligible patients had the most significant impact on the estimated price. In the scenario analysis the profit margin contributed most to a higher CBPs for both drugs. Lower estimates resulted from assumed lower R&D costs. Discussion The estimated CBPs are consistently lower than Dutch list prices for pembrolizumab (€2,861), mainly resulting from larger patient populations in registered indications. However, daratumumab’s list prices fall within the range of modeled CBPs depending on the year or indication (€4,766). Both CBPs decrease over time or with additional indications. The number of eligible patients and initial R&D costs have the most significant influence on the CBPs. These findings contribute to the ongoing discussions on pharmaceutical pricing, especially concerning cancer drugs with expanding indications.
There is little evidence on the costs associated with the route of administration of oncology drugs. We investigated time and resource use for hospitals and patients and compared healthcare and societal costs for intravenous (IV) and subcutaneous (SC) administration of trastuzumab and rituximab. Data for the preparation and administration of both drugs were collected at the hospital pharmacy and at the oncology day care unit. Patients completed a questionnaire for obtaining information on societal costs (productivity losses, informal care and traveling expenses). A total of 126 patients were recruited in six hospitals; 82 received trastuzumab (37 IV and 45 SC) and 44 received rituximab (23 IV and 21 SC). The costs per administration (including societal cost but excluding drug costs) were &OV0556;167 and &OV0556;264 for IV and &OV0556;76 and &OV0556;146 for SC trastuzumab and rituximab, respectively. The costs for SC administration were lower in all categories. The largest cost component was related to time spent at the day care unit (overhead costs). This resulted in savings of &OV0556;47 for SC trastuzumab and &OV0556;69 for SC rituximab. The costs related to time of healthcare professionals was &OV0556;9 lower for both drugs. The costs for consumables resulted in another &OV0556;12 savings. Societal costs were &OV0556;22 lower for SC trastuzumab and &OV0556;28 lower for SC rituximab. Although administration costs are relatively a small part of the total costs, important savings can be generated by switching to an SC route of administration especially because a large number of patients receive oncology drugs and patients receive more than one administration.
Ten behoeve van de besluitvorming over opname van geneesmiddelen
in het basispakket worden in veel landen bevindingen
van economische evaluatiestudies gebruikt. Een
betere internationale afstemming van de assessment kan
een positief effect hebben op de kwaliteit van de besluitvorming.
Internationale kosteneffectiviteitsmodellen kunnen
hierbij een rol spelen, maar het generaliseren van farmacoeconomisch
bewijs van et ene naar het andere land is niet
eenvoudig. Echter, appraisal en besluitvorming zullen een
nationale aangelegenheid blijven.
markdownabstract__Abstract__
Life expectancy is increasing in the Organisation for Economic Co-operation and Development
(OECD) countries. Simultaneously, however, many countries have experienced
rising health care expenditure, albeit at a reduced rate during the last years due to the
global economic recession. For instance, health care expenditure in The Netherlands
has increased from 6.5 billion to 46.9 billion and 94.2 billion euro in 1972, 2000 and 2013,
respectively.4 More apprehensively, not only absolute expenditure but also the relative
share of Gross Domestic Product (GDP) spent on health care has increased across OECD
countries. For example, the Dutch share of GDP spent on health care increased from
8.7%, to 11.2%, and 15.6% in 1972, 2000, and 2013, respectively.
LIST OF ABBREVIATIONS 3 -- 1 INTRODUCTION 5 -- 1.1 BACKGROUND 5 -- 1.2 OBJECTIVES 6 -- 1.3 METHODS AND ANALYTICAL FRAMEWORKS 6 -- 1.3.1 The description of the drug reimbursement systems 6 -- 1.3.2 Assessing accountability for reasonableness 8 -- 1.4 KEY CONCEPTS 9 -- 1.5 STRUCTURE OF THE REPORT 10 -- 2 COUNTRY COMPARISON 12 -- 2.1 CONTEXTUAL BACKGROUND 12 -- 2.1.1 Health care system characteristics 12 -- 2.1.2 Health care system and policy objectives 12 -- 2.1.3 Health care funding and pharmaceutical expenditure 13 -- 2.1.4 Pharmaceutical policies 15 -- 2.2 POLICY IMPLEMENTATION LEVEL 18 -- 2.2.1 Objectives of the drug reimbursement system. 18 -- 2.2.2 Establishment of the system 19 -- 2.2.3 Implementation: a positive reimbursement list 24 -- 2.2.4 Accountability: impact assessment 24 -- 2.3 TECHNOLOGY DECISION LEVEL 26 -- 2.3.1 Assessment and appraisal phase 26 -- 2.3.2 Decision making phase 32 -- 2.3.3 Output and Implementation 36 -- 3 DISCUSSION 39 -- 3.1 ACCOUNTABILITY FOR REASONABLENESS 39 -- 3.1.1 Transparency 40 -- 3.1.2 Relevance of the decision rationales 41 -- 3.1.3 Relevance of the decision criteria 41 -- 3.1.4 Relevance of decision criteria: summarising countries achievements 52 -- 3.1.5 Revisability 53 -- 3.1.6 Enforcement 53 -- 4 GENERAL CONCLUSIONS AND RECOMMENDATIONS 55 -- 4.1 TRANSPARENCY CONDITION 55 -- 4.2 RELEVANCE CONDITION 56 -- 4.3 REVISABILITY CONDITION 57 -- 4.4 ENFORCEMENT CONDITION 57 -- 4.5 FUTURE RESEARCH 57 -- 5 LESSONS FOR BELGIUM 60 -- 5.1 ROLES OF THE DIFFERENT ACTORS IN THE PROCESS 60 -- 5.2 STRENGTHS OF THE BELGIAN REIMBURSEMENT SYSTEM 61 -- 5.3 ISSUES RELATED TO THE DECISION PROCESS 61 -- 5.3.1 Composition of the CRM/CTG 61 -- 5.3.2 Assessment by the RIZIV/INAMI experts 62 -- 5.3.3 Appraisal by the Drug Reimbursement Committee (CRM/CTG) 63 -- 5.3.4 Price and reimbursement basis 64 -- 5.3.5 Reimbursement category 65 -- 5.3.6 Decision by the Minister 66 -- 5.4 DISCUSSION 67 -- 5.4.1 Transparency 67 -- 5.4.2 Relevance of the appraisal criteria 68 -- 5.4.3 Revisions 68 -- 5.5 CONCLUSION 68 -- 6 APPENDICES 69 -- DRUG REIMBURSEMENT SYSTEM IN AUSTRIA 69 -- DRUG REIMBURSEMENT SYSTEM IN BELGIUM 81 -- DRUG REIMBURSEMENT SYSTEM IN FRANCE 99 -- DRUG REIMBURSEMENT SYSTEM IN THE NETHERLANDS 114 -- DRUG REIMBURSEMENT SYSTEM IN SWEDEN 128 -- 7 REFERENCES 143
