Pricing and Reimbursement in Oncology
Scope
Report Highlights
Reasons to Purchase
Table of Contents
- ABOUT DATAMONITOR HEALTHCARE - page 2
- Nish Saini - Lead Analyst, Oncology - page 2
- CHAPTER 1 EXECUTIVE SUMMARY - page 11
- Datamonitor insight into the oncology drug reimbursement/funding decision-making process - page 11
- Market penetration of novel cancer therapeutics challenged by increasing pharmacoeconomic vigilance - page 11
- US market characterized by self-professed poor management of oncology drugs by Managed Care Organizations - page 12
- More bureaucratic and centralized approach to drug reimbursement/funding decision-making prevails in the Europe - page 12
- NICE blight hampering novel oncology drug uptake in the UK - page 14
- Emphasis on pricing- rather than remibursement-control in Japan as regulatory approval virtually guarantees formulary inclusion - page 14
- Independent health technology assessment bodies a rarity - page 15
- Improvement in overall survival viewed as the optimal endpoint in supporting drug reimbursement funding/applications - page 15
- Increased management of oncology drugs by payers viewed as inevitable - page 16
- Datamonitor insight into the oncology drug reimbursement/funding decision-making process - page 11
- CHAPTER 2 OBJECTIVES, SCOPE AND COVERAGE - page 18
- Target Respondents - page 18
- List of abbreviations - page 19
- CHAPTER 3 INTRODUCTION - page 20
- Market penetration of novel cancer therapeutics challenged by increasing pharmacoeconomic vigilance - page 20
- Advances in cancer treatment outstripping available financial resources - page 20
- The rising costs of cancer pharmacotherapy are often disproportionate to improvements in survival rates - page 21
- Oncology is likely to be one of the first specialist disease areas to be targeted by the payer community - page 22
- Improved pharmacoeconomic analysis will be required to communicate the 'value' of novel cancer treatments - page 23
- Lack of flexibility in funding of new drugs in EU hospital settings has a major impact on patient access to innovative cancer drugs - page 23
- CHAPTER 4 DYNAMICS OF THE REIMBURSEMENT/ FUNDING PROCESS - page 25
- US market characterized by self-professed poor management of oncology drugs by Managed Care Organizations - page 25
- Individual benefit design dictates which MCO committee makes formulary-inclusion decisions - page 27
- Early delivery of clinical data to payer organizations is paramount to driving rapid and optimal market uptake - page 28
- Medicare Modernization Act (MMA) introduces defined time line for formulary-inclusion decisions - page 28
- Procedural aspects of formulary-inclusion decision-making appear standardized across interviewed MCOs - page 29
- Academy of Managed Care Pharmacy's dossier introduces consistency into decision-making process - page 29
- Procedure unchanged for second- and third-generation therapies - page 31
- Negative formulary-inclusion decisions have minimal impact on patient access to novel oncology therapeutics - page 31
- Mandatory and self-imposed payer constraints translate into virtually 'open' access to cancer drugs - page 32
- Payers attempt to rationalize oncologists' prescribing behavior through innovative approaches - page 33
- Japan: centralized agency controlling relatively efficient healthcare system - page 35
- Key stakeholders within the MHLW - page 35
- Pricing rather than reimbursement control in Japan since formulary inclusion is virtually guaranteed by regulatory approval - page 36
- Transparency increased with the NHI Pricing Organization - page 37
- Rewarding innovation with price premium - page 39
- Cost accounting method for drugs without comparators - page 41
- Biennial price cuts to contain healthcare costs - page 42
- Increasing importance of pharmacoeconomic data - page 43
- Novel drugs are subject to reassessment due to changes in circumstances - page 43
- France: multi-step pricing and reimbursement decision-making process can delay access to innovative oncology drugs - page 44
- Evaluation of therapeutic benefit by Transparency Commission is crucial to facilitating market access and optimizing manufacturer reimbursement - page 44
- Different agencies have responsibility for pharmacoeconomic and clinical evaluations - page 47
- Hospital formulary-inclusion decisions rely heavily on Transparency Commission evaluation - page 48
- 'ATU' system provides a mechanism for temporary authorization of innovative cancer drugs that would otherwise be delayed by lengthy decision-making processes - page 50
- Recently-mandated reference pricing system for multi-source drugs will have little if any impact on innovative oncology treatments - page 51
- Recent pricing and reimbursement reforms attempt to encourage innovation of drugs - page 51
- Germany: reference pricing system threatens revenue potential for second- and subsequent-generation oncology drugs - page 53
- Patient access to approved innovative cancer drugs relatively unhindered - page 53
- Within Germany's reference pricing system, decisions regarding innovative products are relatively uncomplicated - page 54
- Therapeutic categorization in German reference pricing system has implications for second- and third-in-class drugs - page 54
- Increased cost-containment measures are likely to impact on favorable drug prices for oncology drugs administered in hospitals - page 56
- Italy: single agency responsible for all aspects of drug approval, pharmacovigilance, pricing and reimbursement - page 57
- Regulation of centralized decision-making at local level is particularly relevant for oncology drugs - page 57
- Dynamics of application procedure and structure of AIFA facilitates expeditious decision-making for innovative drugs - page 58
- Elements beyond clinical and pharmacoeconomic data may influence pricing decisions - page 60
- Negative decisions regarding oncology therapeutics are rare - page 60
- Unique reference pricing systems can significantly challenge market penetration for second- and third-generation drugs - page 61
- Re-evaluation of manufacturer reimbursement levels every two years effectively penalizes success - page 62
- Spain is one of the most efficient EU nations in facilitating access to new cancer drugs - page 64
- Ease of access to innovative cancer drugs in Spain is evidenced by Karolinska Institute report - page 64
- Drug developers and suppliers bear the burden for cost-containment efforts - page 64
- Limited effect of reference pricing in Spain? - page 66
- Despite rigorous appraisal process by Directorate of Pharmacy and Healthcare Products, market access is virtually guaranteed following AEMPS approval - page 66
- Pharmacoeconomic comparisons have become steadily more prevalent and influential to the assignment of reimbursement status - page 67
- Autonomous communities (ACs) make efforts to rationalize regional drug use - page 68
- Pricing determinations for reimbursable prescription drugs performed by specialized agency - page 69
- UK: favorable pricing structures are countered by poor and discrepant access to novel cancer treatments across country - page 71
- Cancer networks are responsible for implementing and delivering NHS cancer services - page 71
- Lack of coordination among constituent PCTs in cancer networks challenges access to novel cancer drugs - page 72
- NICE serves as a 'gatekeeper' in managing universal access to novel treatments - page 73
- Attempts proposed to reduce significant delays in NICE appraisal process - page 73
- Complex funding system for oncology drugs, with procedural variation among geographic regions - page 74
- More consistent appraisal procedures will facilitate uniformity of decision-making process - page 75
- Funding decision-making triggered at request of local specialists rather than automatically following drug approval - page 75
- Payers will not routinely consider funding unlicensed drugs or off-label use of licensed drugs - page 76
- Inflexibility of drug-funding system hinders uptake of new technologies - page 76
- 'Exceptional circumstances' procedure offers the only funding route for unlicensed drugs and off-label use - page 77
- Constitution of funding decision-making group somewhat variable - page 78
- Clinical and pharmacoeconomic (PE) data evaluation is rigorous, sourcing information from multiple sites - page 79
- Inefficiency of financial arrangements and weak clinical evidence perceived as 'bottlenecks' to the decision-making process - page 79
- Process and decision-making for entry and evaluation of second- and third-generation products variable - page 80
- Manufacturers at the mercy of payers suffering "NICE blight" - page 81
- Increasing legal challenges to NICE? - page 81
- Cancer networks are responsible for implementing and delivering NHS cancer services - page 71
- US market characterized by self-professed poor management of oncology drugs by Managed Care Organizations - page 25
- CHAPTER 5 REQUIRED LEVELS OF CLINICAL AND ECONOMIC EVIDENCE - page 83
- Independent health technology assessment bodies a rarity - page 83
- Increased emphasis on contextualizing the clinical benefit derived from novel oncology drugs in relation to other therapeutic areas - page 85
- Greater acceptance among US payers of positive supporting data derived solely from Phase II trials - page 85
- French approach places onus on marketing company to fully disclose all supporting clinical and pharmacoeconomic data - page 86
- German and Italian payers less rigorous in their appraisal of the clinical value of novel oncology therapeutics already approved by EMEA - page 87
- Japanese payers primarily focus is on level of reimbursement rather than formulary inclusion decision-making - page 88
- Improvement in overall survival viewed as the optimal endpoint in supporting drug reimbursement funding/applications - page 88
- US payers more pragmatic in their consideration of endpoint designation - page 89
- European payers reticent in quantifying the relative increase in specific endpoints that would justify drug reimbursement/funding - page 90
- US payers more forthcoming in quantifying desired improvements in clinical benefit - page 91
- Automatic listing in Japan but endpoints significantly influence pricing based on prescribed formula - page 92
- Payers generally feel underserved in terms of the provision of pharmacoeconomic information from manufacturers. - page 92
- Discipline of pharmacoeconomics is in its relative infancy, with payers feeling unable to interpret complex information - page 93
- UK payers most familiar with QALY concept, an integral component of the NICE appraisal process - page 95
- Raising awareness of cost-effectiveness in Germany - page 95
- Independent pharmacoeconomic evaluations seen as preferential - page 96
- Pharmaceutical companies needs to shift payer focus away from solely drug acquisition costs - page 96
- Disparate opinions about influence of budget impact on decision making - page 96
- Heightened awareness among French payers with regard to off-label use - page 98
- Discrepant opinions among EU and US payers regarding relative importance of criteria for reimbursement/funding decisions - page 99
- EU payers interviewed placed a greater emphasis on cost-effectiveness than their US counterparts - page 101
- Beyond persuasive clinical and pharmacoeconomic data, other influences may impact on the decision process - page 102
- UK payers misplaced in their denial that payer advocacy or media attention have any influence on funding decisions? - page 102
- Early engagement of patient advocacy groups a valid and effective strategy - page 103
- Distribution channels and patient/caregiver education seen as important 'service elements' influential to US payers - page 103
- CHAPTER 6 FUTURE COST-CONTAINMENT MEASURES - page 105
- US payers believe they have little option other than to increase their management of oncology drugs - page 105
- US payers believe reduced profiteering among oncologists with regard to drug acquisition is essential - page 106
- Community oncologists argue that MMA legislation will significantly impact service provision - page 107
- Concern that financing of MMA may result in future implementation of price controls - page 108
- Pre-emptive move by pharmaceutical companies - page 108
- Impact of MMA will inevitably extend beyond Medicare patients - page 109
- Numerous other cost-containment measures envisaged in US - page 109
- Greater shift of cost-pressures to insureds not met with universal payer enthusiam - page 110
- European payers' favorable attitudes to innovative oncology drugs starting to wane - page 112
- An increased emphasis on disease prevention and drug reimbursement based on treatment outcomes postulated - page 113
- Risk-sharing model for pricing - page 113
- Uniqueness of UK-specific measures reflect complexity of drug funding system - page 114
- 'Payment by results' system may not be flexible enough to react to the introduction of novel therapies associated with significant cost-pressures - page 114
- Structural reforms in the delivery of primary care may help to reduce inequity of patient access to novel treatments - page 115
- Co-pay in the UK? - page 115
- No urgency among Japanese payers - page 116
- US payers believe they have little option other than to increase their management of oncology drugs - page 105
- CHAPTER 7 HYPOTHETICAL SCENARIOS - page 118
- Scenario one: Payer attitudes to a novel high-cost oncology drug with debatable clinical benefit - page 118
- Despite US payers expressing doubt about the drug's true clinical value, all stated that reimbursement was likely - page 118
- UK payers alone in their forthright opposition to funding drug unless acquisition costs were reduced - page 120
- In Germany economic and structural incentives may result in physician preference for intravenous formulations - page 120
- Automatic funding but pricing level depends on comparable drugs - page 121
- Scenario two: Value of second-generation oncology drugs with reduced toxicity accompanied by non-inferiority - page 122
- US payers view less toxic innovation favorably - page 122
- European payers require greater insight into clinical relevance of toxicity reduction - page 123
- Scenario one: Payer attitudes to a novel high-cost oncology drug with debatable clinical benefit - page 118
- CHAPTER 8 APPENDIX - page 125
- Contributing experts - page 125
- US Payers - page 125
- Japanese Payers - page 125
- French Payers - page 126
- German Payers - page 126
- Italian Payer - page 126
- Spanish Payers - page 126
- UK Payers - page 126
- US Payer Transcripts - page 127
- US Payer 1 - page 127
- Reimbursement/funding decision-making process - page 127
- Clinical and economic evidence - page 131
- Health technology assessments - page 138
- Future cost-containment efforts - page 138
- Hypothetical scenarios - page 139
- US Payer 2 - page 141
- Reimbursement/funding decision-making process - page 141
- Clinical and economic evidence - page 143
- Future cost-containment efforts - page 148
- Health technology assessments - page 149
- Hypothetical scenarios - page 150
- US Payer 3 - page 151
- Reimbursement/funding decision-making process - page 151
- Clinical and economic evidence - page 155
- Health technology assessments - page 161
- Future cost-containment efforts - page 161
- Hypothetical scenarios - page 163
- US Payer 4 - page 165
- Reimbursement/funding decision-making process - page 165
- Clinical and economic evidence - page 171
- Health technology assessments - page 178
- Future cost-containment efforts - page 179
- Hypothetical scenarios - page 180
- US Payer 5 - page 182
- Reimbursement/funding decision-making process - page 182
- Clinical and economic evidence - page 182
- Health technology assessments - page 188
- Future cost-containment efforts - page 188
- US Payer 6 - page 191
- Reimbursement/funding decision-making process - page 191
- Clinical and economic evidence - page 194
- Health technology assessments - page 199
- Future cost-containment efforts - page 200
- Hypothetical scenarios - page 201
- US Payer 7 - page 203
- Reimbursement/funding decision-making process - page 203
- Clinical and economic evidence - page 205
- Future cost-containment efforts - page 208
- US Payer 8 - page 212
- Reimbursement/funding decision-making process - page 212
- Clinical and economic evidence - page 215
- Health technology assessments - page 218
- Future cost-containment efforts - page 218
- Hypothetical scenarios - page 219
- US Payer 1 - page 127
- Japan Payer Transcripts - page 222
- Japan Payer 1 - page 222
- Reimbursement/funding decision-making process - page 222
- Clinical and economic evidence - page 223
- Health technology assessments - page 224
- Future cost-containment efforts - page 224
- Hypothetical scenarios - page 224
- Japan Payer 2 - page 226
- Reimbursement/funding decision-making process - page 226
- Clinical and economic evidence - page 227
- Health technology assessments - page 230
- Future cost-containment efforts - page 231
- Hypothetical scenarios - page 231
- Japan Payer 3 - page 234
- Reimbursement/funding decision-making process - page 234
- Clinical and economic evidence - page 235
- Health technology assessments - page 240
- Future cost-containment efforts - page 241
- Hypothetical scenarios - page 242
- Japan Payer 4 - page 245
- Reimbursement/funding decision-making process - page 245
- Clinical and economic evidence - page 247
- Health technology assessments - page 250
- Future cost-containment efforts - page 251
- Hypothetical scenarios - page 252
- Japan Payer 1 - page 222
- France Payer Transcripts - page 254
- France Payer 1 - page 254
- Reimbursement/funding decision-making process - page 254
- Clinical and economic evidence - page 256
- Health technology assessments - page 258
- Future cost-containment efforts - page 258
- Hypothetical scenarios - page 259
- France Payer 2 - page 260
- Reimbursement/funding decision-making process - page 260
- Clinical and economic evidence - page 262
- Health technology assessments - page 265
- Future cost-containment efforts - page 265
- Hypothetical scenarios - page 266
- France Payer 1 - page 254
- Germany Payer Transcripts - page 268
- Germany Payer 1 - page 268
- Reimbursement/funding decision-making process - page 268
- Clinical and economic evidence - page 270
- Health technology assessments - page 275
- Future cost-containment efforts - page 277
- Hypothetical scenarios - page 278
- Germany Payer 2 - page 281
- Reimbursement/funding decision-making process - page 281
- Clinical and economic evidence - page 283
- Health technology assessments - page 287
- Future cost-containment efforts - page 287
- Hypothetical scenarios - page 288
- Germany Payer 1 - page 268
- Italian Payer Transcript - page 290
- Reimbursement/funding decision-making process - page 290
- Clinical and economic evidence - page 294
- Spain Payer Transcripts - page 299
- Spain Payer 1 - page 299
- Reimbursement/funding decision-making process - page 299
- Clinical and economic evidence - page 301
- Health technology assessments - page 303
- Future cost-containment efforts - page 305
- Hypothetical scenarios - page 306
- Spain Payer 2 - page 309
- Reimbursement/funding decision-making process - page 309
- Clinical and economic evidence - page 311
- Health technology assessments - page 316
- Future cost-containment efforts - page 316
- Hypothetical scenarios - page 317
- Spain Payer 1 - page 299
- UK Payer Transcripts - page 321
- UK Payer 1 - page 321
- Reimbursement/funding decision-making process - page 321
- Clinical and economic evidence - page 324
- Health technology assessments - page 331
- Future cost-containment efforts - page 331
- Hypothetical scenarios - page 332
- UK Payer 2 - page 336
- Reimbursement/funding decision-making process - page 336
- Clinical and economic evidence - page 339
- Future cost-containment efforts - page 345
- Hypothetical scenarios - page 345
- UK Payer 3 - page 348
- Reimbursement/funding decision-making process - page 348
- Clinical and economic evidence - page 351
- Future cost-containment efforts - page 356
- Hypothetical scenarios - page 357
- UK Payer 4 - page 360
- Reimbursement/funding decision-making process - page 360
- Clinical and economic evidence - page 362
- Future cost-containment efforts - page 366
- Hypothetical scenarios - page 366
- UK Payer 1 - page 321
- List of abbreviations - page 369
- Bibliography - page 372
- Research methodology - page 372
- Disclaimer - page 373
- Contributing experts - page 125
- List of Tables
- Table 1: Governmental agencies responsible for drug approval and P&R in the EU markets - page 13
- Table 2: Premium rates for reimbursement prices of novel drugs set by the CMC - page 40
- Table 3: ASMR levels and definitions used by the Transparency Commission - page 46
- Table 4: Reimbursement levels in France - page 48
- Table 5: Prioritization of drug assessment criteria by payers (French payers did not respond) - page 100
- List of Figures
- Figure 1: Cancer drug costs as a percentage of total drug expenditure in the US and 5EU (2002-2003) - page 11
- Figure 2: Improved survival in metastatic colorectal cancer - page 21
- Figure 3: The cost of metastatic colorectal cancer treatment regimens - page 22
- Figure 4: Private healthcare insurance dominates US healthcare provision - page 26
- Figure 5: Overview of reimbursement price determination process in Japan - page 38
- Figure 6: Both the SMR and the ASMR rating are required to determine the reimbursement rate - page 45
- Figure 7: AIFA acts as an umbrella to a range of commissions and observatories - page 59
- Figure 8: Spain's healthcare system is largely decentralized with provision made by ACs - page 65
- Figure 9: An overview of the UK healthcare system - page 71
- Figure 10: Number of HTA reports in the HTA database for colorectal cancer, breast cancer, non-small cell lung cancer and non-Hodgkin's Lymphoma between 1990 and 2004 - page 84
- Figure 11: Prioritization of drug assessment criteria by payers (French payers did not respond) - page 101
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