Cost-Effectiveness Evidence to Inform Drug Reimbursement Decisions: Are Countries Converging in Process and Methods?

Cost-Effectiveness Evidence to Inform Drug Reimbursement Decisions: Are Countries Converging in Process and Methods? Mark Sculpher, PhD Professor of Health Economics Centre for Health Economics University of York, UK

Outline Overview of NICE process Overview of NICE methods Contrasts and similarities with other agencies internationally Scope for great harmonisation?

The National Institute for Health and Clinical Excellence (NICE) Background Following election of Labour government 1997 Prolonged controversy about post code prescribing in the UK National Health Service Wish to de-politicize decisions about which technologies to cover in NHS Desire to use best available methods to address difficult questions

250 A short history of NICE 200 150 Interventional Procedures QOF Quality standards Diag MD QS NHSE QOF 100 Clinical guidelines Public health PH IP Technologies CG TA 50 0 2000/1 2001/2 2002/3 2003/4 2004/5 2005/6 2006/7 2007/8 2008/9 2009/10 2010/11

NICE s activities Technology appraisal Pharmaceuticals Diagnostics (separate) Medical devices Guidelines Public health

The NICE process Overview Selection Assessment Appraisal

The NICE process Selection Not all licensed drugs selected NICE has key role in topic selection Criteria: Impact on costs Impact on health Innovative Recent predominance of cancer therapies

The NICE process Assessment multiple technology appraisals (MTAs) Longer review of several related technologies Scope sets up questions to be addressed Assessment period of about 6 months Undertaken by academic groups 3 key elements of the review: systematic review of clinical and economic evidence cost-effectiveness analysis critical review of sponsor (manufacturer) submission(s) Company submissions typically include model and systematic review All documents (and economic model) made available to consultees

The NICE process Assessment single technology appraisals (STAs) Change in 2006 in face of criticism about slowness Relates mainly to new pharmaceuticals Scope developed as for MTA Company provides all assessment clinical review, cost-effectiveness model Academic Evidence Review Group critically reviews submission

The NICE process Appraisal Patient organisation submissions Assessment reports Appraisal committee Manufacturer submissions Professional submissions Expert witnesses Patient witnesses

The NICE process Decisions Unconditional positive guidance Positive guidance conditional on particular patient characteristics Negative guidance Use recommended only in research Opportunity for appeal Decisions are reviewed in future

NICE Reference Case Use of cost-effectiveness analysis NICE. Guide to the Methods of Technology Appraisal, 2008.

NICE Reference Case Use of QALYs to reflect health benefit NICE. Guide to the Methods of Technology Appraisal, 2008.

NICE Reference Case Use of QALYs to reflect health benefit Utility weights from public NICE. Guide to the Methods of Technology Appraisal, 2008.

NICE Reference Case Focus on health system costs alone NICE. Guide to the Methods of Technology Appraisal, 2008.

The NICE threshold Below a most plausible ICER of 20,000 per QALY gained, the decision to recommend the use of a technology is normally based on the costeffectiveness estimate and the acceptability of a technology as an effective use of NHS resources. When the estimated ICERs presented are less than 20,000 per QALY gained and the Committee judges that particular interventions should not be provided by the NHS, the recommendations will make specific reference to the Committee s view on the plausibility of the inputs to the economic modelling and/or the certainty around the estimated ICER. Above a most plausible ICER of 20,000 per QALY gained, judgements about the acceptability of the technology as an effective use of NHS resources will specifically take account of the following factors. - The degree of certainty around the ICER -. the change in HRQL has been inadequately captured - The innovative nature of the technology NICE. Guide to the Methods of Technology Appraisal. London: NICE, 2008.

Process differences in other jurisdictions Drugs vs other technologies Full range of technologies Pharmaceuticals only NICE (England and Wales) HITAP (Thailand) IQWiG (Germany) HAS (France) SMC (Scotland) NCPE (Ireland) TLV (Sweden) Netherlands CDR (Canada) +++ Variation in whether decision making or not Some jurisdictions have separate organisations for drugs and other technologies

Process differences in other jurisdictions All drugs versus a selection Selection All drugs NICE (England and Wales) IQWiG (Germany) HAS (France) SMC (Scotland) TLV (Sweden) CDR Canada +++ Key distinction between organisations providing guidance and recommendations and those with mandatory reimbursement arrangements

Process differences in other jurisdictions Independent analysis versus manufacturer alone Independent analysis Manufacturer alone NICE MTA (England and Wales) IQWiG (Germany) NICE STA SMC (Scotland) NCPE (Ireland) TLV (Sweden) Netherlands CDR Canada +++

Other areas of difference in process Explicit setting of a scope Key part of all NICE activities One off decision versus periodic reviews NICE reviews every 2-4 years Level of detail provided about decisions All documentation and justification on NICE website Role of price in decisions Reference pricing systems (e.g. Netherlands) Free price setting (e.g. UK)

Methods differences between jurisdictions The emergence of some clustering? QALY based CEA - NICE - Canada - Australia - Sweden - Ireland - +++ Other economics - Germany - France - Various US health plans No (explicit) economics - US public sector

Methods differences between jurisdictions Variation remains within clusters - QALYs Issue CEA with QALYs General position Australia Generally Preferred Canada Optional Eng & Wales Required QoL weights MAUI generally Preferred Several options Described EQ-5D preferred Preferences Public Public Public Role of other methods CMA has a role CBA limited role CMA may have a role CMA, CEA and CBA all have a role Limited

Methods differences between jurisdictions Variation remains within clusters - perspective Payer Societal Both Not stated 13 (50%) 6 (23%) 6 (23%) 1 (4%) See Claxton K et al. 2010.

Absence of clear rationale for selected perspective 50% of guidelines offer no rationale for their preferred perspective There is broad consensus nationally and internationally that the societal perspective is the most appropriate choice. (Netherlands) The perspective chosen should fit the needs of the target audience. (Canada) 27% (7/26) make reference to a budget constraint

Methods differences between jurisdictions Variation remains within clusters - comparator Most commonly used Existing, most effective or minimum practice Existing or most effective Justify Existing and no treatment Most common, least costly, no treatment Most common, least costly, no treatment, most effective Most common, least costly, most effective Most likely to be displaced Most efficient, most effective, do nothing All relevant comparators Most effective and no treatment Not clear/specific Tarn and Smith 2004; Sculpher and Drummond 2006 8 2 1 1 2 1 2 1 1 2 2 1 3

Methods differences between jurisdictions Variation remains within clusters sensitivity analysis Need to state and justify Not stated/not specific Probabilistic sensitivity analysis (PSA) One-way, multi-way One-way, two-way Multi-way (of most important) One-way, multi-way and PSA One-way, multi-way and worst-best scenario One-way with tornado diagram 3 10 3 1 2 1 5 1 1 Tarn and Smith 2004; Sculpher and Drummond 2006

More fundamental methodological differences IQWiG in Germany Economic analysis to support decisions of Ministry of Health and Sickness Funds Controversial methods recommendations by international panel Panel referred to constraints imposed by German law and IQWiG Little experience is using these methods in studies Caro et al, 2010; Sculpher and Claxton 2010

IQWiG methods Similar to NICEtype cluster Cost-effectiveness analysis Full range of comparators Use of sub-groups Some modelling permitted Recognised need for sumary measure of health Different to NICEtype cluster Decisions limited to specific disease areas Reluctant to have generic measure of outcome (especially the QALY) No general CE threshold used in decision making Role of modelling long term benefits unclear

More fundamental methodological differences HAS in France Decisions by HAS informed by Transparency Commission and Commission for Economic and Public Health Evaluation (CEESP) Reimbursement clinically driven SMR: benefit of drug ASMR: relative effectiveness versus comparators ASMR 1-3 companies set prices so decisions reflect implicit economic assessment Emerging role of CEESP to use economics but Post launch Part of broader HTA process

Scope for harmonisation? Various activities going on in Europe around HTA But many differences exist between jurisdictions in use of economic evaluation Process Methods major and minor Partly justifiable Scope for harmonisation in assessment Shared evidence (e.g. Reviews/meta analysis) Shared models Limited scope in decision making

References Sculpher MJ, Drummond MF. Analysis sans frontières: can we ever make economic evaluations generalisable across jurisdictions? Pharamacoeconomics. 2006;24(11):1087-99. Tarn TY, Smith MD. Pharmacoeconomic guidelines around the world. ISPOR Connections 2004;10(4):5. Claxton K, Walker S, Palmer S, Sculpher M. Appropriate Perspectives for Health Care Decisions. CHE Research Paper 54 (http://www.york.ac.uk/inst/che/pdf/rp54.pdf). York: Centre for Health Economics, University of York; 2010. Caro J, Nord E, Siebert U, McGuire A, McGregor M, Henry D, et al. The efficiency frontier approach to economic evaluation of health-care interventions. Health Economics. 2010;DOI: 10.1002/hec.1629. Sculpher M, Claxton K. Sins of Omission and Obfuscation: IQWiG s Guidelines on Economic Evaluation Methods. Health Economics. 2010;DOI: 10.1002/hec.1629.