Table 1.
Summary of International Value Determination Frameworks
| Country | Reviewing Body | Criteria for Value Assessment | Evidence | Outcome Variables | Cost Metrics | Type of Economic Assessment | Use of Results | Source |
|---|---|---|---|---|---|---|---|---|
| United Kingdom | NICE, SMC, AWMSG | Strength of available evidence, importance of outcomes, health impact, cost effectiveness, budget impact, inequalities, feasibility of implementation, impact on NHS, acceptability, broad clinical and government policy priorities, health needs | Burden of disease (population affected, morbidity, mortality); resource impact (cost impact on NHS or public sector); clinical and policy importance (whether topic is within government priority area); presence of inappropriate variations in practice; potential factors affecting timeliness of guidance to be produced (degree of urgency, relevance of guideline at expected date of delivery); likelihood of guidance having impact on public health and quality of life, reduction in health inequalities, or delivery of quality programs or interventions; appropriateness and ability of NICE to commence development of guideline. (source: manufacturer data, RCTs, systematic literature reviews) | Mortality, morbidity, quality of life, cost per QALY | Direct costs for NHS and PSS; may also add travel and other public sector costs but typically does not include productivity costs; some consideration for indirect costs | Cost-effectivness or cost-utility analysis; cost-benefit analysis may be used in specific situations; in addition, cost-consequence approach may be adopted to take account of complex and multidimensional character of public health interventions and programs; other issues (eg, equity, distribution) can also inform analysis; budget impact model (template specific to SMC) or cost minimization where clinical equivalence is statistically demonstrated | To develop standards, guide patient-care decisions, inform strategies to meet government indicators and targets, support decision making on NHS funding and resource allocation, guide education and training of health professionals; health authorities are unlikely to accept (reimburse or fund) products that are not recommended (although they can) and are mandated (in England) to reimburse products that are recommended | NICE,30 SMC,31 AWMSG32 |
| Canada | pCODR, INESSS | Overall clinical benefit, cost effectiveness, alignment with patient values, feasibility of adoption into health system | Effectiveness, measured in terms of relevant patient outcomes (eg, mortality, morbidity, quality of life) with magnitude, direction, and uncertainty of effect also considered; safety; burden of illness; need (availability of effective alternative); patient values; cost effectiveness; economic feasibility (net budget impact of new drug, including companion testing); organization feasibility (source: manufacturer data, RCTs, systematic literature review, clinical guidance report, patient advocacy data, other unpublished data) | Mortality, morbidity, safety, quality of life | Direct costs from public payer perspective (usually Ontario MOHTL) | Cost, cost-effectiveness analysis, cost-utility analysis, budget impact assessment; uncertainty of results must be assessed (range for worst-case scenario) | Evaluate clinical data, assess cost effectiveness, make recommendations to guide drug reimbursement decisions at provincial level; decisions can include: list, list with conditions upon clinical criteria or lower ICER, or not list | pCODR,33 INESSS34 |
| Australia | PBAC | Clinical efficacy and costs compared with other medications already in PBS for corresponding indications; cost-effectiveness and cost-utility analyses | Meta-analysis of manufacturer data against available comparator data, including benefits and costs; assessment of direct randomized trials to give superior therapeutic conclusion; translation of these direct trial issues using premodeling provide trial-based or stepped economic evaluation (ie, cost effectiveness); epidemiologic analysis of budgetary implications (source: manufacturer data, RCTs, indirect comparisons of several trials with applicable comparator) | Efficacy (ICERs, QALYs, LYGs), morbidity, mortality, maximum health outcome per dollar spent | Direct and indirect costs; process takes national health budget perspective, looks at costs and offsets to health care system as whole, as well as patient copay amounts | Comparative-effectiveness analysis, relative comparative effectiveness, cost-minimization analysis where clinical equivalence is statistically demonstrated | Recommendations for state subsidization of new pharmaceutical agents | PBAC35 |
| France | HAS Transparency Commission and Public Health and Economic Evaluation Committee, CEPS | Clinical effectiveness of drug and possible side effects, position in therapeutic spectrum relative to other available treatments, disease or condition severity, clinical profile of drug, public health impact, cost-effectiveness for innovative drugs (ASMR I, II, III) expected to have significant budget impact on system | Clinical, epidemiologic, and economic data; financial and public health impact (source: manufacturer data, RCTs, systematic literature reviews, indirect comparisons) | Mortality, morbidity, quality of life | Depends on aim of study or assessment; all relevant costs must be reported and presented in detail; indirect costs must be reported separately | Budget impact models; cost-minimization, cost-effectiveness, cost-utility, or cost-benefit analysis | Reimbursement and pricing decisions | HAS,36 Ministère des Affaires Sociales de la Santé37 |
| Germany | Federal Joint Committee; Institute for Quality and Efficiency in Health Care | Nature and severity of disease, magnitude of additional therapeutic benefit, availability of treatment alternatives, adverse-effect profile | Clinical benefit with respect to patient-relevant outcomes, medical need, efficiency (source: RCTs, systematic literature reviews, indirect comparisons [in special cases]) | Mortality, morbidity, quality of life | All direct and, in some cases, indirect costs | For early benefit only, direct cost comparison; no health economic assessments unless pricing negotiation sent to arbitrage | Supports reimbursement, pricing decisions, guideline development | Bundesministerium für Gesundheit38 |
Abbreviations: ASMR, Amélioration du Service Médical Rendu; AWMSG, All Wales Medicines Strategy Group; CEPS, Comité Économique des Produits de Santé; HAS, Haute Autorité de Santé; ICER, incremental cost-effectiveness ratio; INESSS, Institut National d'Excellence Santé et en Services Sociaux; LYG, life-year gained; MOHTL, Ministry of Health and Long-Term Care; NHS, National Health Service; NICE, National Institute for Health and Care Excellence; PBAC, Pharmaceutical Benefits Advisory Committee; PBS, Pharmaceutical Benefits Scheme; pCODR, Pan-Canadian Oncology Drug Review; PSS, Personal Social Services; QALY, quality-adjusted life-year; RCT, randomized controlled trial; SMC, Scottish Medicines Consortium.