Stefano Capri
Scuola di Economia e Management Università LIUC, Castellanza (VA)
Milano, 17 gennaio 2017
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2001
2009
Comparative Effectiveness Research Methods
Economic Evaluation Methods
Modeling Methods
Observational Study Methods-Database Methods
Observational Study Methods-Medication Adherence Methods
Patient Reported & Clinician Reported Outcomes Methods
Preference-Based Methods
Risk Benefits Methods
Use of Outcomes Research in Health Care Decisions
Budget Impact Analysis Good Practices
Cost Effectiveness Analysis with Clinical Trials
Cost-Effectiveness Analysis alongside Clinical Trials Good Practices II
(in development)
Health Economic Evaluation Publication Guidelines – CHEERS
Measuring Drug Costs in CEA: Issues and Recommendations
Measuring Drug Costs in CEA: A Societal Perspective
Measuring Drug Costs in CEA: A Managed Care Perspective
Measuring Drug Costs in CEA: Medicare/Medicaid Perspective
Measuring Drug Costs in CEA: An Industry Perspective
Measuring Drug Costs in CEA: An International Perspective
Nutrition Economics (in development)
Quality Improvement of Cost Effectiveness Research
Transferability of Economic Evaluations Across Jurisdictions
Conceptualizing a Model: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force Working Group-2
Dynamic Transmission Modeling: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force Working Group-5
Modeling Good Research Practices - Overview: A Report of the ISPOR- SMDM Modeling Good Research Practices Task Force-1
Modeling Studies
Modeling using Discrete Event Simulation: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force Working Group-4
Model Parameter Estimation and Uncertainty: A Report of the ISPOR- SMDM Modeling Good Research Practices Task Force Working Group-6
Model Transparency and Validation: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force Working Group-7
Simulation Modeling Applications in Health Care Delivery Research - Emerging Good Practices Task Force
State-Transition Modeling: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force Working Group-3
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COUNTRY-SPECIFIC PHARMACOECONOMIC GUIDELINES
Published PE
Recommendations PE Guidelines Submission Guidelines
Africa South Africa Egypt
America-Latin
Brazil Colombia
MéxicoCuba America-North United States Canada
Asia China Mainland Taiwan
South Korea Malaysia
Israel Thailand
Europe
Austria Denmark
Hungary Italy
Russian Federation Spain
Croatia
Baltic (Latvia, Lithuania, Estonia)
Belgium France Germany
Ireland The Netherlands
Norway Portugal Slovak Republic
Slovenia Sweden Switzerland
England & Wales Finland
Poland Scotland
Spain - Catalonia Region
Oceania New Zealand AustraliaI
Da tempo le “buone pratiche” sono standardizzate (sebbene con differenti livelli di approfondimento- analisi statistica, modelli)
◦ Possibili evoluzioni/miglioramenti
Come accade per le sperimentazioni cliniche: un bravo biostatistico ed un bravo clinico insieme
sono in grado di disegnare e/o valutare uno studio di qualità.
Le linee guida che servono sono quelle “normative”, quelle richieste per l’ottenimento del P&R (NICE,…)
Studi di di qualità, ma soprattutto pratiche di HTA per l’utiizzo di tali studi:
◦ Pricing & reimbursement
◦ Budget impact
◦ Incremento utilizzo nuove terapie
◦ Abbandono di terapie esistenti
◦ Inserimento della farmaoeconomia nel sistema di incentivi (positivi/negativi) per le strutture di
offerta e per gli operatori
IL VALORE DEL TRATTAMENTO
Un differente modo di attribuire valore ad un trattamento oncologico:
American Society of Clinical Oncology Value Framework
Schnipper et al. JCO 2016
Schnipper et al. JCO 2016
Schnipper et al. JCO 2016
NET HEALTH BENEFIT
NHB score it is derived from:
• overall survival (OS),
• progression-free survival (PFS),
• response rate (RR),
• symptom palliation,
• time off
• treatment,
• QoL, along with the comparative toxicity of the regimen
Clinical benefit, toxicity, net health benefit (NHB), and cost of ipilimumab versus placebo as derived from the prospective randomized trial comparing ipilimumab against placebo after primary treatment of stage III melanoma.
Schnipper et al. JCO 2016
Schnipper et al. JCO 2016
Net health benefit (NHB) scores for doxorubicin plus cyclophosphamide followed by paclitaxel plus trastuzumab versus doxorubicin,
cyclophosphamide, and paclitaxel (control) in the adjuvant treatment of human epidermal growth factor receptor 2–positive breast cancer
NHB(Net Health Benefit) non permette confronti con le altre aree terapeutiche
NHB non contiene alcun elemento di teoria economica (e.g. l’utilità)
NHB è ancora in evoluzione
Proposte
Elaborare un algoritmo per tradurre NHB in QALYs
Approfondire il peso della OS; utilizzo delle proxies (PFS, ORR, ecc.)
creare una scala di preferenze
utilizzare il NHB attraverso un processo di MCDA (Multiple Criteria Decision Analysis)
