Real World Data: Cosa Sono, Come Raccoglierli Giovanni Fiori, PhD, MPH Scientific Director, MediData Studi e Ricerche (Modena, Italy)

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Observational Studies Working Group, Italian Society of Applied

1 Real World Data: Cosa Sono, Come Raccoglierli Giovanni Fiori, PhD, MPH Scientific Director, MediData Studi e Ricerche (Modena, Italy) Coordinator, Observational Studies Working Group, Italian Society of Applied Pharmacological Science (SSFA) Chairman, Late Phase Working Group, European CRO Federation (EUCROF)

2 Summary Definition of Real World Data Methodological issues in designing a RWD collection How to face some operative constraints (e.g. manage the lack or regulations on RWD)

3 Real World Data Definition data used for decision making that are not collected in conventional randomized controlled trials (RCTs). RWD could be characterized by: type of outcome (clinical, economic and patient-reported); hierarchies of evidence (which rank evidence according to the strength of research design); type of data source ISPOR Real-World Task Force Report: Garrison et al. 2007, Value in Health; Vol.10 N. 5 pp

4 Outcomes Research Research addressed to evaluate what comes out after an health care intervention using measures useful for Patients and/or for Decision Makers Outcome Research - the study of the end results of health services that takes patients experiences, preferences, and values into account - is intended to provide scientific evidence relating to decisions made by all who participate in health care Clancy CM, Eisenberg JM. Outcomes research: measuring the end results of health care. Science. 1998;282:

5 From Efficacy to Effectiveness Experimental Studies Disease features E V I D E N C E National health service s members behavior exportability Patient features Structural features O U T C O M E S Clinical morbidity, symptoms, mortality Humanistic patientreported symptoms, quality of life Economical resource use and costs Tumor regression Disease free survival Overall Survival Quality of life Patient Satisfaction Caregivers burnout Appropriateness Direct medical, direct non medical, indirect costs

6 From Effictiveness to Efficiency Pre Marketing Post Marketing EFFICACY EFFECTIVENESS EFFICIENCY + Safety (Small Population) + Safety (Large Population) + Pharma and Health economics

7 Summary Definition of Real World Data Methodological issues in designing a RWD collection How to manage the lack or regulations and other operative constraints

8 Real World Data = Real World Evidence? Data conjures the idea of simple factual information, whereas evidence connotes the organization of the information to inform a conclusion or judgment. Evidence is generated according to a research plan and interpreted accordingly, whereas data is but one component of the research plan. Evidence is shaped, while data simply are raw materials and alone are non informative. In general, real world evidence is what happens to real world data ISPOR Real-World Task Force Report: Garrison et al. 2007, Value in Health; Vol.10 N. 5 pp

9 Hierarchies of Evidence

10 Who s the ugly duckling?

11 Objective Tool Experimental Observational

One Tool Many Applications Clinical Epidemiology Epidemiology and Pharmacoepidemiology Post Authorization Safety Study (PASS/PAES) Registries Outcomes Research Burden of Disease Health Related -

12 One Tool Many Applications Clinical Epidemiology Epidemiology and Pharmacoepidemiology Post Authorization Safety Study (PASS/PAES) Registries Outcomes Research Burden of Disease Health Related - Quality of Life Health Economics Health Service Research Analysis of Health Care Processes Health Technology Assessment Monitoring of Clinical Guidelines New Tool Many Applications! Not only PASS/ PAES

13 Observational Methods Source of RWD Real-World Data can also be categorized by type of data source. 1. Databases: These include cross-sectional and longitudinal databases which essentially provide retrospective data. 2. Patient chart reviews: Used to reflect particular insights in patient management. 3. Patient and population surveys: Primarily for epidemiological information. 4. Registries: patients treated at a particular centre for a particular condition on a continuous basis. 5. Cohort studies: What most people would understand by real life studies. 6. Pragmatic clinical trials: Whether these are strictly real-life studies is open to debate ISPOR Real-World Task Force Report: Garrison et al. 2007, Value in Health; Vol.10 N. 5 pp

14 Matched Cohort Study Exposition A Outcome Exposition B Outcome Time

15 Registries Observational Studies Registries Registries are observational studies of patients who have a particular disease and/or are receiving a particular treatment or intervention. They can be used for understanding natural history, assessing or monitoring realworld safety and effectiveness, assessing quality of care and provider performance, and determining value or reimbursement levels ISPOR REAL WORLD TASK FORCE

16 One Registry Several Secondary Studies PATIENT REGISTRY Cost of Ilness Pharmacoeconomics Appropriateness DATABASE Retrospective Cohort Study Case Control Study

17 Summary Definition of Real World Data Methodological issues in designing a RWD collection How to face some operative constraints (e.g. manage the lack or regulations on RWD)

18 Map of Interactions Real World Data Epidemiology Experimental Observational Pharmaco Epidemiology PASS Pharmaco Vigilance Pharmacology Guidelines on Good Pharmacovigilance practices (GVP), Module VIII (20/Feb/2012) Post Authorization Safety Studies (PASS)

19 PASS Definition Any study relating to an authorized medicinal product conducted with the aim of: identifying, characterizing or quantifying a safety hazard confirming the safety profile of the medicinal product measuring the effectiveness of risk management measures Regulation (EU) No. 1235/2010; Directive 2010/84/EC; Guidelines on Good Pharmacovigilance practices (GVP), Module VIII (20/Feb/2012) Post Authorization Safety Studies (PASS)

20 Operational Impact Lack of Common EU Regulation Country Specific Regulations 20

21 Riferimenti Normativi (Italia) Studi Osservazionali sul farmaco Linea Guida AIFA Linea Guida Privacy Normativa PASS e Farmacovigilanza Studi Osservazionali non sul farmaco: Epidemiologici Qualità di Vita Economia Sanitaria Appropriatezza Diagnostica Linea Guida Privacy

22 Italia: Iter Autorizzativo Tipologia Studio Iter Studi di coorte prospettici sul farmaco (nei quali i pazienti sono inclusi nello studio in base all assunzione di un determinato farmaco e seguiti nel tempo per la valutazione degli esiti) Altre tipologie di studi osservazionali di coorte retrospettivi trasversali caso controllo solo su casi (case cross over/case series) di appropriatezza Parere Unico (45 gg) Accettazione o rifiuto dei CE satelliti (30 gg) Notifica ai CE Passati 60 gg: silenzio assenso

23 Studi Retrospettivi e Privacy Consenso al trattamento dati in SOs retrospettivi non è necessario qualora: 1. non sia possibile informare l interessato per motivi etici, metodologici o per motivi di impossibilità organizzativa; 2. il programma di ricerca sia stato oggetto di parere favorevole del competente Comitato di Etica; 3. il trattamento sia autorizzato dal Garante Dal 2012 annuale Autorizzazione Generale ma valida solo per alcuni casi (es: non per dati genetici)

24 Autorizzazione Generale: Condizioni di Applicabilità Parere favorevole del Comitato Etico competente a livello territoriale Impossibilità di acquisire consenso per: Motivi organizzativi: pericolo di alterazione dei risultati in assenza di arruolamento di soggetti che non possono esprimere consenso (deceduti o persi al follow up) Motivi etici: l interessato ignora la propria condizione; pericolo di danno materiale o psicologico Varie Prescrizioni: Es: ricerca telefonica/anagrafe, rinuncia in caso di rifiuto anonimizzazione finale, varie misure di sicuerzza,

25 Conclusions

26 Benefits of Real World Data Estimate long-term clinical benefits and harms Compare multiple interventions to inform optimal therapy choices Insight in dosing of drugs, duration and compliance Examine outcomes in diverse populations Demonstrate full value of a product, broader range of outcomes (incl. compliance, safety, QoL) Data on resource use for economic analyses Provide evidence to public payers that product is being used in the intended target population Data if no RCT possible ISPOR Real-World Task Force Report: Garrison et al. 2007, Value in Health; Vol.10 N. 5 pp

Real World Evidence For the life-science industry, the current regulatory and payer developments in Europe will have an important impact on the current form of drug

27 Real World Evidence For the life-science industry, the current regulatory and payer developments in Europe will have an important impact on the current form of drug development We are likely to see a continuous generation of RWE throughout the entire life cycle of new health technologies Patrik Sobocki (2013) ISPOR CONNECTIONS - V.19 N. 5 (pp 10-11)

28 Real World Evidence Building the Evidence requires the systematic unbiased collection of data The validity of the Evidence is dependent on the accuracy of the data and the appropriate organization to allow interpretation, analysis and conclusions Transparency Quoted by: A.M. Pleil, Senior Director Worldwide Medical and Outcomes Research - Pfizer in «Using Real World Data in Pharmacoeconomic Evaluations: Challenges, Opportunities and Approaches

29 Pending Issues Huge heterogeneity in terms and definitions Different approaches and methodological standards Lack of Regulations Guidance is more dispersed, with many professional bodies producing guidance on aspects relevant to their respective disciplines (eg. EMA vs ISPOR vs ENCePP)

30 Grazie dell Attenzione

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