A European Com m on Data Model? W hy? W hich and How ? Dr Alison Cave, Principal Scientific Administrator, Pharmacovigilance and Epidemiology Department European Medicines Agency An agency of the European Union
W hy?...... W hat is the need and w hy now ? Unknown generalisability of clinical trial results to normal clinical practice and lack of information in high risk groups demands new approaches to gather complementary evidence Rapidly evolving scientific landscape enabling increasing patient stratification, driving innovative m edicinal approaches and fuelling new possibilities for rare diseases but often results in sm aller, focused and shorter RCTs or presents situations where a RCT is not feasible A need for validation of shorter term surrogate endpoints with long term beneficial outcomes I ncreasing data availability coupled with technological advances which offer new possibilities to store, mine and analyse data across multiple datasources 1
Enhanced Evidence Generation Support the Support targeted Highlight developm ent/ Planning of post I dentify m issing and planned data possible EU am endm ent authorisation studies data/ gaps collection data sources of a registry Life Cycle of a Medicine Data to Direct need for EU support w ide clinical data Approval com m ittee Drug Utilisation Disease decision studies prevalence/ Current clinical m aking practice epidem iology Scientific Advice COMP CHMP PRI ME PDCO PRAC COMP CAT 2
Access to RW E Direct Access I ndirect Access Com m on Protocol Integration and analysis Integration and analysis Fram ew ork Contract Direct interrogation Common Protocol Results Results Multiple RWE Data Multiple RWE Data Sources Sources Advantages: Advantages: More willingness to participate because EMA has direct access to 3 datasources there is no transfer of data which enables quick queries and in house Access to expertise studies to be run Staged implementation Enables collaborative studies to be performed with the EU network Lim itations: Need for internal resources Limited geographical coverage Collaborative studies can be slow
N EMA-funded studies N countries databases A/ H1 N1 pandem ic vaccines and pregnancy outcom es 1 1 I m pact of risk m inim isation in patients treated w ith rosiglitazone-containing products 2 2 I sotretinoin and the effectiveness of the Pregnancy Prevention Program m e in Europe 5 3 Patterns and determ inants of use of oral contraceptives in the EU 5 3 Monitoring the effectiveness of risk m inim isation in patients treated w ith pioglitazone-containing products 3 3 Risk of cardiac valve disorders associated w ith the use of biphosphonates 6 3 Association betw een anxiolytic or hypnotic drugs and total m ortality 2 2 Metform in use in renal im pairm ent 2 2 Study of regulatory com m unication and risk aw areness follow ing the Article 3 1 referral of Com bined n/ a 6 Horm onal Contraceptives in relation to throm boem bolism Characterising the risk of m ajor bleeding in patients w ith Non-Valvular Atrial Fibrillation: non- 9 6 interventional study of patients taking Direct Oral Anticoagulants in the EU Study of utilisation of Com bined Horm onal Contraceptives in Europe 3 3 Anti-m icrobial resistance: choice of therapeutic interventions and outcom es for the treatm ent of infections 4 1 caused by MDR Gram negative pathogens Methods and data sources for determ ining long-term effects of drug exposure during pregnancy, w ith n/ a 2 8 application to antiepileptic m edicines I m pact of EU label changes for system ic diclofenac products: post-referral prescribing trends 4 3 I m pact of EU label changes for hydroxyzine products: post-referral prescribing trends 4 3 4
EMA access to RW E? Direct Access I ndirect Access I ndirect Access Com m on Protocol Com m on Data Integration and analysis Integration and analysis Integration and analysis Fram ew ork Contract Fram ew ork Contract Common Data Model Direct interrogation Common Protocol Results Results Results Multiple RWE Data Multiple RWE Data Multiple RWE Data No one solution – a hybrid approach w ill be required Sources Sources Sources Advantages: Advantages: Advantages: More willingness to participate because More willingness to participate because EMA has direct access to 3 datasources there is no transfer of data there is no transfer of data which enables quick queries and in house Access to expertise Access to expertise studies to be run Staged implementation Staged implementation Enables collaborative studies to be Fast performed with the EU network Lim itations: Lim itations: Slower process for studies to be run Lim itations: Upfront resource investment Potential lack of interest from partners to Need for internal resources Potential loss of information in transfer to participate in regulatory questions Limited geographical coverage CDM Collaborative studies can be slow Need for validation of model
W hat is the problem ? Fragm entation of Lack of sustained resources, lack of funding m echanism s Lack of a fram ew ork to interoperability to secure access to and enable rapid, repeated and sufficient access to analysis of RWD datasources Governance and Lack of cross border privacy challenges collaborations to leverage existing data and knowledge Need to implement methods to integrate Lack of a com m on agreement and analyse across relevant stakeholders heterogeneous data 6
W hat is the problem ? Fragm entation of Lack of sustained resources, lack of funding m echanism s Lack of a fram ew ork to interoperability to secure access to and enable rapid, repeated and sufficient access to analysis of RWD datasources Governance and Lack of cross border privacy challenges collaborations to leverage existing data and knowledge Need to implement methods to integrate Lack of a com m on agreement and analyse across relevant stakeholders heterogeneous data 7
Multiple Approaches 8
This meeting explores just one possible approach The Com m on Data Model A common data model can be defined as a mechanism by which the raw data are standardised to a common structure, format and terminology independently from any particular study in order to allow a combined analysis across several databases/ datasets. Standardisation of structure and content allows the use of standardised applications, tools and methods across the data to answer a wide range of questions. 9
Motivation • To establish access to a m aintained set of healthcare databases which are able to provide answers to m ultiple regulatory questions. • To accelerate response times. • To aid clear docum entation of the methodological process. • To aid interpretation of heterogeneous results. 10
I s a com m on data m odel the solution for Europe? How can w e m anage but exploit the heterogeneity in data across Europe? How do w e balance flexibility w ith speed? How do w e validate the data transform ation? W hat are the key criteria for validation? How do build a operationalise a netw ork? How do w e ensure the expertise of all stakeholders is incorporated? How do w e build a sustainable system ? W hat are the key design choices of a CDM w hich influence data sufficiency? 11
Objectives: To define the opportunities and challenges around implementation of a common data model in Europe to support regulatory decision making. Output: To propose guiding principles for the development of Common Data model in Europe including key criteria for validation in the context of regulatory decision making. 12
Session 1 : A Common Data Model – Why? Session 2 : A Common Data – Which? Session 3 : Validation of CDM - What is needed for regulatory decision making? Session 4 : Solutions for Europe: what is needed? Group 1: What are the specific European barriers and challenges in applying a CDM? Group 2: How do you operationalise a CDM network? Group 3: What are the key criteria necessary for validation of a CDM in Europe? Group 4: What are the key design choices of a CDM which influence the range of regulatory questions that can be addressed? 13
Thank you for your attention! European Medicines Agency 30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom Telephone + 44 (0)20 3660 6000 Facsim ile + 44 (0)20 3660 5555 Further questions: alison.cave@em a.europa.eu Follow us on @EMA_ New s 14
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