to Health Care Databases: : Societal and Legal Challenges Aurore - - PowerPoint PPT Presentation

Pharmacoepidemiology & Risk Management.

Dru rug Util ilisation Studies and Access to Health Care Databases: : Societal and Legal Challenges

Aurore Bergamasco, Caroline Tremblay, Alia Yousif, Yola Moride

Disclaimer

• YOLARX is a privately held company specialized in consulting and study

conduct in pharmacoepidemiology, pharmacovigilance and therapeutic risk management

• Our current global customer base includes pharmaceutical companies as

well as regulatory agencies from across the world

• No funding was received for the present study

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Context

• Drug utilisation studies (DUS) are increasingly requested by health authorities

whether these are regulatory agencies (EMA) or third-party payers (HAS)

• Optimizing the quality of the use of medicines
• According to the EMA, DUS belong to post-authorization safety studies (PASS)

contributing to the evaluation of risk management plans

− Evaluation of the proper use of medicines in real life

• Patient profile, indication, dosages, treatment duration, concomitant medication, …

− Evaluation of the effectiveness of risk minimisation measures

• Implementation
• Clinical outcomes

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DUS = Drug Utilization Study; HAS = Haute Autorité de Santé; EMA = European Medicines Agency; PASS = Post-Authorisation Safety Study

Objectives

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Determine the interest of health care databases for the conduct of DUS. Identify the legislative and ethical frameworks relative to the conduct of DUS and, more particularly to the use of health care databaseses in different European countries.

❶ ❷

Methodology

• Literature Review

− January – April 2015

• Two stages

− Systematic review of the ENCePP electronic register of studies

• Extraction of information regarding DUS (objectives, outcomes, data sources,

regulatory requirement, …)

− Review of existing legislative sources in the European countries

• Identification of the legal and ethical requirements applicable for the conduct of DUS

and use of health care databases

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ENcEPP = European Network of Centers for Pharmacoepidemiology and Pharmacovigilance

Results – Phase I (ENCePP Register)

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EMA = European Medicines Agency; BMI= Body Mass Index

173 DUS (planned, ongoing or finalized) 74 (42,8%) conducted using existing health care databases

(secondary data sources)

99 (57,2%) using other data sources

(prospective studies, ad hoc data collection) 95 (54,9%) initiated upon request from a regulatory body (EMA, national health authority, …) Evaluation of drug utilisation modalities in real life clinical practice setting:

• Off-label use
• Patient profiles

Specific objectives:

• Disease characteristics

(biomarker, radiological examination, …)

• Patients characteristics

(BMI, lifestyle factors, …)

• Specific characteristics of the indication

DUS Regulatory Framework

• Observational studies, also called Non-

interventional studies (NIS)

• Important discrepancies across local

legislations

• Heterogeneous legal and ethical

requirements from different institutions regarding observational studies

• Ethics board approval
• Protocol format (national

requirements)

Databases Accessibility

• Different modalities according to the

countries and databases considered

• Authorization from data protection

agencies

• Restricted access to certain institutions
• Access fees

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Results – Phase II (Legal Sources)

NIS = Non-Interventional Studies

Legal and Ethical Requirements – Examples

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Competent Authority

ANSM

Agence nationale de sécurité du médicament et des produits de santé

MHRA

Medicines and Healthcare products Regulatory Agency

DHMA

Danish Health and Medicines Authority

BfArM

Federal Institute for Drugs and Medical Devices

Paul-Ehrlich-Institute

Vaccines, Biomedicines

AIFA

Agenzia Italiana del farmaco

AEMPS

Agencia Española de Medicamentos y Productos Sanitarios Notification to the competent authority No EXCEPT for studies requested by the authorities No

Yes Yes

No At the national- level: No At the regional- level:

Yes

Approval by a research ethic board No

Yes

No

Yes Yes Yes

Study registration No No No

Yes

VFA local register or clinicaltrials.gov

Yes

AIFA and Data Protection Agency No

Databases and Accessibility – Examples

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Databases SNIIRAM-PMSI EGB CPRD National registers GePaRD Accessibility Authorization from the CNIL List of authorized institutions established by ministerial decree Protocol approved by a research ethic board Authorization from the Data Protection Danish institutions authorised by Statistics Denmark Project approval by the health insurance providers and local authorities Persons employed by the BIPS Leibniz Benefits Exhaustiveness ₋Representa- tiveness ₋Depth (20 years)

• Representativeness
• Sample size

(> 52 millions)

• Follow-up duration
• Data linkage
• Single identifier

(linkage)

• Data access

through the internet

• Représentativeness
• Sample size

(17 millions)

• Depth (> 10 ans)

Disadvan- tages No clinical or environmental information (lifestyle, …) Sample Size (1/97th)

• Cost
• Claims database
• Time for access

Conclusion

• Health care databases= Opportunity for the conduct of DUS in a timely

mannner

− Existing data − Interesting for public health

• Early identification of misuse situations
• Implementation of appropriate corrective measures
• Optimization of risk minimization measures
• Databases’ interest depends on study objectives and available data
• Utility of the database mapping to identify fit-for-purposes databases for

the conduct of DUS

− Systematic and pragmatic literature review based on required data − Matrix of identified databases including their characteristics − Usefulness assessment to determine the most appropriate datasources for the DUS

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