An agency of the European Union

How are medicines evaluated at the EMA

Presented by: Nathalie Bere Patient interaction / Stakeholders and communication Division

1 1

Optimised utilisation of resources Harmonised scientific opinions

Harmonised information to healthcare professionals & patients

All Systems allow Centralised Procedure (via EMA)

Mutual Recognition/ Decentralised

Procedure

The European System

2

Centralised procedure

• 1 application

• 1 evaluation

• 1 authorisation for all EU

• 1 invented name

• 1 product information (SPC, Labelling, PL)

• All EU languages

Centralised procedure

The EMA is not responsible for pricing or reimbursement

4

The Agency is responsible for:

• The evaluation of marketing authorisation for human and veterinary applications submitted by pharmaceutical companies

• The coordination of European pharmacovigilance (supervision of the medicines on the market)

• The provision of scientific advice on the development of medicines

• The evaluation of applications for orphan designation in EU

• The evaluation of paediatric investigation plans (or waivers)

• The evaluation of arbitration and referral procedures

• The provision of good quality and independent information on the medicines it evaluates to patients and health

• The coordination of Member States’ inspections (GMP, GCP, GLP)

The various roles of the EMA

5

Eligibility: “Mandatory Scope”

Since May 08

Auto-immune diseases and Other immune dysfunctions

Viral diseases

AIDS Cancer Neurodegenerative disorders

Diabetes

Orphan medicines

Recombinant DNA technology Controlled gene expression Monoclonal AB Since Jan 95

Gene therapy products Somatic Cell therapy products Tissue engineered products

ADVANCED THERAPY MEDICINAL PRODUCTS:

Since Dec 08

6

Eligibility “Optional Scope”

Art 3(3) Generic of a product authorised via EMA

New Active Substances

Significant Innovation

(Therapeutic, &/or Scientific, &/or Technical)

Interest of Patients at

Community Level

OR

The centralised procedure attracts most innovative medicines. Decentralised and MRP mainly do generics and new indications for existing products

7

approval

Type of Approvals

Conditional Approval: • Comprehensive data not available; to be provided after approval • Must fulfil scope (orphan drugs, emergency threats, serious and life-threatening diseases)

Approval valid for 1 year, renewable

Exceptional Circumstances: • Comprehensive data not available and cannot be provided • Must meet criteria (rarity, medical ethics, state of scientific knowledge)

Normal: Comprehensive data

8

28 EEA Member States + 4,500 European experts

EU institutions: Commission - Parliament

Committee for Orphan Medicinal Products

(COMP)

Committee for Herbal Medicinal Products

(HMPC)

EMA

Secretariat

Committee for Veterinary Medicinal Products

(CVMP)

Management Board

Committee for Human

Medicinal Products (CHMP)

Committee for Advanced Therapies

(CAT)

Paediatric Committee (PDCO)

EMA-EU Network

Pharmacovigilance Risk Assessment Committee

(PRAC)

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1 scientific expert member nominated by each MS + 1 alternate 5 co-opted members

Chairperson: Tomas Salmonson

CHMP

Other working parties Biosimilar

Biostatistics Blood Products Cardiovascular

Central Nervous System Infectious Diseases Oncology Working Pharmacogenomics Pharmacokinetics

Rheumatology/Immuno. Vaccines

10

Working Parties and other Groups

SAG diagnostics

SAG Neurology

SAG Psychiatry

SAG HIV /

Antiviral

SAG Oncology

SAG CVS

SAG Diabetes Endoc.

HCPWP Healthcare

professionals

SAG Anti-infectives SAG

Vaccines

ad-hoc expert groups

CMDh Co-ordination Group

for Mutual Recognition and Decentralised

Procedures

PCWP Patients and consumers

BWP Biologics

SWP Safety

QWP Quality

SAWP Scientific advice

GCP Inspectors Working group

QRD Working Group on Quality Review of

documents

Working Parties

Centralised procedure - product life-cycle

CHMP CAT

PRAC

CHMP SAWP

CHMP PRAC

Orphan Designation

Scientific Adv. Protocol assist.

Clinical trials

Paediatric investigation

Post Marketing Authorisation

MAA Evaluation

COMP PDCO

SAGs WPs

CAT

Patient input

SAGs WPs

Patient input

Patient input

Draft PI & RMP

Submission of application

Assessment of Risk

Management Plan

Assessment of Product Information

Assessment on need for post safety/efficacy

studies

Final Product

Information & RMP

Evaluation of benefit/risk

Preparation of RMP summary

CHMP

D1 Start D80 AR D120 LoQ D150 JAR D180 LoOI/OE D210 Opinion D277 CD

Timelines dependent on specific procedure/medicine

RevisedPI

Submission of change

authorisation

Update of Product Information?

Assessment of safety update reports

Decision on need for new post safety

studies

Update of Product

Information /RMP

Re-evaluation of benefit/risk

Update of RMP summary?

EMA - CHMP - PRAC

Signal detection

Annual re-assessment / conditional renewal 5 yr -Renewal

Safety variations

Safety Referrals

Pharmacovigilance and Risk Management

14

What we know at the end of the clinical trial programme…

What we don’t know! . What happens when the medicinal product is used in

normal practice? . What is its adverse event

profile?

Pharmacovigilance Risk Assessment Committee (PRAC)

Assesses aspects of risk management (detection, assessment, minimisation and communication of risk of adverse reactions)

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• 1 member (+ 1 alternate) per MS • + NO & IS

• 6 experts nominated by EC • 1 member (+ 1 alternate)

healthcare professionals • 1 member (+ 1 alternate)

patients organisations

Chair: Dr June Raine

OR

MAH NCA

Pharmacovigilance and Risk Management;

Data Collection and Management

Safety monitoring

Patient with ADR

Healthcare professional

ADR report

Pharmacovigilance and Risk Management;

Signal Detection and Data Analysis

MAH EU

Assessors Signal detection

Other MS

PRAC CHMP

Assessment of the signal

Propose appropriate regulatory action

Acronyms

• ADR = Adverse Reaction

• AR = Assessment Report

• CHMP = Committee for Medicinal Products for Human Use

• CD = Commission Decision

• D1, etc = Day 1 (procedural timeline)

• GCP – Good Clinical Practice

• GLP = Good Laboratory Practice

• GMP = Good Manufacturing Practice

• LoQ = List of Questions

• LoOIs = List of Outstanding Issues

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• MAH = Marketing Authorisation Holder

• MS = Member State

• OE = Oral explanation

• PASS = Post Authorisation Safety Study

• PI = product information

• PRAC = Pharmacovigilance Risk Assessment Committee

• PSUR = Periodic Safety Update Report

• RMP – Risk Management Plan

• SmPC = Summary of Product Characteristics