How are medicines evaluated at the EMA Presented by: Nathalie Bere - - PowerPoint PPT Presentation

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

Optim ised utilisation of resources Harm onised scientific opinions Harm onised inform ation to healthcare professionals & patients

All System s 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 m arketing authorisation for hum an and veterinary

applications submitted by pharmaceutical companies

• The coordination of European pharm acovigilance (supervision of the medicines
• n 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 inform ation 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 0 8

Auto-im m une diseases and Other im m une dysfunctions Viral diseases AI DS Cancer Neurodegenerative disorders Diabetes Orphan m edicines Recom binant DNA technology Controlled gene expression Monoclonal AB

Since Jan 9 5

Gene therapy products Som atic Cell therapy products Tissue engineered products ADVANCED THERAPY MEDI CI NAL PRODUCTS:

Since Dec 0 8

6

Eligibility “Optional Scope”

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

New Active Substances Significant I nnovation ( Therapeutic, &/ or Scientific, &/ or Technical) I nterest of Patients at Com m unity 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 Circum stances:

• Comprehensive data not

available and cannot be provided

• Must meet criteria

(rarity, medical ethics, state of scientific knowledge) Norm al: 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 Netw ork

Pharmacovigilance Risk Assessment Committee (PRAC)

9

1 scientific expert m em ber nom inated by each MS + 1 alternate 5 co-opted m em bers

Chairperson: Tomas Salmonson

CHMP

Other w orking parties Biosimilar Biostatistics Blood Products Cardiovascular Central Nervous System Infectious Diseases Oncology Working Pharmacogenomics Pharmacokinetics Rheumatology/ Immuno. Vaccines

10

W orking Parties and other Groups

SAG diagnostics SAG Neurology SAG Psychiatry SAG HI V / Antiviral SAG Oncology SAG CVS SAG Diabetes Endoc. HCPW P Healthcare professionals SAG Anti- infectives SAG Vaccines

ad-hoc expert groups

CMDh Co-ordination Group for Mutual Recognition and Decentralised Procedures

PCW P Patients and consumers BW P Biologics SW P Safety QW P Quality SAW P Scientific advice

GCP Inspectors Working group QRD Working Group on Quality Review of documents

W orking Parties

Centralised procedure - product life-cycle

CHMP CAT PRAC CHMP SAW P CHMP PRAC Orphan

Designation Scientific Adv. Protocol assist. Clinical trials Paediatric investigation Post Marketing Authorisation MAA Evaluation

COMP PDCO SAGs W Ps CAT

Patient input

SAGs W Ps

Patient input Patient input

Draft PI & RMP Subm ission

• f application

Assessment

• f Risk

Management Plan Assessment of Product Information Assessment on need for post safety/ efficacy studies

Final Product I nform ation & 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 Revised PI Subm ission of change authorisation

Update of Product Information? Assessment of safety update reports Decision on need for new post safety studies

Update of Product I nform ation / RMP

Re-evaluation

• f benefit/ risk

Update of RMP summary? EMA - CHMP - PRAC Signal detection Annual re-assessment / conditional renewal 5 yr -Renewal Safety variations Safety Referrals

Pharm acovigilance and Risk Managem ent

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?

Pharm acovigilance Risk Assessm ent Com m ittee ( PRAC)

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

15

• 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

Pharm acovigilance and Risk Managem ent; Data Collection and Managem ent

Safety monitoring Patient with ADR Healthcare professional ADR report

Pharm acovigilance and Risk Managem ent; Signal Detection and Data Analysis

MAH EU Assessors Signal detection Other MS PRAC CHMP Assessm ent

• f the signal

Propose appropriate regulatory action

Acronym s

• 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

18

• 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