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

How are medicines evaluated at the EMA Presented by: Nathalie Bere Patient interaction / Stakeholders and communication Division An agency of the European Union

The European System Mutual Centralised Recognition/ Procedure Decentralised All System s allow ( via EMA) Procedure Optim ised utilisation of resources Harm onised scientific opinions Harm onised inform ation to healthcare professionals & patients 1 1

Centralised procedure 2

Centralised procedure • 1 application • 1 evaluation • 1 authorisation for all EU • 1 invented name • 1 product information (SPC, Labelling, PL) • All EU languages The EMA is not responsible for pricing or reimbursement

The various roles of the EMA 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 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 inform ation on the medicines it evaluates to patients and health • The coordination of Member States’ inspections (GMP, GCP, GLP) 4

Eligibility: “Mandatory Scope” ADVANCED THERAPY MEDI CI NAL PRODUCTS: Gene therapy products Auto-im m une diseases and Other im m une dysfunctions Som atic Cell therapy AI DS Cancer Neurodegenerative products disorders Viral Recom binant DNA diseases technology Diabetes Tissue engineered Controlled gene products expression Orphan m edicines Monoclonal AB Since Jan 9 5 Since May 0 8 Since Dec 0 8 5

Eligibility “Optional Scope” Significant I nterest of New Active I nnovation Patients at Substances Com m unity OR ( Therapeutic, Level &/ or Scientific, &/ or Technical) Art 3(3) Generic of a product authorised via EMA The centralised procedure attracts most innovative medicines. Decentralised and MRP mainly do generics and new indications for existing products 6

Type of Approvals Norm al: Comprehensive data Exceptional Circum stances: • Comprehensive data not approval available and cannot be provided • Must meet criteria (rarity, medical ethics, state of scientific knowledge) 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 7

EMA-EU Netw ork 28 EEA Member States EU institutions: + 4,500 European experts Commission - Parliament Management Board Committee for Veterinary Committee for Human Medicinal Products Medicinal Products (CHMP) (CVMP) EMA Committee for Orphan Paediatric Committee Secretariat Medicinal Products (PDCO) (COMP) Committee for Herbal Pharmacovigilance Risk Committee for Medicinal Products Assessment Committee Advanced Therapies (HMPC) (PRAC) (CAT) 8

CHMP 1 scientific expert m em ber nom inated by each MS + 1 alternate 5 co-opted m em bers Chairperson: Tomas Salmonson 9

W orking Parties and other Groups CMDh W orking Parties Co-ordination Group for Mutual Recognition SAW P QW P and Decentralised Scientific advice Procedures Quality Other w orking parties Biosimilar Biostatistics Blood Products SAG SW P Cardiovascular HI V / SAG Anti- Central Nervous System Safety SAG Antiviral infectives Infectious Diseases Vaccines Oncology Working SAG Pharmacogenomics ad-hoc Diabetes Pharmacokinetics SAG Rheumatology/ Immuno. Endoc. expert diagnostics Vaccines groups BW P SAG CVS SAG Psychiatry Biologics SAG SAG Oncology Neurology QRD Working Group on PCW P HCPW P Quality Review of GCP Inspectors Patients and documents Healthcare Working group consumers professionals 10

Centralised procedure - product life-cycle Orphan Clinical Scientific Adv. Post Marketing Paediatric MAA Evaluation trials Protocol assist . Authorisation Designation investigation Patient Patient Patient input input input CHMP PDCO CHMP COMP CHMP SAW P PRAC CAT PRAC CAT SAGs W Ps SAGs W Ps

Subm ission of application CHMP D1 Start D80 AR D120 LoQ D150 JAR D180 LoOI / OE D210 Opinion D277 CD Draft PI & RMP Assessment on Evaluation of Final need for post benefit/ risk Product safety/ efficacy I nform ation studies & RMP Assessment of Product Assessment Information of Risk Preparation of RMP Management summary Plan

Subm ission of change authorisation EMA - CHMP - PRAC Timelines dependent on specific procedure/ medicine Revised PI Signal detection Decision on need for new Update of post safety Product Re-evaluation I nform ation studies of benefit/ risk / RMP Update of Product Information? Update of RMP Assessment of safety summary? update reports Annual re-assessment Safety Safety 5 yr -Renewal / conditional renewal variations Referrals

Pharm acovigilance and Risk Managem ent What we don’t know! What we know at the end of the clinical trial programme… . What happens when the medicinal product is used in normal practice? . What is its adverse event profile? 14

Pharm acovigilance Risk Assessm ent Com m ittee ( PRAC) Assesses aspects of risk management (detection, assessment, minimisation and communication of risk of adverse reactions) • 1 member (+ 1 alternate) per MS • + NO & IS • 6 experts nominated by EC • 1 member (+ 1 alternate) healthcare professionals Chair: Dr June • 1 member (+ 1 alternate) Raine patients organisations 15

Pharm acovigilance and Risk Managem ent; Data Collection and Managem ent Patient with ADR OR MAH NCA ADR report Healthcare professional Safety monitoring

Pharm acovigilance and Risk Managem ent; Signal Detection and Data Analysis EU MAH Assessors Signal detection Assessm ent Other MS of the signal CHMP PRAC Propose appropriate regulatory action

Acronym s • ADR = Adverse Reaction • AR = Assessment Report • CHMP = Committee for Medicinal Products for Human Use • CD = Commission Decision • MAH = Marketing Authorisation Holder • D1, etc = Day 1 (procedural timeline) • MS = Member State • GCP – Good Clinical Practice • OE = Oral explanation • GLP = Good Laboratory Practice • PASS = Post Authorisation Safety Study • GMP = Good Manufacturing Practice • PI = product information • LoQ = List of Questions • PRAC = Pharmacovigilance Risk Assessment Committee • LoOIs = List of Outstanding Issues • PSUR = Periodic Safety Update Report • RMP – Risk Management Plan • SmPC = Summary of Product Characteristics 18