How are medicines evaluated at the EMA Presented by: Nathalie Bere Patient interaction / Stakeholders and communication Division An agency of the European Union
Overview of medicines development Safety and Drug discovery Identification of Non-clinical Non-clinical Efficacy tests candidate testing Phase I Clinical Phase II/III Regulatory Submission of Availability and Approval after marketing pricing dependent evaluation by authorisation upon individual authorities request country Marketing Phase IV Post- 1
The European System Mutual Centralised Recognition/ National Procedure All Systems allow Decentralised Procedures Procedure Optimised utilisation of resources Harmonised scientific opinions Harmonised information to healthcare professionals & patients 2 2
EMA: focal point of the centralised procedure EU languages Marketing Authorisation application Evaluation Authorisation in all EU Invented name Product information (Summary of Product Characteristics (SmPC), Labelling, Package Leaflet (PL)) 3
Which medicines are mandatory for evaluation at the EMA? • Rare diseases • HIV, cancer, neurodegenerative disorders, diabetes • Auto-immune diseases, viral diseases • All biotech products • Gene therapy • Monoclonal antibodies + Other innovative products The EMA is not responsible for pricing or reimbursement 4
Eligibility “Optional Scope” Significant Interest of New Active Innovation Patients at Substances OR Community (Therapeutic, Level and/or Scientific, and/or Technical) Medicines outside the mandatory scope can also be evaluated at EMA if they meet certain criteria. 5
The various roles of the EMA 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 healthcare professionals • The coordination of Member States’ inspections 6
Medicines Lifecycle: Development and Regulatory Regulatory Procedure Paediatric Orphan Scientific Advice Marketing Authorisation Post Marketing Investigation Designation Protocol assistance Application Evaluation Authorisation Plan Medicines Development Process Drug Candidate Non-clinical Clinical Development Phase IV Discovery Identification tests 7
Type of Approvals Normal: Comprehensive data Exceptional Circumstances: Comprehensive data not • available and cannot be approval 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 8
EMA-EU Network 28 EEA Member States EU institutions: + 4,500 European experts Commission - Parliament Management Board Committee for Human Committee for Veterinary 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) 9
CHMP * 1 scientific expert member nominated by each member state + 1 alternate 5 co-opted members Chair: Tomas Salmonson 10 *Committee for Human Medicinal Products
Working Parties and other Groups CMDh Working Parties Co-ordination Group for Mutual Recognition SAWP QWP and Decentralised Scientific advice Procedures Quality Other working parties Biosimilars Biostatistics Blood Products SWP HIV / Cardiovascular Anti- Safety Central Nervous System Antiviral infectives Infectious Diseases Vaccines Oncology Working Diabetes Pharmacogenomics Diagnostics ad-hoc Endocrinol Pharmacokinetics Rheumatology/Immuno. expert ogy Vaccines Cardio groups vascular BWP Psychiatry issues Biologics Neurology Oncology QRD Working Group on PCWP HCPWP Quality Review of GCP Inspectors Patients and documents Healthcare Working group consumers professionals 11
CHMP Joint List of Outstanding Assessment List of Commission Start Assessment Issues/ Oral Opinion Report Questions Decision Report explanation Day 210 Day 1 Day 80 Day 120 Day 277 Day 150 Day 180 Evaluation of Assessment on need for post benefit/risk safety/efficacy studies Assessment of Product Information Assessment Preparation of RMP of Risk summary Management Plan 12
EMA - CHMP - PRAC Timelines dependent on specific procedure/medicine Signal detection Decision on need for new post safety Re-evaluation studies of benefit/risk Update of Product Information? Update of RMP Assessment of safety summary? update reports Annual re-assessment Safety Safety 5 year / conditional renewal variations Referrals Renewal 13
Pharmacovigilance and Risk Management 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? 14
Pharmacovigilance Risk Assessment Committee (PRAC) Assesses aspects of risk management (detection, assessment, minimisation and communication of risk of adverse reactions) • 1 member (+ 1 alternate) per Member State • plus Norway & Iceland • 6 experts nominated by EC • 1 member (+ 1 alternate) Chair: healthcare professionals Dr June Raine • 1 member (+ 1 alternate) patients organisations 15
Pharmacovigilance and Risk Management; Data Collection and Management Patient with OR Adverse Drug National Reaction Competent (ADR) Authority (NCA) Marketing Authorisation Holder ADR report (MAH) Healthcare professional Safety monitoring 16
Package Leaflets (PL) Documents for the Public EPAR summaries Patient Public input Risk Management Plan Package Leaflets (PL) Safety Summaries summaries (renewal) Communications of Opinion (tentative) Patient (PSO) Patient Patient Patient input input input input Regulatory Procedure Paediatric Orphan Scientific Advice Marketing Authorisation Post Marketing Investigation Designation Protocol assistance Application Evaluation Authorisation Plan Committee s and Working Parties CHMP CHMP- CHMP COMP PDCO CAT SAWP PRAC PRAC Patient Patient Patient Patient Patient input input input input input CAT SAG SAG Patient Patient Patient input input input 17
Acronyms • 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 • SAG = Scientific Advisory Group • SmPC = Summary of Product Characteristics 18
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