Enpr-EMA Joint DIA/EFGCP/EMA Paediatric Forum 26 September 2011 Paolo Rossi Chairman Dept. of Pediatrics Tor Vergata University-Children s Hospital Bambino Gesù, Rome EnprEMA-PDCO Representative An agency of the European Union European Paediatric Network Legal Basis European Paediatric Regulation: 1
When should the PIP be requested? MA Non-clin Phase 1 Phase 2 Phase 3 Post approval 3 Paediatric Investigation Plan (PIP Amendments) Paediatric Committee (PDCO) The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi Compliance check Therapeutic areas of applications (all applications, 2007 today) Uro-nephrology 2% Pneumology - Allergology 17% Oto-rhino-laryngology 2% Oncology 10% Pain 4% Psychiatry 2% Other 4% Ophthalmology 3% Nutrition 0% Vaccines 4% Neurology 5% Anaesthesiology 1% Dermatology 4% Diagnostic 1% Neonatology - Paediatric Intensive Care 1% Cardiovascular Diseases 9% Endocrinology- Gynaecology-Fertility- Metabolism 10% Gastroenterology- Hepatology 3% Haematology- Hemostaseology 4% Immunology-Rheumatology- Transplantation 7% Infectious Diseases 6% 2
An Approximate Analysis of Economical Impact of Pediatric Regulation (next 10 Years) The evaluation of 270 PiPs out of the 950 ongoing estimates tha 307 Trials will be performed. Calculated Cost to Conduct Trials : 270 Mln Euros Estimated total cost in the next 10 Years 1,2 Billions Euros CRO cost to organize-monitor-data management of Trials Approximate value: 1.3 Billions Euros Aggregate Economical Value (in the next 10 years ) 2.5 Billions Euros A substantial proportion of patients in adult CTs is from outside the EU 3
European Paediatric Network Legal Basis Key operation goals 4
History of Enpr-EMA EMA Management Board adopted an implementing strategy for the network on 15 January 2008 May 2010, participants from 38 national research networks and clinical trial centres and EMA agreed an organisational structure for the network. membership of Enpr-EMA was opened to networks that fulfil the requirements laid down in the recognition criteria. EMA published a full list of the networks that applied for Enpr-EMA membership in January 2011, indicating those that met all of the criteria and those that needed to provide further clarification or did not yet qualify Enpr-EMA workshops Every year, Enpr-EMA holds a workshop at the EMA in London: First workshop on European paediatric network (16/02/2009) Second workshop on European paediatric network (16/03/2010) Third workshop on network of paediatric research at the EMA (10-11/03/2011) 5
International collaboration Enpr-EMA works with international partners specialising in the regulation of medicines for children. It works with: World Health Organization through the European Medicines Agency's membership of the Paediatric Medicines Regulators' Network (PmRN); United States Food and Drug Administration (FDA) through the Agency's existing interaction on paediatric therapeutics. Enpr-EMA membership total of 33 networks and centres have submitted self-assessment reports: Category 1 includes the 18 networks and centres that are now members of Enpr-EMA; Category 2 includes the networks and centres currently undergoing clarification before membership of Enpr-EMA (1 centre) Category 3 includes the networks and centres that do not currently qualify for membership (14 centres) 6
Proposed Structure Coordinating Group ENPREMA Coordinating Group (20) 3 year membership EC (DG) 13 After the PIP Decision: modification of the agreed PIP, validation, compliance check members of Enpr-EMA European Cystic Fibrosis Society - Clinical Trials Network (ECFS-CTN) EUNETHYDIS (the European Network for Hyperkinetic Disorders) European paediatric oncology off-patent medicines consortium (EPOC) FINPEDMED- Finnish Investigators Network for Pediatric Medicines German Neonatal Network (GNN) Innovative Therapies for Children with Cancer (ITCC) International BFM Study Group (I-BFM-SG) FIMP - MCRN (Italian Paediatric Federation- Medicines for Children Research Network) Medicines for Children Research Network (MCRN) Mother Infant Child Youth Research Network "MICYRN National Institute for Health Research (NIHR) Medicines for Children Research Network (MCRN) Newcastle CCLG Pharmacology Studies Group PENTA: Paediatric European Network for the Treatment of AIDS Pediatric Rheumatology International Trials Organisation (PRINTO) Scottish Medicines for Children Network UKPVG - United Kingdom Paediatric Vaccines Group EBMT PD WP European Group for Blood and Marrow Transplantation Paediatric Network of Clinical Investigation Centers - CICPed 7
Why PDCO is important for EU paediatric network? PDCO DEMAND how to connect? OFFERING deriving from new EU Regulation PIP paediatric studies Chosen by the Companies Driven by PDCO NETWORKS Why PDCO is important for EU paediatric network? PDCO as an employment agency (organization and transparency) BENEFITS reduction in timing for recruiting patients high quality of the planned studies (high standards of the Networks) assess paediatric Needs and avoid the replication of the studies 8
Key Operational points for EmprEMA The role of EMA staff, PDCO and PIPs How industry can refer and use Enpr-EMA How patients can be involved in networks and in trials The role of EMA staff, PDCO and PIPs PDCO - Ensure regular interaction ti ENPREMA/PDCO how? (regular meetings, forums, written interactions?) - Early involvement of networks before PIP submission - PDCO to decide priority list of model PIPs - Conflict of interest: experts for network/applicant/pdco - Publication of expert opinion when general 9
How industry can refer and use Enpr-EMA Identification of therapeutic needs for future research, advise PDCO on priorities Help identify best standard of care in Europe Scientific input for PIP design: both ways, EMA and Industry Cooperation between EU networks with EMA/industry, cooperation with US networks How industry can refer and use Enpr-EMA Aligning study protocols with patient needs, scientific needs, and paediatric practice (driver for feasibility) Resources for investigator sites Provide resources for high-quality study conduct Training to sites for high-quality study conduct 10
How industry can refer and use Enpr-EMA How and When to use Network Consulting role Early dialog with network before PIP application on paediatric strategy with focus on feasibility timelines, patients availability, competing studies 2 nd step dialog when finalizing PIP/studies (eg, clock-stop) Generator of facilitation tools Model PIPs in collaboration with PDCO Agreement on age-appropriate endpoints Discussion about safety aspects Industry input to Network Shared expertise in study design, training to sites Share data with network How patients can be involved Added value of patient involvement: Information (informed consent) Need to be complete vs be understood Two levels of information? Public awareness of the need of CTs Involvement in ethics committee Participation vs consultation? Lobbing industry in deciding on paediatric research. Lobbing public research programmes (e.g. areas where no paediatric development exist) 11
How patients can be involved How to improve interaction/communication among all parties: The example of HIV patients groups (well informed patients) Link EmprEMA with registry of existing networks/organisations at national level (e.g. in the field of neonatology) Link EmprEMA with EMA list of European eligible organisations with interest in paediatrics Make them operational Implications for PDCO Modalities of regular interaction Enpr-EMA/PDCO EMA/PDCO? (regular meetings, forums, written interactions?) Model PIPs in collaboration with Enpr-EMA How to improve exchange of information with Patient organisations? CoI: experts for network/applicant/pdco 12
Improving the system 13