CDDF CDDF-ITCC-SIOPE PAEDIATRIC ONCOLOGY PLATFORM PLATFORM WORKING GROUPS

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1 CDDF PLATFORM CDDF-ITCC-SIOPE PAEDIATRIC ONCOLOGY PLATFORM WORKING GROUPS

2 WORKING GROUP 1 New Strategies for Improved Development of Oncology Drugs for Children and Adolescents Lead: Andy Pearson (Innovative Therapy for Children with Cancer Consortium) 1. To develop and publish a model of mechanism of action (MoA), biology-driven drug development, with prioritisation of drugs within the current regulations. 2. To develop the aggregated database of paediatric tumour targets as part of the joint ITCC Personalised Cancer Medicine EU Paediatric Program. 3. To develop a lifecycle-approach an iterative approach where the direction of drug development is continually reviewed especially after the conclusion of pivotal clinical trials. 4. To develop a strategy for the paediatric development of multiple compounds of the same class where the number of children with tumours expressing the target is very limited. 5. To prioritise drugs within pipelines across companies (especially when several companies develop drugs against same target) i.e. to produce an aggregated pipeline of drugs -- early portfolio analysis. 6. To implement the inclusion of teenagers (thirteen years of age and older) in adult early phase clinical studies. Deliverables: Publication describing a model of MoA biology driven drug development with prioritisation of drugs December 2015 Plan for the implementation of the inclusion of teenagers (thirteen years of age and older_ in adult early phase clinical studies leading to a publication December 2015 Aggregated database of paediatric tumour targets 2016 Description of a life cycle approach to paediatric cancer drug development Strategy for the paediatric development of multiple compounds of the same class Process for prioritisation of drugs An aggregated pipeline of drugs The proposed model of MoA biology-driven drug development would align an aggregated pipeline of drugs with an aggregated database of paediatric tumour targets and enable the conduct of early phase clinical paediatric trials to rapidly and efficiently evaluate a drug against a target and generate information in less than two years. It is proposed that this model is delivered through a life-cycle approach (which is an iterative method where the direction of drug development is continually reviewed especially after the conclusion of pivotal clinical trials [early phase clinical trials, randomised multi-arm trials]). - Academia: Andy Pearson (The Institute of Cancer Research, UK), Gilles Vassal (Gustave Roussy Institute), Louis Chesler (The Institute of Cancer Research / The Royal Marsden NHS Foundation Trust), Nathalie Gaspar (Institut Gustave Roussy), Birgit Geoerger (Gustave Roussy Institute), Pamela Kearns (University of Birmingham), Lynley Marshall (The Institute of Cancer Research / The Royal Marsden NHS Foundation Trust), Lucas Moreno (University Hospital of Niño Jesús), Stefan Pfister (Heidelberg University Hospital), Gudrun 1

3 Schleiermacher (Curie Institute), Olaf Witt (Deutsches Krebsforschungszentrum Heidelberg), Beate Wulff (University Hospital of Essen) - Regulators: Jacqueline Carleer (Federaal Agentschap voor Geneesmiddelen en Gezondheidsproducten), Ralf Herold (EMA), Koen Norga (University Hospital Antwerpen), Jaroslav Sterba (University Hospital Brno), Hendrick van den Berg (Medicines Evaluation Board) - Pharma: Brigid Bradley-Garelik (BMS), Renaud Capdeville (Novartis), Anne Charoin Pannier, Hubert Caron (Hoffmann-La Roche), Robert Iannone (AstraZeneca), Regina Jakacki (AstraZeneca), Raphael Rousseau (Genentech), Mark Russo (GSK), Jeffrey Skolnik (TetraLogic Pharmaceuticals), Tillmann Taube (Boehringer Ingelheim Pharma), Martina Uttenreuther- Fischer (Boehringer Ingelheim) - Parents: Debbie Binner, Chris Copland (National Cancer Research Institute), Cesare Spadoni (apodd Foundation) Operational Approach of the Group: Through TCs, s and face-to-face meetings. 2

4 WORKING GROUP 2 New Incentives for Specific Paediatric Drug Development and Drug Repositioning Lead: Patricia Blanc (Imagine for Margo, France) The aim is to propose best incentives to develop specific paediatric drugs and reposition the drugs developed for adults for children, and so Create Hope also in Europe based on a contextualised US model. Strategy to move forward: 1. Identify a range of experts who can generate ideas; 2. Interview these experts by challenging them with targeted questions; 3. Produce a summary of main obstacles to invest in paediatrics drugs and a list of proposals to reduce those obstacles 4. Analyse the Creating Hope Act regulation on incentives in the USA and its adaptation in Europe 5. Define what would be the most efficient and feasible incentives in Europe 6. Have these final selected incentives presented at the January event along with an intervention from EMA regarding implementation challenges. Deliverable for the January 2016 meeting: Presentation of a List of Incentives that fall under the scope of EU law and that can be proposed for new legislation or/and amendment of existing legislation. - Academia: Riccardo Riccardi (Università Cattolica Roma), Judith Landman-Parker (Armand Trousseau Hospital), Natalie Hoog Labouret (Institut National du Cancer), François Doz (Institut Curie), Michel Zwaan (Erasmus MC), Pamela Kearns (University of Birmingham) - Regulators: Koenraad Norga (Paediatric Oncology Antwerp University Hospital, PEDCO vicepresident), - Parents: Nancy Goodman (Kids vs Cancer), Patricia Blanc (Imagine for Margo), Gerlind Bode (Stellv. Vorsitzende Förderkreis für krebskranke Kinder und Jugendliche Bonn e.v.) and Childhood Cancer International), Lydie Meheus (AntiCancer Fund), Cesare Spadoni (apodd Foundation), Chris Copland (National Cancer Research Institute) - Industry: Bouchra Benettaib (Celgene), Jeffrey Skolnik (TetraLogic Pharma), Stefan Schwoch (Lilly) Operational Approach of the Group: Through TCs, s and one face-to-face meeting. 3

5 WORKING GROUP 3 Implementation of Long-term Follow-up Measures of Children and Adolescents Receiving New Anticancer Drugs Lead: Raphael Rousseau (Genentech, USA) The aim is to create a common proposal that can be implemented as a position paper. This document will aim at: 1. Presenting the current status on LTFU initiatives as part of the survivor s clinic from an academic stand point and as part of the LTFU program set by various companies. 2. Defining a proposal for a common set of guidelines. Joint forces between academia, industry, parents group and ultimately regulators to designing specific LTFU measures in their respective environment. Deliverables: produce a position paper on existing and future long-term follow up initiatives that would fit the needs of all 4 stakeholders (parents, academics, regulators and industry) - Academia: Gilles Vassal (Institut Cancérologie Gustave Roussy), Lars Hjorth (PanCare Association), Brice Fresneau (Institut Cancérologie Gustave Roussy), Leontien Kremer (Emma Children s Hospital), Rod Skinner (PanCareSurFup), Riccardo Haupt (ENCCA, PanCareSurFup), Ruth Ladenstein (Children s Cancer research Institute) - Industry: Christina Theodore-Oklota (Genentech), Davy Chiodin (Genentech), Raphael Rousseau (Genentech), Wouter Hanekom (Takeda), Armela Joset (Novartis), Ellen Bolotin (Bayer), Bouchra Benettaib (Celgene), Jürgen Maares (Novartis) - Regulators: Ralf Herold (EMA), Koen Norga (Antwerp University Hospital & EMA) - Parents / Patients: Danielle Horton Taylor (PORT), Nicole Scobie (ZOE4LIFE), Peter Lack (Childhood Cancer International), Jaap den Hartogh (Dutch Childood Cancer Parent Organization) Operational Approach of the Group: Through Conference Calls, and one face-to-face meeting 4