Joint EFGCP & DIA Better Medicines for Children Conference

From development to access

Description

Event Conference
Type Face To Face
Language English
Date(s) 25 & 26 Oct. 2018
Day(s) 2 Day(s)
Duration 7 hours/ day

The Paediatric Conference “Better Medicines for Children” is an annual event jointly organised by EFGCP and DIA with a focus on new developments in children medicine.
In 2018, the event is proudly brought to the public under the name of “Better Medicines for Children Conference, From Development to Access” by the EFGCP Children Medicines Working Party in collaboration with DIA and Regulators with the aim to take stock of the progress achieved after 10 years of Paediatric Regulation and, to move the discussion toward the experience of patients.
It provides a unique platform where all the stakeholders involved in the process, from research to treatment of children diseases, including HTA and payers, as well as patients are invited to share updates in the field and discuss challenges related to the development of paediatric medicines.

The intention of this year event is to shade light on what could be done to optimise children’s access to new medicines. In the context of this conference, “access” can be broadly defined as :

  1. Inclusion of paediatric patients into clinical trials,
  2. Health authority approval of a new medicine and,
  3. Children’s access to a medicine that has been approved by regulators.

The unique programme of the 2018 event gives a new perspective over current status and developments in the paediatric treatment arena by addressing the three access perspectives.

In the opening, the conference will benefit of keynotes from Francoise Grossetête, Member of the European Parliament (France) and Florian Schmidt from DG SANTE, European Commission, followed by addresses from key speakers representing EMA, FDA, National Authorities, healthcare professionals, clinical researchers and patients.

Interactive breakout sessions will allow all participants to actively engage in the discussion to identify innovative solutions to address access issues specific to paediatric subpopulations, such as adolescents, or neonates. A Q&A session with members of the EMA Paediatric committee (PDCO), moderated by Dr Dirk Mentzer (PDCO chair) will help audience to understand PDCO's specific activities and priorities, and to further address clarifying questions to PDCO members.
A panel discussion held by patients and parents will highlight the priorities from a societal perspective and enable participants to reflect on the goals of paediatric research activities. Stakeholders recognize that more treatment access can be achieved by accelerating development of new medicines. Current delays in clinical trial conduct relating to recruitment difficulties will be addressed, e.g. by optimising the number of children needed for clinical trials, or recruitment strategies.

Information on constructive multi-stakeholder collaborations, which were set up to address the perceived long-term gaps will be provided; an opportunity to understand further what could help in fostering paediatric development and children’s access to new medicines. The conference will end with a panel discussion to explore what needs to be further achieved to meet the specific goal of, increasing children’s access to new treatments.

NEW: By means of a poster session, the conference will also show case some best practice examples of paediatric research & developments that address unmet medical needs in children and that have been successfully developed under the EU Paediatric Regulation.
The presenting authors of the winning posters will have the possibility to publish an abstract of their work in “Therapeutic Innovation & Regulatory Science” magazine.

Programme

Thursday 25 October 2018
08:30 Welcome & Introduction
Dirk Mentzer, EMA PDCO Chair, UK & PEI, Germany
Martine Dehlinger-Kremer, EFGCP & ICON plc, Germany
Session 1: Setting the Scene - What is Done in Europe to Enhance Children Access to Medicines Chaired By: Dimitrios Athanasiou, PDCO EMA, World Duchenne Organization / UPPMD and MDA Hellas, Greece Solange Corriol-Rohou, AstraZeneca, GMD, France
08:45 Keynote Speeches
Florian Schmidt, DG SANTE, EU Commission
Françoise Grossetête, Member of the European Parliament
09:00 Panel Discussion
Regulator view
Dirk Mentzer, EMA PDCO Chair, UK & PEI, Germany
HTA view
Edith Frénoy,
Payer view
Ri de Ridder, Doctors of the World, Belgium
Patient view
Virginie Hivert, Eurordis, France
Industry view
Samuel Blackman, CureSearch, USA
Healthcare provider view
Dominique Haumont, Neonatal Medicine, Saint-Pierre University Hospital Brussels, Belgium
10:30 Questions and Answers
11:00 Coffee Break
Session 2: Meet the Expert Sessions - Enabling Access for Different Paediatric Populations Christina Bucci-Rechtweg, Novartis Pharmaceuticals Corporation, USA
11:15 Breakout Sessions
BOS 1 Adolescents Dirk Mentzer, EMA PDCO Chair, UK & PEI, Germany
Samuel Blackman, CureSearch, USA
Solange Corriol-Rohou, AstraZeneca, GMD, France
BOS 2 Neonates Dina Apele-Freimane, P. Stradins Clinical University Hospital, PDCO Member, Latvia
Dominique Haumont, Neonatal Medicine, Saint-Pierre University Hospital Brussels, Belgium
Martine Dehlinger-Kremer, EFGCP & ICON Plc, Germany
BOS 3 Children Marieke van Meel, NephcEurope Foundation, The Netherlands
Angelika Joos, MSD (Europe), Belgium
Ri de Ridder, Doctors of the World, Belgium
Session 3: Access Through Approval: View of the Regulators and Q&A Session with the PDCO Dirk Mentzer, EMA PDCO Chair, UK & PEI, Germany Martine Dehlinger-Kremer, EFGCP & ICON Plc, Germany
14:15 Q&A with PDCO Members
Dirk Mentzer, EMA PDCO Chair, UK & PEI, Germany
Hugo Tavares, Hospital Centre Vila Nova de Gaia / Espinho, Portugal
Koenraad Norga, Paediatric Oncology, University Hospital Antwerp, Belgium
Ninna Gullberg, Medical Product Agency, Sweden
15:30 Coffee Break
Session 4: Access after Marketing Authorisation - Sharing Experience from the Patient's Perspective Dimitrios Athanasiou, PDCO EMA, World Duchenne Organization / UPPMD and MDA Hellas, Greece Katie Rizvi, Youth Cancer Europe, Romania
16:00 An overview on the different systems of patients access in Europe
Solange Corriol-Rohou, AstraZeneca, GMD, France
16:15 Case study: Patient perspective
Elizabeth Vroom, United Parent Projects Muscular Dystrophy, The Netherlands
16:30 Spinraza case study: Patient perspective
Mencía de Lemus, SMA Europe, Spain
16:45 Access to essential medicines
Anca Colita, Fundeni Clinical Hospital, Romania
17:00 Payer perspective
Ri de Ridder, Doctors of the World, Belgium
17:15 Panel discussion Dimitrios Athanasiou, PDCO EMA, World Duchenne Organization / UPPMD and MDA Hellas, Greece Katie Rizvi, Youth Cancer Europe, Romania
17:30 POSTER SESSION Claudio Fracasso, Pfizer, Italy
18:00 Social Event (The Hotel, networking cocktail)
Chris Copland, ACCELERATE platform’s FAIR, Unite2Cure, National Cancer research Institute, United Kingdom
Friday 26 October 2018
08:15 Welcome Coffee
Session 5: Access Through Clinical Trials: Enhancing Effectiveness in Paediatric Clinical Trials Sabine Fürst-Recktenwald, F. Hoffmann-La Roche, Switzerland
08:45 EnprEMA Working Group on Clinical Trial Preparedness
Angeliki Siapkara, MHRA, United Kingdom
09:05 Connect4Children - Pan-European Paediatric Clinical Trial Network
Mark Turner, University of Liverpool, United Kingdom
09:25 Think Big Project Proposal - Remote and Decentralised Clinical Trials
Kimberly Hawkins, Sanofi, France
09:45 Use of wearables in Paediatric Clinical Trials - the experience of Aparito
Elin Haf Davies, Aparito, United Kingdom
10:05 General Discussion
10:45 Coffee Break
Session 6: Facilitating Access : Multistakeholders' Collaborations & Best Practices Angelika Joos, MSD (Europe), Belgium Marieke van Meel, NephcEurope Foundation, The Netherlands
11:15 Identification of Paediatric Needs
Begonya Nafria Escalera, Patient Engagement in Research Coordinator, Research Institute Sant Joan de Déu, Spain
11:30 Multistakeholder Paediatric Strategy Forum
Gilles Vassal, Institut Gustave Roussy, France
11:45 European Reference Networks
Ruth Ladenstein, ERN, Austria
12:00 Panel Discussion
Dirk Mentzer, EMA PDCO Chair, UK & PEI, Germany
Marieke van Meel, NephcEurope Foundation, The Netherlands
Samuel Blackman, CureSearch, USA
12:45 General Discussion Q&A
13:15 Lunch
Session 7: Changing the Landscape of Paediatric Research & Access Duane Schulthess, VitalTransformation, Belgium
14:15 Introduction & Panel Discussion
FDARA: what to expect in the US?
Gregory Reaman, CDER, FDA/OCE, USA
Do our children get access to the new medicines? Nordic Experience
Pirkko Lepola, Helsinki University Hospital, Department of Children and Adolescents, Finland
HTA view
Payer view
Menno Aarnout, International Association of non-profit healthcare payers, (AIM), Belgium
Regulator view
Dirk Mentzer, EMA PDCO Chair, UK & PEI, Germany
Company view
Marie-Yvonne Douste-Blazy, Servier, France
Changing the landscape of paediatric research: Real world data, real world experience and data protection
Dominique Haumont, Neonatal Medicine, Saint-Pierre University Hospital Brussels, Belgium
Patient view
Emily Crossley, Duchenne / HERCULES project, United Kingdom
15:30 Conclusions
Dirk Mentzer, EMA PDCO Chair, UK & PEI, Germany
Martine Dehlinger-Kremer, EFGCP & ICON Plc, Germany
16:00 End of Conference