Joint EFGCP & DIA Better Medicines for Children Conference on

October, 202012 & 13

VIRTUAL CONFERENCE

The future of paediatric medicines:putting patients at the heart of innovation

and regulatory science

where science and ethics meet

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Agenda at a Glance

MONDAY 12 OCTOBER 202012:00 l Opening of conference

12:30 l Welcome and introduction to the conference

OPTIMISING THE IMPLEMENTATION OF THE PAEDIATRIC REGULATION

12:45l Keynote: Where has progress been made and what remains to be discussed?l Panel discussionl Q&A session

VOICE OF YOUNG PATIENTS INCLUDING Q&A

14:10l What does the future of paediatric drug development mean and look like for Young patientsl Three young patients

14:40 l Introduction to the breakout sessions

14:50 Break

BREAK – OUT SESSIONS

15:10l Extrapolation / RWD in drug development l Identifying paediatric unmet needsl Regulatory overview: Optimising PIP processes and regulatory approval (PDCO & CHMP)

17:00 Break

17:10 l Fireside Chat

18:10 WHAT DRUG DEVELOPMENT MEANS FOR PAEDIATRICIANS

18:55 Break

19:00 Special Event

TUESDAY 13 OCTOBER 202010:30 Debrief from Day one and introduction to Day 2

10:45 PANEL SESSION ON PATIENT ACCESS

12:15 ENPR-EMA: CLINICAL TRIAL PREPAREDNESS & INTERNATIONAL COLLABORATION WORK GROUP

12:35 l Debrief & Conclusions from the breakout sessions

13:05 THE GO- FAIR INITIATIVE / USE DATA

13:35 THE FAIR (FOSTERING AGE INCLUSIVE RESEARCH) INITIATIVE – INCLUSION OF ADOLESCENT

IN ADULT TRIALS

14:20 l Conclusions & Farewell

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This year, in 2020, 13 years after the EU Paediatric Regulation came into force, our aim is to build on achievements and lessons learnt so far and to look at the future of paediatric drug development, with a specific focus on putting patients at the heart of innovation and regulatory science.

The opening panel session moderated by Fabio d’Atri (DG SANTE, European Commission) and involving key speakers representing EMA, FDA, National Authorities, Healthcare Professionals, and Industry, will be the opportunity to share updates in the field and discuss the future of the development of paediatric medicines: what to do more to be better at delivering innovations to children in needs? How the EC/EMA action plan has been progressed? To give them even more attention, in a separate panel session, the Young Patients, will tell us what the future of paediatric drug development means and looks like for them. Active engagement of conference participants is expected to discuss challenges and identify solutions on what could be done to optimise children’s access to new medicines:

1. What data sources and digital technologies could bring to paediatric development on top of the use of extrapolation? 2. How to best define children unmet medical needs? 3. How to optimise the PIP process and regulatory approval?

A fireside chat with PDCO moderated by Elin Haf Davies (Aparito) and involving the PDCO chair & vice-chair, will give the audience the opportunity not only to understand PDCO’s specific activities and priorities but also to further address clarifying questions to PDCO members.

The value of multi-stakeholder collaborations has been shown, since an opportunity to address important scientific gaps, but what is ongoing to foster paediatric development?

The conference will end with an important discussion to explore what drug development means for Paediatricians.

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Acknowledgement

EFGCP thanks its Partners & Members for their continued support

WORKSHOP PARTNER

EFGCP INSTITUTIONAL MEMBERS

EFGCP CORPORATE MEMBERS

Commissie

medische ethiek

Clinical Research Network

Faculty

Programme Committee

5

Commissie

medische ethiek

Clinical Research Network

Rime Allouche Kids FranceDimitrios Athanasiou EURORDIS, GreeceFabio d’Atri European Commission, DG Santé, BelgiumGesine Bejeuhr Bayer, GermanyChristina Bucci-Rechtweg Novartis, USASolange Corriol-Rohou AstraZeneca, FranceElin Haf Davies Aparito, United KingdomMartine Dehlinger-Kremer EFGCP & PRA Health Sciences, GermanyMarie-Yvonne Douste-Blazy Servier, FranceSabine Fürst-Recktenwald Roche, Switzerland FacultyFemida Gwadry-Sridhar Pulse Infoframe, CanadaNiklas Hedberg EunetHTA, SwedenRob Hemmings Concilium, United KingdomKarl-Heinz Huemer EMA PDCO, Austrian Medicines and Medical Devices Agency, AustriaIvett Jakab EPF’s Youth RepresentativeAngelika Joos MSD, BelgiumPirkko Lepola FinPedMed, FinlandDaniel Marin Young Advocate of Kids Barcelona, Sjd Children’s Hospital, SpainZinedine Metenani UCB, BelgiumBegonya Nafria Escalera Sjd Children’s Hospital Barcelona, SpainKoenraad Norga EMA, Antwerp University Hospital, Belgium & Chair of PDCO, EMA,The NetherlandsCécile Ollivier Aparito, United KingdomKatie Rizvi Youth Cancer Europe, HungaryAngeliki Siapkara MHRA, United KingdomSabine Scherer Vice-Chair, PDCO, EMA, The Netherlands & BfArM, GermanyAnja Schiel Norwegian Medicines Agency (NOMA), Norway & EMA SAWP ChairThomas Severin Novartis, SwitzerlandRobert Shaddy Pediatrician-in-Chief, Children’s Hospital Los Angeles, Los Angeles, USAAnna Sherriffs ScotCRNElke Stahl CTFG & Federal Institute For Drugs and Medical Devices (BfArM), GermanyPeter-Bram ‘t Hoen Radboud UMC, The NetherlandsRhian Thomas-Turner NHS, United KingdomAndrew Thompson EMA, The NetherlandsLynne Yao FDA, USAEvert Jan van Lente AOK-Bundesverband, GermanyNawel Van Lin Duchenne Data Foundation, The NetherlandsGilles Vassal Institut Gustave Roussy, FranceJoe Wilmshurst Lead Paediatric Neurology, South Africa

Dimitrios Athanasiou EURORDIS, GreeceSolange Corriol-Rohou AstraZeneca, FranceElin Haf Davies Aparito, United KingdomMartine Dehlinger-Kremer EFGCP & PRA Health Sciences, GermanySabine Fuerst-Recktenwald Roche, Switzerland FacultyAngelika Joos MSD, BelgiumBegonya Nafria Escalera Sjd Children’s Hospital Barcelona, SpainKoenraad Norga EMA, Antwerp University Hospital, Belgium & Chair of PDCO, EMA,The NetherlandsKatie Rizvi Youth Cancer Europe, HungaryThomas Severin Novartis, SwitzerlandAngeliki Siapkara MHRA, United KingdomRhian Thomas-Turner NHS, United Kingdom

Faculty

Programme Committee

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Monday 12 October 2020

12:00 Opening of conference

12:30 Welcome and introduction to the conference Martine Dehlinger-Kremer, EFGCP & PRA Health Sciences, Germany

Optimising the implementation of the Paediatric Regulation

The European Commission’s (EC) report released in 2018 identified a number of areas where short-term actions could address the identified shortcomings under the current legal framework. In order to follow up on the report’s conclusions the EC and the European Medicines Agency (EMA) have developed a detailed plan to boost the development of medicines for children in Europe, in consultation with all relevant stakeholders. The action items focused on 5 topic areas:

l Identifying paediatric medical needs l Strengthening cooperation of decision makers l Ensuring timely completion of paediatric investigation plans (PIPs) l Improving the handling of PIP applications l Increasing transparency around paediatric medicines

This session will review the progress of the activities so far, and discuss the next steps to follow through their completion.

12:45 Keynote: Where has progress been made and what remains to be discussed? Invited 13:00 Panel discussion Moderator: Fabio d’Atri, European Commission, DG Santé, Belgium Panellists will outline their viewpoints on their specific priorities as well as their contribution to address and complete the EC-EMA actions, where appropriate and discuss the future of paediatric drug development.

Koenraad Norga, EMA, Antwerp University Hospital, Belgium & Chair of PDCO, EMA, The Netherlands Elke Stahl, CTFG & Federal Institute For Drugs and Medical Devices (BfArM), Germany Lynne Yao, FDA, USA Gilles Vassal, Institut Gustave Roussy, France Solange Corriol-Rohou, AstraZeneca, France

13:50 Q&A session

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Voice of Young Patients including Q&A

14:10 What does the future of paediatric drug development mean and look like for Young patients? Moderators: Begonya Nafria Escalera, Sjd Children’s Hospital Barcelona, Spain Angeliki Siapkara, MHRA, United Kingdom

Daniel Marin, Young Advocate of Kids Barcelona, Sjd Children’s Hospital Barcelona, Spain Ivett Jakab, EPF’s Youth Representative Rime Allouche, Kids France Anna Sherriffs, ScotCRN

14:40 Introduction to the breakout sessions

14:50 Break

Break - Out Sessions

15:10 Breakout sessions to discuss topics in more detail with participants in a workshop fashion: Breakout session agenda: l Introduction led by the Moderator l Short 5 min impulse statements from selected key participants l Interactive discussion with participants to capture ideas and best practice

1. Extrapolation / RWD in drug development

Moderator: Sabine Fuerst-Recktenwald, Roche, Switzerland Faculty Co-Chairs and Rapporteurs: Cécile Ollivier, Aparito, United Kingdom Femida Gwadry-Sridhar, Pulse Infoframe, Canada Zinedine Metenani, UCB,Belgium Andrew Thompson, EMA, The Netherlands

A new ICH guideline is currently under development (ICH E11A) to recommend international standards for, and promote harmonisation of, the use of paediatric extrapolation to support the development and authorization of paediatric medicines. Meanwhile, the value and importance of health-related data derived in real life settings or Real-world data are increasing significantly. RWD can be medical health records, registries, biobanks, administrative data, health surveys, observational studies, health insurance data, data generated from mobile applications etc. The EMA Reflection Paper specifies that data sources other than clinical trials can be used in addressing uncertainties in paediatric development. Additionally, the Reflection Paper mentions particularly the use of registries in the context of extrapolation, also important in the context of Real-World Evidence.

This session is the opportunity to update on the ICH activities and will focus on how leveraging the EMA extrapolation framework as a regulatory tool to generate RWD of appropriate quality can be done and give examples on application of RWD in paediatrics.

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2. Identifying paediatric unmet needs Moderator: Marie-Yvonne Douste-Blazy, Servier, France Rapporteurs: Solange Corriol-Rohou, AstraZeneca, France Karl-Heinz Huemer, EMA PDCO, Austrian Medicines and Medical Devices Agency, Austria Gilles Vassal, Institut Gustave Roussy, France Anja Schiel, Norwegian Medicines Agency (NOMA) & EMA SAWP Chair

In December 2019, the European Federation of Pharmaceutical Industries and Associations (EFPIA) partnered with EFGCP to organise a multi-stakeholder workshop to explore how to best define and tackle unmet medical needs (UMN) in children, in order to provide a basis for strategic decision making on paediatric medicine development. The workshop objective was to find a broadly accepted approach to defining UMN among paediatric patients, in order to make better and safer medicines for children, and to make sure they are available and affordable. The outcome of the workshop was an outline of potential guiding principles for defining UMN in children, and a programme for testing the principles in selected therapeutic areas over the course of 2020.

This session is the opportunity to keep the momentum and gather additional insights that will feed into the generation and subsequent agreement on a more widely acceptable and agreed by end of 2020.

3. Regulatory overview: Optimising PIP processes and regulatory approval (PDCO & CHMP)

Moderator: Angeliki Siapkara, MHRA, United Kingdom Rapporteurs: Angelika Joos, MSD, Belgium Sabine Scherer, Vice-Chair, PDCO, EMA, The Netherlands & BfArM, Germany Rob Hemmings, Concilium, United Kingdom Gesine Bejeuhr, Bayer, Germany

This breakout session will facilitate interactive exchange with the session attendees to generate ideas for better integrating regulatory discussions around PIPs within the overall regulatory context. The session will focus on the regulatory discussions related to paediatrics within a broader life-cycle approach and how PDCO and CHMP are collaborating to improve regulatory outcomes for paediatric medicines.

The session’s goal is to identify what is needed to ensure that the PIP process is efficient and agile, for generating new authorisations for paediatric medicines.

Participants are encouraged to share practical case study experience that will shine a light on best practice examples and hurdles faced when implementing agreed PIP programs and submitting paediatric data for regulatory approval.

17:00 Break

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17:10 Fireside Chat Moderator: Elin Haf Davies, Aparito, United Kingdom

l Koenraad Norga, EMA, Antwerp University Hospital, Belgium & Chair of PDCO, EMA, The Netherlands (Chair) l Sabine Scherer, Vice-Chair, PDCO, EMA, The Netherlands & BfArM, Germany l Dimitrios Athanasiou, PDCO Patient Representative & EURORDIS, Greece

Questions to be submitted ahead of the conference

Session “What Drug Development means for Paediatricians”

Impact COVID-19 pandemic on paediatrics? Rapporteurs: Rhian Thomas-Turner, NHS, United Kingdom Angeliki Siapkara, MHRA, United Kingdom Thomas Severin, Novartis, Switzerland

This session aims to highlight, following the agreement of a PIP, what drug development means to clinicians and how to balance clinical care with delivering drug research. The session will outline the experience of hospital teams in delivering studies, including those smaller sites that do not have access to dedicated research facilities. Along these lines, the session will also ask whether current PIPs address the day to day needs of children and young people.

18:10 What happens at regional paediatric hospitals when trying to deliver paediatric drug research and top-level clinical care, by presenting the experience of a research operations manager, delivering research during the COVID 19 pandemic as a case study and lessons learned for the future Rhian Thomas-Turner, NHS, United Kingdom

18:30 What does drug development research mean to a paediatric clinician. This session will include the experiences of a paediatric specialist who is delivering research alongside a clinical service. The session will include an overview of how they balance the competing priorities of research and clinical commitments and why it is important to consider these parallel activities when designing future research. Robert Shaddy, Pediatrician-in-Chief, Children’s Hospital Los Angeles, USA

18:55 Break

19:00 Special Event with Dr Joe Wilmshurst, Lead Paediatric Neurology, South Africa Moderator: Elin Haf Davies, Aparito, United Kingdom

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Tuesday 13 October 2020

10:30 Debrief from Day one and introduction to Day 2 Martine Dehlinger-Kremer, EFGCP, PRA Health Sciences, Germany

Panel session on Patient Access

10:45 Moderators: Angeliki Siapkara, MHRA, United Kingdom Solange Corriol-Rohou, AstraZeneca, France

Despite the clear benefits of the EU Paediatric Regulation towards increased licensed paediatric treatments, even when a new medicinal product is approved for use in children, this does not necessarily result in children access to it. Optimising availability and access to medicines that would greatly benefit children, would improve consistently the tangible effect of the Paediatric Regulation.

This is an important issue which requires consideration from all stakeholders in order to make appropriately studied and authorised medicines available to children. Actual availability and accessibility depend on further arrangements for placing on the market, reimbursement and sufficient pricing, which have to be agreed in each Member State.

For this session we will bring together HTAs, payers, patients and industry representatives for an open discussion of the actions needed.

Panellists: l HTA representative - Niklas Hedberg, EunetHTA, Sweden l Payer representative - Evert Jan van Lente, AOK-Bundesverband, Germany l Patient representative - Katie Rizvi, Youth Cancer Europe, Romania l Industry representative - Invited

Enpr-EMA: Clinical Trial preparedness & Internationalcollaboration Work Group

12:15 Moderators: Angeliki Siapkara, MHRA, United Kingdom Martine Dehlinger-Kremer, EFGCP, PRA Health Sciences, Germany

Speaker: Pirkko Lepola, FinPedMed, Finland

12:35 Debrief & Conclusions from the breakout sessions

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The Go- FAIR Initiative / Use Data

13:05 Moderators: Elin Haf Davies, Aparito, United Kingdom Dimitrios Athanasiou, PDCO Patient Representative & EURORDIS, Greece

Panellists: l Nawel Van Lin, Duchenne Data Foundation, The Netherlands l Peter-Bram ‘t Hoen, Radboud UMC, The Netherlands

The FAIR (Fostering Age Inclusive Research) Initiative – Inclusion of Adolescent in Adult trials

13:35 Moderators: Solange Corriol-Rohou, AstraZeneca, France Katie Rizvi, Youth Cancer Europe, Romania Panellists: l Patient representative - Invited l Christina Bucci-Rechtweg, Novartis, USA

14:20 Conclusions & Farewell Martine Dehlinger-Kremer, EFGCP & PRA Health Sciences, Germany and all

EFGCP & DIA Better Medicines for Children Conference 2020“The future of paediatric medicines:

putting patients in the heart of innovation and regulatory science”

Conference LanguageThe language of the conference will be English.

Registration & Informationevents@efgcp.eu

visit:www.efgcp.eu

Virtual Conference