IntroductionGilles Vassal

an international multistakeholder organisationworking to solve the central challenges

in the development of innovative anticancer medicines for children and adolescents

OUR NAME IS OUR MISSION

United States 55United Kingdom 33Belgium 18Netherlands 17Switzerland 15France 12Germany 12Italy 7Japan 7Sweden 4Spain 4Canada 3Australia 3Greece 2Denmark 2Austria 1Lithuania 1New Zealand 1TOT 197

Industry 70

Academia 67

Patient Advocates 44

Regulators 10

European Commission 1

Staff 5

TOT 197

Geographical origin Affiliation

8th ACCELERATE Conference

Two anticancer medicines approved for children

• US August 15, 2019 Entrectinib – ROZLYTEK™a pan-TRK, ROS1 and ALK inhibitorsolid tumors with NTRK fusion (12 years and older)

• EU September 19, 2019 Larotrectinib – VITRAKVI™a pan-TRK inhibitorsolid tumors with NTRK fusion

ACCELERATE-ing Pediatric Oncology Drug Discovery and Development

ATLANTAApril, 2, 201910:30am

2017DAY -194

December 2019

FDARA Guidance – last paragraphAdditionally, FDA encourages participation in international multi-stakeholder meetings including the Pediatric Strategy Forums organized by the ACCELERATE Platform which bring sponsors, investigators, patient advocates, and regulators together to discuss development strategies for specific pediatric cancers in the context of the number of investigational drugs available for assessment and the highly variable unmet medical needs of distinct pediatric populations with specific childhood cancers. We recommend stakeholders, including sponsors, investigators, and patient advocates consider coordinating early multi-stakeholder input to inform decision-making related to the initial pediatric clinical evaluation of appropriate

1. To set-up the 5th Pediatric Strategy Forum in the US and to implementthe break out session recommendations

2. To develop the agenda of the FAIR Working Group and create 2 new working groups (see next slide)

3. To define a plan to implement break out session recommendations on International Collaboration

4. To explore the field of HTA evaluation across specialties and the ongoing initiatives to make a proposal at the next ACCELERATE meeting

5. To define a communication strategy for ACCELERATE

Workplan 2019 (agreed by the Steering Committee on April 29, 2019)

ongoing

Working Groups

FAIR Working Group – Nathalie Gaspar and Chris CoplandObjectives : Develop the survey, implement the tool kit, new webpage, monitor implementation

LTFU Working Group – Mark Kieran and Daniele HortonObjective: Create an international data repository

Fit For Filing Working Group – Elly Barry and Pam KearnsObjective : develop best principles on how the design and deliver a trial with a dataset that can be included in a package for filing

International Collaboration – Nicole Scobie and Greg Reaman

NEW

NEW

NEW

79 participants

Break Out SessionsMeeting Group Chairs

Group 1A Andy Pearson + Scott Diede

Group 1B Sam Blackman + Andy Kolb

Group 2A Lynley Marshall + Darshan Wariabharaj

Group 2B Dominik Karres + Max Williamson

Group 3A Hubert Caron + Nicole Scobie

Group 3B Leona Knox + Stefan Pfister

Group 4A Elly Barry + Pam Kearns

Group 4B Peter Adamson + Davy Chiodin4. Implementing the Race for Children Act

3. Preclinical testing in order to benefit children

2. Beyond randomized controlled trials: ‘non-conventional’ data sources for regulatory decision-making

1. Challenges in prioritisation – The way forward

A calatyst and a sentinel*

*ACCELERATE is a sentinel whose goal is to warrant that implementation of new regulatory measures benefit children

OUR NAME IS OUR MISSION

The value of working together

Link: https://live.voxvote.com PIN: 23124