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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