Platform Publications:

• FAIR (Fostering Age Inclusive Research) Trials is aiming to lower the age limits for clinical trials, SciTech Europa, 26 February 2019
• “4 ways cancer is changing how we test medicines“, POLITICO, 18 October 2018
• “Tragedy of children dying from cancer because drug companies won’t include them in trials”, Daily Mail, 27 June 2018
• “Horizon: Teenagers vs Cancer: a User’s Guide, review – this film strove to be cheerful but was hampered by lack of clinical trials”, Telegraph, 26 June 2018
• “FDA Calls for Including Adolescents in Adult Oncology Trials”, RAPS, 1 June 2018
• ‘ACCELERATE-ing access to drugs for adolescents with cancer‘, Medicine Matters, 27 April 2018
• ‘Creating a unique, multi-stakeholder Paediatric Oncology Platform to improve drug development for children and adolescents with cancer’, Vassal et al., European Journal of Cancer, January 2015, Volume 51 , Issue 2 , 218 – 224
• ‘Implementation of mechanism of action biology-driven early drug development for children with cancer’, July 2016, Pearson et al., European Journal of Cancer, Volume 62, 124-131

Press Releases:

• ITCC P4 FIRST PRESS RELEASE: ‘ITCC-P4: a new platform to accelerate drug development for children and adolescents dying of cancer‘, 12 June 2017
• ACCELERATE Press Release ‘State-of-the-art Cancer Medicines Should Not Forget Children‘, 3 March 2017

Relevant Publications:

• International consortium to accelerate safer, more effective treatment for children’s brain tumours, eCancer, 7 Mar 2017
• The Importance of Pediatric Drug Development: A Conversation with Pr. Gilles Vassal, Dr. Gregory Reaman, and Mr. Raymond Rodriguez-Torres, Friends of Cancer Research, February 2017
• New drugs for childhood cancers: could biotechs end the drought?, Fessl S., CancerWorld, September 2016
• Urgent need to speed up life-saving innovation – Proposal by the Stakeholders, January 2016
• ‘Will the revised class waiver list make it?’, Vassal G. et al., The Lancet Oncology , Volume 16 , Issue 9 , e425 – e426, September 2015
• The SIOPE European Cancer Plan for Children and Adolescents, September 2015
• ‘Hope for More Paediatric Medicines in Europe’, SIOPE news, July 2015
• ‘New drugs for children and adolescents with cancer: the need for novel development pathways’, Vassal et al., The Lancet Oncology, March 2013
• ‘Is the European pediatric medicine regulation working for children and adolescents with cancer?’, Vassal et al., Clinical Cancer Research, March 2013

Relevant Regulations:

• EU Regulation on medicinal products for paediatric use [EU Regulation on medicinal products for paediatric use [Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006 on medicinal products for paediatric use and amending Regulation (EEC) No 1768/92, Directive 2001/20/EC, Directive 2001/83/EC and Regulation (EC) No 726/2004e]

Relevant Links:

• Class waiver list on EMA
• Paediatric investigation plans decision on EMA

Abbreviations:

• ALK: anaplastic lymphoma kinase
• BPCA: Biologics Price Competition & Innovation
• CDDF: Cancer Drug Development Forum
• EMA: European Medicines Agency
• ENCCA: European Network for Cancer Research in Children and Adolescents
• FDA: Food and Drug Agency
• IMI: Innovative Medicine Initiative
• ITCC: Innovative Therapies for Children with Cancer
• LTFU: long-term follow-up
• MA: marketing authorisation
• MoA: Mechanism of Action
• PDCO: Paediatric Committee [of the EMA]
• PIP: Paediatric Investigation Plan
• PK: pharmacokinetic
• PPoC: paediatric proof of concept
• PREA: Pediatric Research Equity Act
• PRINTO: Paediatric Rheumatology International Trials Organisation
• SIOPE: European Society for Paediatric Oncology
• US[A]: United States [of America]
• TYA: teenagers and young adults
• WG: Working Group
• WR: written requests