Platform Publications:

• How the EU Pharmaceutical Strategy can help children and adolescents with cancer. Interview with Prof. Gilles Vassal and Prof. Pam Kearns, The Parliament Magazine, 1 October 2020
• PSF for medicinal product development of epigenetic modifiers for children. Paerson et al., European Journal of Cancer November 2020; 139:135-148
• PSF for Medicinal Product Development for Acute Myeloid Leukaemia in Children and Adolescents, Pearson et al., European Journal of Cancer, European Journal of Cancer September  2020; 136:116-129
• Accelerating the Global Development of Pediatric Cancer Drugs: A Call to Coordinate the Submissions of Pediatric Investigation Plans and Pediatric Study Plans to EMA and US FDA.  Journal of Clinical Oncology. September 2020.
• The RACE to accelerate drug development for children with cancer, The Lancet Child & Adolescent Health,
  Pearson et al., August 2020
• PSF medicinal product development of checkpoint inhibitors for use in combination therapy in paediatric patients,Paerson et al., European Journal of Cancer, January 2020.
• ‘ACCELERATE and European Medicine Agency Paediatric Strategy Forum for Medicinal Product Development for Mature B-cell Malignancies in Children’, Paerson et al., European Journal of Cancer, March 2020;110:74-85
• ‘ACCELERATE and European Medicines Agency Paediatric Strategy Forum for Medicinal Product Development of Checkpoint Inhibitors for Use in Combination Therapy in Paediatric Patients’, Paerson et al., European Journal of Cancer January 2020; 110:74-85
• 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:

• PRESS RELEASE: New U.S. Law to Improve International Research for Childhood Cancer
• NEWS RELEASE: Worldwide Pharmaceutical industry associations call for a better deal for young people with cancer in clinical trials, 12 August 2020
• PRESS RELEASE: Teenagers with #cancer set to benefit from international initiative aimed at lowering the age of participation to potentially life-saving treatments, 18 February 2020.
• PRESS RELEASE: Groundbreaking Childhood Cancer Collaborative Comes to the U.S. for the First Time, 29 January 2020
• ITCC P4 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:

• Inclusion of Adolescents in Adult Clinical Trials: Report of the Institute for Advanced Clinical Trials for Children’s Pediatric Innovation Research Forum. Therapeutic innovation & regulatory science. Noel, G.J. et al., 2021.
• Adolescents and young adults (AYA) with cancer: a position paper from the AYA Working Group of the European Society for Medical Oncology (ESMO) and the European Society for Paediatric Oncology (SIOPE). Ferrari, A. et al., 2021. ESMO OPEN
• Accelerating the Global Development of Pediatric Cancer Drugs: A Call to Coordinate the Submissions of Pediatric Investigation Plans and Pediatric Study Plans to EMA and US FDA.  Reaman, G., Karres D. et al., JCO, September 2020
• Pediatric cancer research: Surviving COVID‐19. Auletta J et al., Pediatric Blood & Cancer, June 2020. 
• Can a multi‐stakeholder prioritisation structure support regulatory decision making? A review of paediatric oncology strategy forums reflecting on challenges and opportunities of this concept. Karres D. et al., Clinical Pharmacology & Therapeutics, June 2020
• Bio-pharmaceutical industry letter of support to Fostering Age-Inclusive Research Trials Initiative
• 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:

• FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs: Amendments to Sec. 505B of the FD&C Act, December 2019 (DRAFT GUIDANCE in PDF).
• 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