Current Challenges in Paediatric Oncology in Europe Ruth R th Ladenstein L d t i SIOPE President SIOPE Head Office in Partnership with ECCO: Brussels Brussels, Belgium St. Anna Children‘s Hospital and Research Institute (Vienna, Austria)

Are Children Different ?

Heterogeneous in many aspects! „ Children are not miniature adults” adults ...from a clinical trial operational point of view either

20% of the European Population 100 Millions < 18 Years !

Improving Survival Rates in Paediatric Cancer

>90% 2015 !? 80% today 60 different diseases from newborns to teenagers (even more when biomarkers are considered)

• No private practice  250 EU public specialized centres • Networked since 1968 for clinical research – 50% of patients treated within trials (phase I to III) – 30% of patients treated according to standard within prospective studies – Less than 5% of p pharma-sponsored p trials

• Many high-level research teams dedicated to paediatric tumour biology A unique situation for an orphan disease !

Children are still „Pharmaceutical Orphans “ The need is high BUT ….. > 50% 0% off DRUGS GS are used  Off-label  Unlicenced  80% Paediatric Oncology

 Need to STOP daily experiments

Conroy, Arch Dis Child 1999 Turner, Lancet 1996 Conroy BMJ 2000 Conroy,

 Support needed from Policy Makers and Pharma

Current Scope of Paediatric Oncology in Europe I-BFM group SIOP Brain Tumour Group SIOP Wilms Tumor

EuroEwing Hodgkin’s lymphoma group

Rare Cancers Group

EIC-NHL

EORTC Childhood Leukemia group

Myelodysplasia Group

Histiocyte society y

• 15 EU groups on solid tumors, hematological diseases, early drug development including joint programs with adult oncology •A A track t k record d off high ranking publications Parents O Organisations i ti

Current challenges and limitations  Struggling to run investigator driven clinical trials within the  2004 clinical trial directive – S Safe f environment i t if enrolled ll d iin pediatric di t i oncology l Ph Phase III studies t di – IMPDs – Risk adjustment for pediatric trials

 Extremely poor access to new drugs despite the 2007  Paediatric Medicine Regulation Paediatric Medicine Regulation  Off Label use for children in standard treatments unsolved  80% in Paediatric Oncology

 Unequal access to standard therapies  Lack of sustained &  sufficient funding 

A Network of Excellence Structuring Clinical Research in Paediatric Structuring Clinical Research in Paediatric  and Adolescent Oncology in Europe HEALTH.2010.2.2.1--3 HEALTH.2010.2.2.1

33 Partners 11 E European C Countriesti 18 WP – 80 Milestones – 82 Deliverables

The ENCCA Goals  

 

To improve both cure and quality of cure of children and  adolescents suffering of cancer To facilitate access to   innovative therapies and tailored medicines  and standard care across Europe  To develop biology‐guided therapies To propose a Virtual European Institute for Cancer  Research in Children and Adolescent (sustainability)

ENCCA Actions 2011- 2014  

Implement a European Strategy for pediatric and  Implement a European Strategy for pediatric and adolescent oncology research Provide integrated tools and platforms   (clinical trials, tumor banking, methodology, biology)

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Run a limited number of clinical and translational studies as  examples Facilitate sharing and partnerships in the chain of all  stakeholders  (academia, parents and patients organizations, pharmaceutical  companies, regulatory bodies, governmental bodies, charities)

 

Train and educate Train and educate Provide common ethical definitions and solutions    

The Needs of Paediatric Oncology In Europe  Revise the Clinical Trial Directive to facilitate  multinational investigator‐driven clinical trials g  Make the Paediatric Medicine Regulation a success  Consider the Paediatric Malignancies as a Health  Consider the Paediatric Malignancies as a Health issue  Secure funding & Sustainability S f di & S t i bilit

Clinical Trials with Children are Trials FOR Children Securing g a Brighter g Future!

20% more survive in controlled trial environments!

Thank a you For Considering Our Children with Cancer in Future Policy Decisions !

Back up slides

STRATEGIC Management General Assembly (GA) ((All NoE members )

Ethics Advisory Committee (EAC) Parents / Patients Advocacy Committee (PAC)

Intellectual Property Rights Committee (IPRC)

Executive Committee (ExeCom) ((NoEM,, PM,, DM,, 3 ACs,, 3 R_AdCom, 2 R-ECRC)

Industrial Club of Interest (ICI)

European Clinical Research Council (ECRC)

INTEGRATIVE Management

Network of Exellence Manager (N EM) (NoEM)

Project Manager (PM)

OPERATION NAL Manage ement

Ma anag gem men nt

Scientific Advisory Committee (SAC)

Integrating Activities Coordinator (IAC)

EC

Dissemination Manager (DM)

IA Activities (WP1.1 to WP1.6)

Joint Research Activities Coordinator (JRAC)

JRA Activities (WP2.1 to WP2.6)

S Spread d off Excellence Activities Coordinator (SEAC)

SEA Activities (WP3.1 to WP3.5)

Adavanced management of costs, ressources, planning, knowledge, communication, quality indicators

Towards a European Virtual Network in Paediatric Oncology Clinical Trials

ECRC E ropean Clinical Research Co European Council ncil •

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European clinical groups will be represented by a Council – Chairs of each tumour group – Chairs of the national paediatric oncology societies European level of coordination for paediatric di t i and d adolescent d l t cancer clinical research activities Capacity to address the many common issues that cannot be solved by each group working in isolation.

Neuroblastoma

Acute Myeloid Leukemia Infant ALL

Soft Tissue Sarcomas

European E Clinical Research Council (ECRC)

Renal Tumours

Ewing Sarcoma Family Tumours

Osteosarcoma

Embryonal Brain Tumours

Relapsed ALL

Acute Lymphoblastic Leukemia

Brain Glioma