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ITCC Hopp: International Data Integration Platform to prioritize drug development and access for children with cancer

Background

Cancer is very rare in children and adolescents. The probability of a new born child suffering from a malignant disease within the first 18 years of life is 0.3%. Nevertheless, cancer is the most common fatal disease and (after accidents) the second most common cause of death among children and adolescents in industrialized countries.

In Germany, there are approximately 2,250 new cases of the disease each year in children under 18. In children and adolescents under 18, leukaemia and lymphoma account for almost 50% of all new cases, with acute forms predominating. The overall prognosis is significantly better than in adults. However, there is still a group of about 20% of all patients who, unfortunately, ultimately die from their disease despite intensive treatment.

Children are not small adults. Cancers that occur at this young age therefore differ in many ways from cancers in adults. This concerns not only the type of disease and the frequency of its occurrence, but also the type of treatment and the prospects for recovery. Ideally, more cancer drugs would be developed directly for use in children - but since the market size is so small, this option is unprofitable for the pharmaceutical industry. An EU-wide change in the law in 2007, according to which all new drugs coming onto the market for adults must also be tested for use in children, tried to tackle this problem. Given the disproportionate number of drugs for adults, these must be prioritized for clinical use in children based on existing data. 

To date, however, these data have not been evaluated centrally and systematically, but usually regionally or at most nationally. Unfortunately, given the rarity of each type of cancer in children, this only contributes insignificantly to the knowledge gained. To reach a representative number of study participants, the ITCC Hopp paediatric cancer data portal initiative is an international partnership involving clinical programs spanning seven nations aiming to address this problem by aggregating and harmonizing genomic data from over 6000 paediatric cancer, holding the potential to revolutionize diagnostic approaches and therapeutic strategies, impacting young patients worldwide.

In order to consider, the different data protection regulations in different countries, the raw data is not transferred, but the examination algorithms are coordinated and then applied locally to the respective data sets. Such a relatively new and technically challenging approach requires a large number of infrastructural, technical, procedural, legal and ethical requirements, which are to be created in this project. However, these will also be used as quickly as possible to answer very specific academic and industrial inquiries regarding the suitability of new cancer drugs for childhood cancer.

Ethics

Within the project, there are several ethical challenges we try to tackle: we evaluate how the envisioned data platform will affect patients` informal rights, searching for an ideal form of consent, that will keep the patients` right to self-determination. While there are already many guidelines on informed consent for children in general, it is our main task to develop or update existing guidelines for informed consent for children regarding genomic sequencing in personalized oncology programs, so we can provide all participating project partners with a general guideline. 

We also give ethical recommendations and establish rules for Data Use and Access Committees and Governance on how to access applications for the release and use of patient data. Several stakeholders, including patients and their proxies, clinicians as well as researchers from academia and industry are going to demand access to the patient`s data. Ethical challenges here concern e.g. the safeguarding of patients’ informational rights and trust, fair recognition of contributors, open science, good scientific practice and non-discrimination.

Using clinical and genomic data from patients for research use might lead to so-called incidental findings. We develop an ethical guidance for a policy for responsible handling of incidental findings. Incidental findings involve several ethical challenges such as assessment of potential clinical benefit for patients and return-of-results-process

Program: Individualized Therapy FOr Relapsed Malignancies in Childhood (INFORM)

Hopp Children’s Cancer Center Heidelberg (KiTZ)

German Cancer Research Center (DKFZ)

 

Program: Molecular and Immunological Characterization of high risk CHildhood cancer at diagnOsis (MICCHADO)

Institut Curie, Paris, France

Institute Gustave Roussy, Villejuif, France

 

The individualized THERapies for children with cancer (iTHER)

Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands

 

Program: The Danish Childhood Cancer Initiative

Rigshospitalet, Copenhagen, Denmark

 

Program: PROFYLE (PRecision Oncology For Young peopLE) (Canada):

The Hospital for Sick Children, Toronto, 

The Research Institute of the McGill University Health Centre, Montreal, Quebec, Canada 

BC Cancer, part of the PROVINCIAL HEALTH SERVICES AUTHORITY, Vancouver

Centre Hospitalier Universitaire Sainte-Justine, Montreal, Quebec, Canada

 

Program: The ZERO Childhood Cancer Programm (Australia):

University of New South Wales, Kensington, Australia

 

Program: Stratified Medicine Paediatrics (SMPEDS) (UK)

The Institute of Cancer Research, Royal Caner Hospital, London

Dietmar Hopp Foundation