WP07 - Innovation and access to innovation

Leader: Marc Van Den Bulke


WP 7: Innovation and access to innovation

The goal of WP7 is to establish optimal ways to bring the best treatment and care to patients by advancing translational and clinical research on rare cancers trough European Reference Networks.

The objectives are:

  1. to stimulate translational research on RCs by exploiting networking, namely through the new ERNs, as a mean to promote banking
  2. to promote exploitation of “big data”, namely those generated through the new ERNs, as a mean to advance our knowledge on rare cancers to provide recommendations on how to optimize long-term surveillance of rare cancer patients in an effort to address the many survivorship’s issues
  3. to make proposals on how to exploit available regulations across the EU, and/or how to improve them, on collaborative prospective interventional clinical research, especially academic clinical research joining national collaborative groups (inter-group research, etc.).

Four main tasks:

Task 7.1 RC Translational Research landscape

This Task will be aimed at standardizing clinical data and patient-centred clinical outcomes as well as defining a robust quality assurance / quality control (QA/QC) framework for SPECTARare with regard to biological samples, data collection and molecular screening in order to address the specific research needs of the 12 different families of rare cancers included in the present JA.

Task 7.2. Big data

Recommendations will be provided with regard to the requirements for long-term surveillance of rare cancer patients, given the peculiarities of their natural history and treatments, so that these criteria may be added to those which are relevant to accreditation of ERNs and reference centres on rare cancers. Thus, this task will address the many regulatory and legal issues posed by clinical big data. It will address the issues concerning the harmonization of these data, especially in terms of their semantics. The goal will be to explore the possibility to set up a clinical epidemiology framework program for outcome research in rare cancers including the use of patient-reported outcomes, including quality of life end-points. This Task will contribute to solve the theoretical and methodological issues posed by the use of these data to advance medical.

Task 7.3. Collaborative clinical trials

This Task will try to make an inventory of regulatory constraints for collaborative international clinical trials in the EU, particularly inter-group clinical trials. The Task may also provide proposals to improve national and European regulations and/or their interpretation. Recommendations will be made about the optimal ways to connect the new ERNs to collaborative clinical research, whether investigator-driven or industry-sponsored including quality criteria for reference centers and ERNs based on clinical research performances.

Task 7.4. Precision medicine in rare cancer care

The aim is to provide recommendations on integrating translational research innovations into rare cancer care, based on conclusions and recommendations developed in the Joint Action and benchmarking with worldwide initiatives in the field.

The output will be a Roadmap on the integration of translational research into routine rare cancer care within the evolving scenario of ERNs.