WP 4 – EPIDEMIOLOGY GEMMA Gatta General Assembly, 16th October, 2017, Milan   WP 4 – EPIDEMIOLOGY GEMMA Gatta

Objectives agreement on the list of rare cancers and endorsement of MSs (task 1); recommendations to improve the data quality of rare cancers in population based registries (task 2); recommendations for rare cancers indicators by Europe and country, minumum data set for registries (task 3); to propose a model to evaluate the impact of ERNs using population-based CRs (task 4).

List of rare cancers (task 1) published by the two EU projects

List of rare cancers (task 1) also available at the RARECAREnet website search tool

List of rare cancers (task 1) for solid tumours ERN (EURACAN) list used by MS to identify health care providers to include in the ERNs for rare cancers Which cancers are rare? Updated list : first deliverable at 18° month

Present the list, its rationale and support use in different context List of rare cancers (task 1) Meeting on rare cancer list in January, 2018 Points: Present the list, its rationale and support use in different context Discuss major issues with the list from different stakeholder prospective Find solution to have a list of rare cancers useful for all the stakeholders

Stakeholders involved JARC MSs Patients associations EMA Orphanet List of rare cancers (task 1) Meeting on rare cancer list in January, 2018 Stakeholders involved JARC MSs Patients associations EMA Orphanet ENCR (network of European registries) Scientific societies ERNs

Cancer registration (task 2) leader FFIS, Spain

Cancer registration (task 2) Teleconferences, mail exchange and yesterday meeting Definition of the working group Identification of major challenges for rare cancer registration Circulation of first documents/proposals First meeting February 2018

Cancer registration (task 2) Dissemination and meetings Annual meeting of cancer registries (GRELL, ENCR/JRC, …) To present the study and discuss recommendations Final publication of the study (proposal) as special issue of a scientific journal.

Methods for descriptive and outcome indicators (task 3), leaders INT&EASP (Italy, Spain) Descriptive indicators: Incidence, survival and prevalence Outcome indicators: ad hoc collection through high resolution studies The major aim is to provide surveillance in Europe for the ERN and Health Care System

Methods for descriptive and outcome indicators (task 3) deliverable (M24) Recommendations for standardised estimation of rare cancer indicators by Europe and country

Methods for descriptive epidemiology in rare cancers (task 3) Definition of the group of interested partners √ Each participant indicated relevant papers on the topics in discussion √ In preparation a proposal concept document based on our experience and on the literature Prepare and circulate a first draft of the final document Prepare the final draft and submit the Task document

Methods descriptive epidemiology rare cancers (task 3) Topics to be addressed (already circulated in a document, milestone 4.5) Providing rare cancers epidemiological estimators for incidence, survival, prevalence, and mortality from registry data. Relative advantages and disadvantages of point estimates vs model based estimates. Relative advantages and disadvantages of frequentist vs Bayesian approach. Performance indicators: precision, accuracy, robustness. Incidence, survival, and cancer type distribution homogeneity across regions. At which level of rarity normal methodology breaks down and special methods are required Estimates in populations not covered by cancer registration

Definition of outcome indicators to assess the ERN impact at the population level (task 3) ERN 3 groups of rare cancers: solid paediatric, haematologic, evaluation using cancer registry data on: Access to ERN Quality of care Survival differences between patients accessing or not the ERN Definition of a minimum data set of clinical variables to be collected by registries, in connection with task 2

Pilot study (task 3) neuroblastoma and nephroblastoma Staging childhood cancer in population based cancer registries, endorsed by the UICC

Cancer registration (task 3) pilot study on neuroblastoma and nephroblastoma Staging childhood cancer in population based registries (in collaboration with Prof. Kathy Pritchard-Jones from SIOPE) First draft of the protocol circulated 13 European countries accepted to participate First teleconference 25°Oct 2017 By the end of April the preliminary results Final meeting presenting the results before the end of June

Model to evaluate impact of ERN (task 4) leader UVEG, Spain Linkage between clinical and population based databases (neuroblastoma) Several steps already done SIOPEN approuval Questionnaires to registries both population and clinical based Still collecting and sollecitating for the completated questionnaires

To conclude We are aware that the second year is strategic for conclude with the recommendation at 30° month 2nd year milestones: Several small meetings to discuss data quality 2nd year deliverables: List of rare cancers (M18) Document on methodology for rare cancers burden estimation (M24)