Rare Cancers Conference 2012

Exploring ways to improve clinical research on rare cancers

Organised by the European Society for Medical Oncology (ESMO) and Rare Cancers Europe, the Rare Cancers Conference, held on 10 February 2012 in Brussels, provided a multi-stakeholder platform for rare cancer and rare disease experts from across Europe to exchange views and share insights into what can be done to improve the methodology of clinical research on rare cancers.

The first two conference sessions offered an overview of rare cancers and associated challenges for clinical research and drug development and also presented a variety of (potential) solutions as well as best practice examples. Where traditional frequentist clinical research approaches are not possible, due to the small numbers of patients, it is particularly challenging to make sure that rare cancer patients are not being left without appropriate clinical research and therapeutic progress.

The third session of the conference therefore also highlighted the need for reaching a broad multi-stakeholder consensus on a set of recommendations on improving the methodology of clinical research on rare cancers. These recommendations will be the product of an ongoing multidisciplinary and multi-stakeholder online consensus discussion, promoted by Rare Cancers Europe. They will focus on best methods, including innovative ones, for clinical research on rare cancers, and rare subgroups of frequent cancers, with the goal of encouraging:

  • clinical researchers to exploit innovative solutions for the design and analysis of clinical studies;
  • clinicians to exploit innovative solutions for the combination of all available knowledge;
  • regulators to accept evidence built through these solutions;
  • clinicians’ and patients’ communities to exploit all forms of collaboration to put together as large series as possible for prospective and retrospective clinical and translational research;
  • methodologists to advance research into new methodological solutions better fitting the needs of studies on small series

All interested stakeholder groups are encouraged to actively participate in this open discussion, the result of which will be a consensus paper to be publicly presented in autumn 2012. This paper could then be used for related advocacy efforts. All parties interested in joining this discussion are invited to contact Rare Cancers Europe.

A final panel discussion rounded off the Rare Cancers Conference by giving all stakeholders the opportunity to share their respective views on what each stakeholder group can do to help drive clinical research on rare cancers. An article on the conference, published in the May/June 2012 issue of Cancer World magazine, can be accessed here. The full programme, links for downloading the conference presentations and links to available audio webcasts are attached below.

Rare Cancers Conference Programme, 10 February 2012

Welcome and Introduction

  • P. G. Casali, European Society for Medical Oncology (ESMO), IT
  • Y. Le Cam, European Organisation for Rare Diseases (EURORDIS), FR, on behalf of EURORDIS and European Cancer Patient Coalition (ECPC)
  • Audio webcast

Rare Cancers – An Overview

Co-chairs:

  • M. Pierotti, Organisation of European Cancer Institutes (OECI), IT
  • K. Oliver, International Brain Tumour Alliance (IBTA) and Cancer 52, UK
  • Audio webcast

The epidemiology of rare cancers in Europe

A list of rare cancers

Significant benefit and clinical added value of orphan drugs

Rare cancers and drug development in Europe

Remaining obstacles towards pharmaceutical development of treatments for rare cancers in Europe

Clinical practice guidelines to set standards at the European level

Best practice: An attempt of a rare cancer community to set European multidisciplinary standards

Best practice: Le Cercle des Tumeurs Rares and INCa, France

Discussion

Clinical Research on Rare Cancers – An Introduction

Co-chairs:

  • S. van Belle, University Hospital Ghent, BE
  • J. Geissler, CML Advocates Network, DE

The changing environment of cancer science

Regulatory and legal EU constraints to clinical studies on rare cancers

New collaboration models in rare cancers

Methodological challenges

Best practice: The International Rare Cancers Initiative

Best practice: International collaboration on sarcomas

Best practice: International collaboration on a very rare cancer (adrenal carcinoma)

Best practice: Paediatric oncology

Best practice: Patient involvement in clinical studies

Consensus Recommendations on Improving the Methodology of Clinical Research on Rare Cancers

Co-chairs:

  • M. Piccart, European Society for Medical Oncology (ESMO), BE
  • K. Redmond, Cancer World Magazine, European School of Oncology (ESO), CH

Rare Cancers Europe

Designs of clinical studies in rare cancers

Bayesian approaches to analysing studies and summarizing evidence

Bringing evidence within clinical decision-making

Organizational and regulatory aspects of clinical studies

What Stakeholders Can Do to Drive Clinical Research on Rare Cancers (Panel Discussion)

Co-chairs:

  • A. Cervantes, European Society for Medical Oncology (ESMO), ES
  • W. Yared, Association of European Cancer Leagues (ECL), BE

Medical practice / Clinical research

  • P. G. Casali, European Society for Medical Oncology (ESMO), IT
  • J.-Y. Blay, European Organisation for Research and Treatment of Cancer (EORTC), FR
  • A. Margulies, European Oncology Nursing Society (EONS), CH Download presentation
  • Audio webcast

Statistics / Epidemiology

  • J. Bogaerts, European Organisation for Research and Treatment of Cancer (EORTC), BE
  • P. Bruzzi, National Cancer Research Institute, IT
  • Audio webcast

Regulatory Affairs / Health administration

Pharmaceutical industry

  • S. Comis, Novartis Oncology, IT
  • E. Colajori, Pfizer Oncology, IT
  • J. Adamczewski, Sanofi, FR
  • Audio webcast

Biology / Pathology

  • A. P. Dei Tos, European Society of Pathology (ESP), IT
  • Audio webcast

Patient advocacy

Outlook and Concluding Remarks

  • P. G. Casali, European Society for Medical Oncology (ESMO), IT;
  • F. De Lorenzo, ECPC, IT, on behalf of ECPC and EURORDIS
  • Audio webcast
200. Call to Action