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 frequent 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)
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
The epidemiology of rare cancers in Europe
A. Trama, Fondazione IRCCS Istituto Nazionale dei Tumori, IT
A list of rare cancers
A. P. Dei Tos, EuroBoNeT/EUROSARC, IT
Significant benefit and clinical added value of orphan drugs
Y. Le Cam, EURORDIS, FR
Rare cancers and drug development in Europe
B. Jonsson, Läkemedelsverket (Swedish Medical Products Agency, MPA), SE
Remaining obstacles towards pharmaceutical development of treatments for rare cancers in Europe
A. Fehervary, Novartis Oncology Region Europe, IT
Clinical practice guidelines to set standards at the European level
A. Cervantes, ESMO, ES
Best practice: An attempt of a rare cancer community to set European multidisciplinary standards
L. Licitra, Fondazione IRCCS Istituto Nazionale dei Tumori, IT
Best practice: Le Cercle des Tumeurs Rares and INCa, France
J.-Y. Blay, European Organisation for Research and Treatment of Cancer (EORTC), FR
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
J. E. Celis, European CanCer Organisation (ECCO)
Regulatory and legal EU constraints to clinical studies on rare cancers
A. Negrouk, European Organisation for Research and Treatment of Cancer (EORTC), BE
New collaboration models in rare cancers
D. Lacombe, European Organisation for Research and Treatment of Cancer (EORTC), BE
Methodological challenges
J. Bogaerts, European Organisation for Research and Treatment of Cancer (EORTC), BE
Best practice: The International Rare Cancers Initiative
M. Seymour, National Cancer Research Network, UK
Best practice: International collaboration on sarcomas
A. Gronchi, Fondazione IRCCS Istituto Nazionale dei Tumori, IT
Best practice: International collaboration on a very rare cancer (adrenal carcinoma)
A. Berruti, University Hospital, Orbassano, Torino, IT
Best practice: Paediatric oncology
G. Vassal, European Society for Paediatric Oncology (SIOPE), FR
Best practice: Patient involvement in clinical studies
Š. Narbutas, ECPC, LT
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
R. Schaefer, Rare Cancers Europe, DE
Designs of clinical studies in rare cancers
J. Bogaerts, European Organisation for Research and Treatment of Cancer (EORTC), BE
Bayesian approaches to analysing studies and summarizing evidence
P. Bruzzi, National Cancer Research Institute, IT
Bringing evidence within clinical decision-making
P. G. Casali, European Society for Medical Oncology (ESMO), IT
Organisational and regulatory aspects of clinical studies
J.-Y. Blay, European Organisation for Research and Treatment of Cancer (EORTC), FR
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
Statistics / Epidemiology
J. Bogaerts, European Organisation for Research and Treatment of Cancer (EORTC), BE
P. Bruzzi, National Cancer Research Institute, IT
Regulatory Affairs / Health administration
B. Jonsson, MPA, SE
Pharmaceutical industry
S. Comis, Novartis Oncology, IT
E. Colajori, Pfizer Oncology, IT
J. Adamczewski, Sanofi, FR
Biology / Pathology
A. P. Dei Tos, European Society of Pathology (ESP), IT
Patient advocacy
S. Craine, The CML Support Group, UK
J. Bressington, GIST Support UK & PAWS-GIST, UK
J. Geissler, EURORDIS and CML Advocates Network, DE
J. Pelouchová, ECPC, CZ
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