Organised by the European Society for Medical Oncology 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 were the product of an ongoing multidisciplinary and multi-stakeholder online consensus discussion, promoted by Rare Cancers Europe. They focused 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 were encouraged to actively participate in this open discussion, the result of which was a consensus paper 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.
Rare Cancers Europe
Rare Cancers Europe is a multi-stakeholder initiative addressing specific challenges posed by rare cancers. It is based on a partnership between the European Society for Medical Oncology (ESMO), the European Organisation for Rare Diseases (Eurordis), the European Cancer Patient Coalition (ECPC), the European Organisation for Research and Treatment of Cancer (EORTC), CONTICANET, EuroBoNeT, the Association of European Cancer Leagues (ECL), the Chronic Myeloid Leukaemia Support Group, the International Brain Tumour Alliance (IBTA), Orphanet, the Chronic Myeloid Leukaemia Advocates Network, GIST Support UK & PAWS-GIST, the Fondazione IRCCS Istituto Nazionale dei Tumori, the European Institute of Oncology (IEO), the European Society for Paediatric Oncology (SIOP Europe), the European School of Oncology (ESO), the European Oncology Nursing Society (EONS), the European Society of Pathology (ESP), Novartis Oncology (initiating sponsor and sustaining industry partner), Pfizer Oncology (sustaining industry partner), and Sanofi-Aventis (sustaining industry partner). The initiative is moreover supported by additional corporate supporters.