5 October 2010
Links to presentations are below

Today MEP Frieda Brepoels hosted a lunch meeting in the European Parliament on problems with development and availability of orphan drugs for rare cancers. The meeting was organized by the  Association of European Cancer Leagues, the Foundation Against Cancer and the Flemish League Against Cancer. The aim was to raise the awareness about this issue and motivate the parliament to take action. Mrs. Brepoels opened the meeting by explaining the vital importance of the topic. Orphan drugs are drugs for rare diseases, e.g. rare cancers.


But rare cancers are not rare: Taken together they represent 20% of all cases of cancers. Some of these cancers have a very low survival rate. The 5 year survival of glioblastoma, a kind of brain tumour, is below 10%. So there's an urgent need for new treatment modalities. The availability of orphan drugs already improved considerably, because of the European regulation on orphan drugs of 2000 but there are still bottlenecks in the development and availability of these orphan drugs. “The development of orphan drugs is hampered by the small expected return on investment and difficulties in the organisation of clinical trials. Patient access to orphan drugs could be faster and better if EU member states were to exchange more information on the clinical added value of an orphan drug during the reimbursement decision. These issues can only be tackled on a European level,” Jean-Jacques Cassiman, President of the Flemish League Against Cancer and moderator of the meeting, said in his  introduction of the panel discussion.



A testimony from a patient living with a brain tumour made these bottlenecks tangible. After several other treatments, her doctor proposes a treatment with AVASTIN (in August 2009), a cancer drug showing promising results in patients with her disease. Only there is this problem: the drug is very expensive  (several thousands of euros per month) and not yet reimbursed by the Belgian public health insurance. The patient starts out on the treatment but this is also the beginning of months of financial insecurity, during which she did not know how much the treatment was going to cost her. "What I find deplorable is that I am spending all my physical and mental energy on solving financial issues when these should be spent fighting for my life", she said.

tezer  Callens  Yann  paolo  


After presentations by Paolo Casali, (Board Member, European Society for Medical Oncology (ESMO)), Michiel Callens (President, Belgian Cancer Registry), Yann Le Cam (CEO, Rare Diseases Europe (EURORDIS)), André Lhoir, (Committee for Orphan Medicinal Products (COMP), Belgium) and after a vivid discussion between these speakers and the audience consisting of MEPs and other stakeholders, Didier Vander Steichel spoke for Michel Symann, President of the  Foundation Against Cancer (FCC)) with the following conclusions:

-There is a need for innovative methodologies in clinical trials on rare diseases and regulatory requirements for clinical trials should be kept to a minimum. This will facilitate the development of new orphan drugs.
-European networks of centers of expertise are a prerequisite for the organisation of clinical trials on rare cancers  and for the development of novelties in the field of human tissue and cell derived products.
-To improve the treatment of rare cancers, all available data sources should be exploited. Anecdotal reports and population registries are important. We need European-wide databases consisting of pooled nationwide registries. There is a definite need for networks of clinical databases and tissue banks which would improve collection, harmonisation and provision of data on rare cancers.

-New therapies for rare cancers should be as soon as possible available and accesible for every European citizen. But reimbursement agencies may find it difficult to deal with the uncertainty on efficacy of drugs in rare cancers, which is inherently higher than uncertainty about the efficacy of drugs for more common cancers.


Another challenge for reimbursement and solidarity is posed by the coming marketing of the expensive  cell and tissue derived products. A common European assessment of the clinical added value of an orphan drug by EMA, the European Medicine Agency, can help to overcome these challenges. By sharing expertise and knowledge between the member states and EMA, we can gain time and discover new ways to deal with uncertainty.


MEP Frieda Brepoels concluded the meeting, by calling upon the MEPs to:   

1) make proposals to keep regulatory requirements for clinical trials on orphan drugs to a minimum without endangering patient safety;   2) ensure that there is EU funding for networks of centers of clinical and scientific excellence;  3) support the development of EU-wide registries, amongst others by securing full interoperabilty of national e-Health services, by creating a regulatory and legal framework; 4)  support close collaboration between member states during the phase of deciding on the reimbursement so that common arguments are used and comparable prices can be negotiated with industry; and to 5) install a non-binding European evaluation of the clinical added value of a new orphan drug, as one way to improve European collaboration in the reimbursement decision.

Presentations from the event are now available:

What can the EU do to improve how we measure efficacy of orphan drugs in rare cancers? Speaker: Paolo Casali, Board Member, European Society for Medical Oncology (ESMO)

Importance of cancer registries and e-health for development of and access to orphan drugs. Speaker: Michiel Callens, President, Belgian Cancer Registry

Sustainable and improved access to orphan drugs: why and how Member States should collaborate in the European Union.  Speaker: Yann Le Cam, Rare Diseases Europe (EURORDIS)


Conclusions - Michel Symann, President, Foundation Against Cancer (FCC) (Presented by Dr. Didier Vander Steichel)

For more information about this event, please contact: This email address is being protected from spambots. You need JavaScript enabled to view it..

For information about ECL events at the European Parliament, please contact the This email address is being protected from spambots. You need JavaScript enabled to view it..




 The Association of European Cancer Leagues implements activities which receive financial support from the European Commission under an Operating Grant from the European Union's Health Programme (2014-2020). The views expressed on our website and reports do not necessarily reflect the official views of the EU institutions.
EU flag and co-funded by health programme LOGO  




The text of this webpage is available for modification and reuse under the terms of the Creative Commons Attribution-Sharealike 3.0 Unported License and the GNU Free Documentation License (unversioned, with no invariant sections, front-cover texts, or back-cover texts).


Go to top