Innovative Therapies for Children with Cancer in Europe

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Why is a new regulation necessary? What will it change ?

The use of unlicensed and off-label medicine in children is widespread and it has been an increasing concern over the last few years (An unlicensed and off label drug is being used in a situation for which the drug has not been tested in a clinical trial).


In the EU, fifty per-cent or more of medicines used in children have never been investigated in this population, but only in adults and not necessarily for the same indication (or the same disease).


Of 258 active substances registered (1995- 2006), 32% have a paediatric indication, 44% have a potential paediatric indication and 24% have no paediatric indication.


The general lack of information and appropriate pharmaceutical formulations to support the administration of many medicines to children may expose them to unwanted side effects or under dosing without the expected efficacy.Children are not “small adults” and represent a vulnerable population who differ from adults developmentally, physiologically and psychologically . Therefore age -and development-related clinical research is important.


In addition to the general lack of paediatric drug development in Europe, major disparities currently exist between different European member states. In some countries, such as the UK and France, only a small number of experimental studies can be conducted because of major difficulties in obtaining new drugs for evaluation. In other countries, such as Germany, every family has the right to receive any drug that is marketed in adults, even if no safety or efficacy data are available to support its use in children. Finally, in many countries, new drugs are not available at all for children, and this can lead to desperate parents inappropriately sending terminally-ill children to foreign countries (particularly the USA).


In paediatric oncology, access to innovative therapies developed by pharmaceutical companies has so far been extremely limited for children in Europe, one reason being that paediatric oncology does not represent a large, and hence financially attractive area for drug marketing.

Of 25 authorised products (1995-2002) for the diagnosis or treatment of a malignancy or cancer-related condition, only two of them were evaluated in children prior to submission.


Paediatric oncologists have conducted academic prospective clinical trials during the last 40 years without support from the pharmaceutical industry. Using drugs available on the market for adults, they set up prospective trials to improve the cure rates. Through this clinical research network, paediatric oncologists have learned to work together. Cure rates have been increased from less than 20% in the 1950s to over 75% in the 1990s and this level was achieved without any input from pharmaceutical companies because of limited commercial interest.


The European Commission recently issued a New Regulation called “Medicines for Children”. This regulation stipulates that a new drug should be evaluated in children before it can obtain a marketing authorisation from the EMEA (European Medicines Agency). A large number of new compounds with novel mechanisms of action are being developed in oncology in adults. We need access to these new agents developed by the pharmaceutical industry to further improve the cure rate and the quality of cure of children with cancer. More than 90% of anticancer drugs that should be evaluated in children within the next 10 years are expected to come from big pharmaceutical companies or small “start up» companies.


Further improvement will require the commitment of pharmaceutical companies and this is now the case because of the new regulation. With effective collaboration between the paediatric cancer networks, clinicians, the pharmaceutical companies and regulatory authorities, it should be possible to keep new drug development for children with cancer on the agenda.