Marc dooms elaborated in a recent interview on some of the issues and challenges associated with off-label use of medicines across Europe. Additionally, he shared his views on the principles laid down in the Good Off-Label Use Practice (GOLUP) declaration and how off-label use affects patient safety.










What is off-label use of medicines? And why is it a practice that affects patients?

Every medicinal product comes on the market with a package insert that is called a label. All information on the label regarding the indication, required dose, route of administration, etc. is proven to be safe and effective.

Sometimes, medicinal products are used outside that label. This means that the medicine is used to treat a condition for which it is not approved or in a higher or lower dose than indicated on the label. Medicinal products are used off-label if they are prescribed for another indication, in another dose, via another route of administration or for other patient groups mentioned on the label.

There are currently thousands of rare diseases but very few treatments. Therefore, instead of telling patients that there is no treatment that has proven to be effective for that specific disease, healthcare professionals use other medications that could potentially work. Prescribing medicinal products off-label for patients with rare diseases who have no on-label treatment is therefore positive in my opinion.

What are your views on the prevalence of off-label use of medicines across the EU?

It is difficult to give an exact number. I think that on average it must be between 20 and 60 percent. However, 99 percent of medications used to treat children with rare cancers are prescribed off-label. This is because the labels of the medicinal products used to treat these cancers do not indicate that the medications can be safely used for children. Therefore, medicinal products are prescribed off-label as alternative to not treating children with rare cancers. This has happened for many years and in some cases it has proven to be effective.

What is, in your opinion, the most pressing risk associated with using medicines off-label?

For me, the biggest risk is patient safety. We are not sure yet about the safety of the patient when prescribing medicines off-label.

In my opinion, we need excellent exchange of information in order to enhance patient safety. Certain medicinal products could have been used to successfully treat patients in healthcare centers in Amsterdam, Paris or Warsaw. Though healthcare professionals in other cities might not have access to information on the clinical outcomes in those cases. Reports on off-label treatments can be found in the literature, though the results of many treatments are often not published if the clinical effects are not positive. Therefore, I think that we need a very good system that allows healthcare professionals to exchange information on the safety of using medicines off-label.

Are the current legislative frameworks at EU level effectively addressing the risks presented by the off-label use of medicines?

They are until a certain extent. EudraVigilance allows patients to report secondary effects of medical treatments. Though this system is not well known and not effectively used. It would therefore be much better if patients use it more frequently. But in my opinion, policy-makers really need to work towards establishing a good system for exchanging information.

Are the principles laid-down in the Declaration on Good Off-Label Use Practice (GOLUP) declaration addressing some of the challenges presented by off-label use in the EU?

With these guidelines, patients can be treated in a very effective way that is not yet on-label. The GOLUP-criteria are therefore, in my opinion, very positive for patients. What is more, they are particularly beneficial to people affected by rare diseases because approved treatments are often not available in these cases.

The good off-label use practice declaration is the result of close to 30 years of experience with off-label use. 5 golden rules were formulated and I am absolutely sure that the safety of off-label use of medicinal products will be improved if healthcare professionals stick to them.

What could be done by patient representatives and advocates to support the principles laid down in the declaration?

I think that they need to approach their MEPs and ask them to bring the best possible regulation for improving the exchange of information of off-label treatments as soon as possible. This would be advantageous for patients living with rare diseases.