Event Report: “Patient Safety and Informed Consent: The Importance of Good Off-label Use Practices” 

On 17 June 2021, the GOLUP consortium held a virtual event, led by the European Brain Council, to disseminate the Declaration on Good Off-Label Use Practices.

The event was hosted by MEP Cyrus Engerer (S&D, Malta), who is a member of the public health (ENVI) committee  of the European Parliament. There was a broad interest in a renewed discussion on this topic, with the audience comprised of a wide range of stakeholders, with representatives from the scientific and clinical community, patient organisations and industry.

The event was an opportunity to promote the declaration and resume the dissemination process started five years ago by the first co-signatories of the document. The time seemed right to take up the discussion again from a patient safety perspective, with health given the spotlight due to the COVID-19 pandemic and planned revision of the general pharmaceutical legislation as well as the cross-border healthcare directive.

The roundtable was opened by an intervention from MEP Cyrus Engerer, followed by EBC’s Executive Director Frédéric Destrebecq who presented the potential role of the GOLUP Declaration in promoting a common European approach for off-label use and the four criteria that are the basis of the declaration.

The event was also the occasion to showcase for the first time an awareness raising video on off-label use and the GOLUP declaration which was produced by EURACTIV. The video explains what off-label use means and the aims of the GOLUP Declaration, with the ultimate goal of raising awareness of the topic and reaching a wider audience

Key takeaways:

Cyrus Engerer  
Member of the European Parliament, Malta 

Mr Engerer emphasised that this phase we are living through, the end of the pandemic and the lessons we must learn from it, is a unique opportunity to try to rebuild the European healthcare system with a patient-focused approach.. With the own initiative report of the European Parliament, he said “…we will have the opportunity to address the lack of a harmonised framework for off-label use…”. The European Union should aim for the setting-up of guidelines based on best practices for appropriate use of off-label medicines, and put the patient at the centre of care.

“There is a clear momentum for more action and more ambition in this area. This is the time to put on the table certain topics that up to now have not been prioritized by EU and which require coordinated action.“


Jana Hlaváčová 
Member of the Executive Committee, European Multiple Sclerosis Platform (EMSP)

Ms Hlaváčová brought the patient perspective explaining how off-label treatments are commonly offered to MS patients both for disease modifying treatments and for symptomatic treatments. Off-label use is particularly used in the care of children living with MS as there is a limited choice of care. One noticeable issue for MS patients is when off-label use is promoted for budgetary reasons, a decision which is more economic than strictly medical, and can lead to a drastically reduced number of viable treatment options

“The harmonised criteria for the off-label use are vital. From a patient’s point of view everything that you need to have in a good doctor-patient relationship is the necessary information to give an informed consent.”

Adela Maghear,  
Senior EU Affairs Officer, European Cancer Patient Coalition 

Ms Maghear underlined how relevant the topic is as drugs are commonly used off-label in oncology. Despite widespread off-label use, the practice remains controversial due to the uncertainty around the clinical benefits and the limited evidence to support the clinical decision-making process. Ms Maghear  went more in depth on the use of off-label drugs in cancer treatment, and explained how this type of treatment is on the rise due to the ever-increasing proportion of cancer survivors for whom doctors want to ensure a better quality of life in the absence of precise guidelines. She made it clear that the EU should adopt common guidelines at the EU level to prevent the differing approaches between Member States. This would provide clearer guidance for clinicians and signal to governments how to appropriately use drugs off-label.

“Consent forms should be shortened and drafted in a language that makes them easy to understand for patients.”


George Casimir 
President, Belgian Royal Academy of Medicine 

Georges Casimir
President, Royal Academy of Medicine of Belgium

Professor Casimir provided a pediatrician’s experience on the driving forces behind the widespread use of off-label drugs for children. Even with the most common drugs, it is in general difficult to carry out scientific studies on children and infants. In addition, children are less often treated with drugs than adults and they therefore represent a much smaller patient population on a purely commercial basis. For parents it is almost impossible to authorize such studies unless a child’s life is compromised by illness and the drug offers the possibility of saving them.

“These complex drivers of off-label use of medicines for children justify bringing all stakeholders together in order to find the most efficient solution”


Coriene Catsman–Berrevoets 
Chair, EPNS Education and Training Committee 

Professor Catsman-Berrevoets explained how the prevalence of off-label use of medicines in children with paediatric neurological problems is high. In current daily practice the field is highly dependent on off-label use in many types of treatments. This is due to two main reasons: medications are designed and registered for adults and only later for younger age groups and because of the rarity of these diseases efficacy of drugs can only be demonstrated in large multicenter studies, which are hard to organise and to finance.

“From the paediatric neurology perspective we need to enable a framework within the EU that can take account of the lacking evidence of off-label drug efficacy and possible side effects in paediatric disease, which is up to EU marketing standards.”


Mike Isles 
Executive Director, European Alliance for Access to Safe Medicines  

Mr Isles brought to light some examples of patients being harmed due to adverse events in Europe over the past years, and underscored that due to the lack of reporting these cases might only be the tip of the iceberg. In 2015 the EAASM wrote a letter to Commissioner Vytenis Andrukaitis explaining that patients had suffered serious eye infections during a procedure in the ophthalmology department of the hospital of Careggi in Florence, Italy. The case fundamentally calls into question the effectiveness of the current reporting of adverse events following off-label use in the EU. Following this, EASSM launched a series of calls to actions: establishing the number of adverse events relating to off-label use of medicine; introducing a professional code of practice for mandatory report by healthcare professionals of adverse events involving off-label use and improving public awareness so that patients are aware of when off-label drugs are used.

“Due to the lack of reporting of adverse events caused by off-label use, there are a number of unknowns with regards to how many patients have actually suffered harm from improper practices. Decisions have to be made in conjunction with the patient and easily understandable consent forms must be used together with an effective reporting system.”


Marc Dooms
Senior Orphan Drug Pharmacist, University Hospital Leuven 

Professor Dooms who originally authored the GOLUP declaration appreciated the renewed opportunity for EU decisionmakers to consider the establishment of EU guidelines for off-label use. He explained that it is important that the healthcare system is set up to ultimately take care of the patient, and that is why we need guidelines such as those that are clearly expressed within the GOLUP Declaration.

“Informed consent can now be given electronically. The patient with a smartphone can read all the necessary information and give informed consent for off-label use.”