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

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

The event was hosted by MEP Cyrus Engerer (S&D, Malta), member of the Committee on the Environment, Public Health and Food Safety. The panel and the audience comprised a wide range of key 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, as the legislative framework is very dynamic with the discussion of different health-related issues that are connected to the principles of this Declaration (e.g. EU Pharma Strategy, revision of the cross-border care directive, Green Paper on Ageing). 

The roundtable was opened by an intervention from MEP Cyrus Engerer, followed by EBC’s Executive Director Frédéric Destrebecq who presented the role of 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 the first GOLUP video, produced by Euractiv, explaining what off-label use means and the aims of the Declaration. The video has the goal of raising awareness to the topic and reach a wider audience. 

Key takeaways:

Cyrus Engerer  
Member of the European Parliament, Malta 

MEP 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 patient-focused approach to healthcare on a shared ground. With the own initiative report of the European Parliament, we will have the opportunity to address the lack of a harmonised framework for off-label use. In addition to best practices for use of off-label medicines that put the patient at the centre, there should be a framework in place which makes repurposing of medicines attractive. Such a framework should support the marketing and use of an existing medicine ‘on-label’, with an extension to a new therapeutic area, rather than resorting to using medicines off-label. 

“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. We now know that we greater cooperation among Member States is a winning formula that can be extended beyond the emergency situation.” 


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

Ms Hlaváčová brought the patient perspective explaining how often off-label treatments are offered to MS patients both for disease modifying treatments and for symptomatic treatments. The problem is particularly acute with regard to the care of children living with MS as there is a limited choice of care. It also raised the issue of off-label use for budgetary reasons, making the decision more economic than strictly medical. 

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


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

Ms Maghear underlined how relevant is the importance of off-label use in oncology, remaining an issue of controversy due to the uncertainty around the clinical benefits and the limited evidence to support the clinical decision-making process. She then went more in deep on the use of off-label drugs in cancer treatment, explaining 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. 

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


George Casimir 
President, Belgian Royal Academy of Medicine 

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

“This complex aspect of the use of medicines for children, the ethics of pharmacological studies and the cost of the research justify bringing pharmaceutical companies and European health authorities to the table with doctors and ethicists as well as economists in order to find the most efficient solution considering the GOLUP Declaration.” 


Coriene Catsman–Berrevoets 
Chair, EPNS Education and Training Committee 

Professor Catsman represented the European Paediatric Neurology Society, a society for physicians with a research or clinical interest in Paediatric Neurology. Prevalence of off-label use of medication in children with paediatric neurological problems is high. In current daily practice we highly depend on off-label use in many types of treatments. This is due for two main reasons: medications are designed and registered for adults and only later for younger age groups; because of the rarity of these diseases efficacy of drugs can be demonstrated in large multicenter studies, hard to organise and to finance by independent University centers. 

“From the paediatric neurology perspective we need to enable multicenter programmes within the EU to raise the level of evidence of off-label drug efficacy and possible side effects in paediatric disease to EU marketing standards.” 


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

Mr Isles brought some practical examples of patients being harmed. 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. This fundamentally calls into question the reporting of adverse events following off-label use in the EU. EASSM launched a series of call to actions: establishing the number of AEs relating to off-label use of medicine; introducing a professional code of practice for mandatory report by healthcare professionals of AEs involving off-label use; improving public awareness so that patients are aware of when off-label drugs are used. 

“Decisions have to be made in conjunction with the patient. The consent form has to be easily understandable.” 


Marc Dooms
Senior Orphan Drug Pharmacist, University Hospital Leuven 

Professor Dooms explained how it is important to take care of patients with rare diseases as we only have few types of orphan drugs. Therefore, we need guidelines 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.”