This initiative aims to provide a clear framework on when and how the off-label use of medicinal products could safely take place.
A clear approach on the issue is becoming increasingly urgent due to the current trend of promoting the prescription of medicines off-label for reasons other than the medical need of patients. In fact, some European Union Member States are imposing prescribing guidelines that effectively promote off-label use for the sole purpose of reducing healthcare spending.
These criteria reaffirm that patient safety should always prevail over considerations of cost and physicians should be supported by the public bodies and authorities responsible for the approval and use of medicines whose role is to protect public health.
These criteria stems from decades of research and clinical practice. In fact, while a Europe-wide framework in this area is still lacking, similar principles for off-label use have been recommended by the UK General Medical Council or the Belgian Healthcare Knowledge Centre.
Using his 40-year long experience in the area, the author has identified the best balance between allowing enough therapeutic options, while protecting patients and healthcare professionals from risks coming from the off-label use of medicines. The principles have also gone through a number of discussions with experts and stakeholders in this area, which resulted in the Declaration endorsed.
No, this is not the intention of the signatories. The off-label use of medicines is a common and important practice in a number of medical areas such as in paediatrics, gynecology, psychiatry, palliative care, or for the treatment of rare diseases.
These criteria, that were drawn together by Professor Marc Dooms, an independent expert, aim to provide a harmonised approach on when and how the off-label use of medicinal products could safely take place.
Their aim is thus to ensure the highest levels of patient safety and minimise adverse events when the use of off-label medicines is the viable option.
The signatories are aware that several diseases still lack authorised option and they individually advocate for increased medical research in these areas.
For patients with limited or no authorised options, a clear framework for the prescription and use of off-label medicines is especially important since the only alternative available could be off-label.
Due to the complexity of the matter and the specific scope of this initiative, the extension of the medicines’ indications have not been covered at this point.
We believe health professionals should be supported by the public bodies and authorities responsible for the approval and use of medicines whose role is to protect public health. Physicians need to be given the freedom to uphold their pledge towards their patients to act ethically and put the patient’s interest first.
We thus call on the European Medicines Agency to adopt strict guidelines to support healthcare practioners to take their decision on the basis of the medical need of their patients and ensure economic benefits do not prevail over patients’ health.
If you are representing a European or national organization that supports the principles and objectives of the Declaration on Good Off-Label Use Practice, you can join this initiative by contacting as at the email address provided in the website.
As an individual, you can share your experience on off-label us with us by sending an email to the address provided in the section contact us. It will help us to strengthen our messages!