EBC organises a breakfast meeting on the GOLUP Declaration in the European Parliament

Good off-label use practices are often a necessity in areas of unmet medical need. The prevalence of off-label use in the EU in both the paediatric and adult populations is high in a broad range of therapeutic areas such as rare diseases, oncology, psychiatry, neurology, in both hospitals and outpatient settings. Off-label practice poses a range of quite different challenges such as ethical and legal issues for healthcare professionals, increased risk for patients, patient information and consent. While not optimal but essential to address unmet medical needs, the manner in which countries deal with the off-label use of medicines is not harmonised across the EU.

In 2016, the Good Off-Label Use Practice (GOLUP) Declaration was launched, supported by a coalition of European organisations dedicated to ensuring that high standards of patient care are upheld and that progress in medical research and innovation is achieved. The Declaration put forward the basis for a harmonised approach to when and how off-label prescription should take place across Europe.

The Declaration put forward the basis for a harmonised approach to when and how off-label prescription should take place across Europe:

• Presence of a medical therapeutic need based on a current examination of the patient by a suitably qualified health care professional;
• Absence of authorised treatment and licensed alternatives tolerated by the patient or repeated treatment failure;
• A documented review and critical appraisal of available scientific evidence favours off-label use to respond to the unmet medical needs of the individual patient;
• Patients (or their legal representative) must be given sufficient information about the medicines that are prescribed to allow them to make an informed decision;
• Presence of established reporting routes for outcomes and adverse events linked to off-label use.

The goals of this event are to reconnect with the GOLUP Declaration promoters, exchange ideas and experiences to secure EU and national recognition of the Declaration with the ultimate goal to promote a common European approach for off-label use, call for and propose EU guidelines for the good use of off-label medicinal products. This event is timely, as the topic of repurposing has gathered attention with :

• the European Medicines Agency (EMA) and the Heads of Medicines Agencies' Repurposing of Authorised Medicines pilot launched in 2021 to support not-for-profit organisations and academia to support the repurposing of medicines as a follow-up to the European Commission’s Expert Group on Safe and Timely Access to Medicines for Patients (STAMP) discussions on a proposal for a medicines repurposing framework,
  • the Spanish EU presidency event on the topic of ‘Repurposing and better use of clinical data’ on 27 September, in Madrid,
  • the revision of the pharmaceutical legislation with the Directive on medicinal products for human use looking inter alia at adverse reactions reporting in the case of off-label use products (Article 105) and unmet medical needs that are one reason and objective of the proposal (Exploratory memorandum and article 83) as well as the Regulation for the authorisation and supervision of medicinal products for human use and the expected European Commission proposal on rare diseases.