PROMOTING GOOD PRACTICE
FOR THE OFF-LABEL
WHAT IS OFF-LABEL USE?
Before being placed on the market, medicines obtain an authorisation and a label for the indication for which they are tested. This testing follows very rigorous regulatory standards and procedures to scientifically demonstrate that the medicine is efficacious and of high quality but also minimises as much as possible risks to patient safety.
Off-label use is the practice of using a medicine outside its authorised indication. It plays an important part in some medical areas, since it can be of benefit to patients when no other treatment option is available, such as it is often the case in rare diseases or in the case of child and adolescent care.
WHAT SHOULD YOU KNOW ABOUT OFF-LABEL USE?
Some European Union Member States are imposing prescribing guidelines that effectively promote off-label use for the sole purpose of reducing healthcare spending. We are concerned that this trend is creating unnecessary and avoidable risks for patients and often puts healthcare professionals in the position of being liable for a decision that they have not taken in full autonomy, and is potentially disrespecting the medical needs of the patients.
WHAT IS GOOD PRACTICE?
We believe that off-label use of medicinal products should only occur if the following criteria are met:
- 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 need 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.
These criteria, drawn together by an independent expert, stem from decades of research and clinical practice and serve to provide a clear framework on when and how the off-label use of medicinal products could safely take place.
They do not aim to limit off-label use, but seek to promote a harmonised approach for its occurrence in order to maintain the highest levels of patient safety and minimise adverse events.
CALL TO ACTION!
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.
A clear, Europe-wide approach on the issue is becoming increasingly urgent to preserve the European regulatory framework and ensure the safety of patients.
NEWSLETTER
We thus call on the European Medicines Agency and other national regulatory bodies 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 benefit does not prevail over public health.
Endorse the Declaration on Good Off-Label Use Practice
The Declaration on Good Off-Label Use Practices is open to any national and European organisation which supports its principles and objectives.
Download the declaration
Share your experience
We are interested in knowing your experience with off-label use. If you are interested in sharing it,
FOLLOW THE CAMPAIGN
EVENTS
Patient safety and informed consent:
The importance of good off-label use practices
The purpose of this event is to allow an exchange of ideas and experience among stakeholders and local actors, to understand which paths to take in individual Member States and to allow a continuous exchange of ideas and information on the off-label use of medicines.
17th June 2021
National Workshop on Off-label Use of Medicines Declaración sobre buenas prácticas del uso fuera de guía de medicamentos en España – 25th April 2018
Good Off-Label Use Practice (GOLUP) National Roundtable – Italy On 13th December 2017, the European Brain Council (EBC) participated in a roundtable on the use of off-label medicines in Rome, Italy. This was the first of a series of national roundtables to be organised by the Good Off-Label Use Practice (GOLUP) coalition.
EBC Hosts Roundtable on Off-Label Use of Medicines On the 10th of July, the European Brain Council (EBC) hosted an extremely insightful roundtable on “Ensuring the safe prescription of medicines off-label in Europe: what role for the European Union?”.
Kick-off debate on off-label use of medicines in Europe
Safeguarding patient safety and quality of care in europe Tuesday, 27 September 2016, European Parliament
NEWS
A harmonised EU approach to the off-label use of medicines?
The European Brain Council’s Frédéric Destrebecq and MEP Cyrus Engerer explain how European patients can benefit from a properly regulated off-label medicine framework.
Professor Marc Dooms presents the GOLUP declaration at the ECRD
On 10th-12th May 2018, EURORDIS hosted the ninth European Conference on Rare Diseases & Orphan Products (ECRD) in Vienna, welcoming almost 1000 participants from more than 50 countries.
Off-label use of medicines: The need for good practice guidelines – the International Journal of Risk and Safety in Medicine
Article written by Dooms and Killick in which they present a set of guidelines for best practice in off-label use rooted in the need to ensure patient safety above all other concerns.
“EU needs guidelines on off-label us of medicines” argues MEP Piernicola Pedicini Since the European Parliament requested better guidance on the widespread off-label use of medicine, very little has been done, writes Piernicola Pedicini
Response to Professor Guido Rasi: Roundtable to discuss solutions to regulate off-label use
Working together towards clear EU Guidelines on the Off-Label Use of Medicinal Products
Our response to the European Commission’s study on off-label use in the EU
Promoting off-label use: where to draw the line?
Research study by JAMA Internal Medicine:
Association of Off-label Drug Use and Adverse Drug Events in an Adult Population
Response from EMA: Upholding the Good Off-Label Use Practice
Call to EMA: Upholding the Good Off-Label Use Practice
Letter addressed to Professor Guido Rasi, Executive Director, European Medicines Agency, on behalf of the GOLUP Coalition
European Multiple Sclerosis Platform A patient safety initiative: promoting good practice for off-label use of medicines