Action Needed to Protect Patients From Unlicensed /Off-Label Medicines

LONDON, September 20, 2011 -

European patient groups call for Code of Practice following FDA alert

Four European patient organisations today published a consensus statement raising concerns about the unlicensed and off-label use of medicines, and calling for a professional Code of Practice.

The consensus statement follows recent warnings by the US Food and Drug Administration (FDA) about a cluster of serious eye infections in Florida associated with the off-label use of an unlicensed medicine.¹ Although the exact cause is currently under investigation, the common link for the infections is the repackaging of an unlicensed medicine for use in eye clinics.

Whilst all the patients involved had visual defects before their injections with this unlicensed medicine, some patients lost all remaining vision in that eye due to endophthalmitis (an inflammatory condition in the eye usually caused by infection). There have since been other cases reported by the media in other parts of the United States. The New York Times reported five patients blinded through off-label eye injections in Los Angeles, and also reports another earlier situation in Tennessee which is subject to legal action.^2,3

There have also been reported cases of adverse events in Europe caused by similar off-label use of the same medicine. In Germany, five cases of serious eye inflammation has resulted in legal action by the patients involved, and in Austria, eight cases of eye inflammation have been reported.^4,5

The patient organisations’ concern is that the true extent of the problem is not known and there are likely to be unreported occurrences across Europe due to there being no formal and obligatory mechanisms (unlike the case where a medicine is prescribed within its licensed clinical indication) for reporting adverse events involving unlicensed and off-label medicines.

“Our message is enough is enough,” said Jim Thomson, Chair of the European Alliance for Access to Safe Medicines (EAASM). “The FDA was able to act because it was made aware of the problem. In Europe it’s a matter of chance as to whether the regulatory authorities are made aware of similar problems because there is no mandatory mechanism to report adverse events in medicines used this way.

“We strongly believe unlicensed and off-label medicines should only be used when a licensed product is unavailable. In addition, patients should be informed and provide written consent for the medicine to be used and there should be a mandatory adverse event reporting system in place, just like there is for medicines when used for their approved licensed indications.”

The patient organisations involved have put their name to the consensus statement and will be urgently seeking meetings with European regulatory and professional bodies over the coming weeks.

The consensus statement reads:

Adverse Events and unlicensed/off-label use of medicines

A Consensus Statement

We believe there is an urgent need to address patient safety issues around the unlicensed/off-label use of medicines.

As a minimum criterion, we believe that unlicensed/off-label medicines should normally only be used in cases where there is no licensed product available that meets the medical need. If this condition is satisfied, a number of points still need to be met to ensure patient safety is not compromised.  

Adverse Events (AE) reporting is a critical component in the continuous monitoring and assessment of medicines safety. However there is currently no mandatory mechanism in place for recording and/or reporting AEs in the use of unlicensed/off-label medicines. In addition, the level of awareness of AE reporting amongst the general public is alarmingly low.

We believe action is required to:

• Establish the number of AEs relating to unlicensed/off-label use of medicines
• Introduce a professional Code of Practice for mandatory reporting by healthcare professionals of AEs involving unlicensed/off-label medicines as currently there is no mandatory mechanism for recording or reporting AEs in this context
• Improve public awareness so that patients are aware of when unlicensed/off-label medicines are used and the importance of AE reporting, and that they have to give their formal written consent for those medicines to be used

Jim Thomson       Chair: European Alliance for Access to Safe Medicines

Mary Baker        President: European Federation of Neurological Associations

Dr Ian Banks      President: European Men’s Health Forum

Amelia Mustapha   Vice-President: European Depression Association

Note to editors: An unlicensed medicine is a drug that does not have a marketing authorisation. Off-label refers to the use of a drug which has a marketing authorisation but is used for a condition, at a dose, via a route or for an age that is not listed in the Summary of Product Characteristics for that drug.

References:

1. Food and Drug Administration (FDA). Website. Available at: www.fda.gov/Drugs/DrugSafety/ucm270296.htm Accessed September 2011
2. New York Times. Website. Available at: www.nytimes.com/2011/08/31/health/31drug.html Accessed September 2011
3. New York Times. Website. Available at: www.nytimes.com/2011/09/02/business/more-reports-of-avastin-causing-blindness.html?_r=2 Accessed September 2011
4. Georgopoulos et al, Characteristics of severe intraocular inflammation following intravitreal injection of bevacizumab (Avastin). British Journal of Ophthalmology, 2009;93(4):457-462
5. Der Spiegel. Website. Available at: wissen.spiegel.de/wissen/image/show.html?did=60666870&aref=image038/2008/09/27/ROSP200804001400141.PDF&thumb=false.  Accessed September 2011

For further enquiries or to request an interview contact: Jim Thomson, Chair, EAASM, T: +44 (0)1702 474230, M: +44 (0)7901 800608, E: jim.thomson at eaasm.eu; Claire Nicholson The Red Consultancy, T: +44 (0)207 025 6524, M: +44 (0) 07715 002984, E: Claire.nicholson at redconsultancy.com