Revue Prescrire, article en une, Medicines in Europe Forum leaflets (8)
Prescrire     Accueil  
Article en Une - Archive
Medicines in Europe Forum leaflets
Why must there be a European regulatory framework to guarantee "compassionate use" of medicines in terminally ill patients?
Leaflet N°8
09 September 2002

Medicines in Europe Forum has proposed an amendment to the proposed Regulation aiming to strengthen the framework of compassionate use through incentive, constraints and recommendations that will allow exceptional access to new medicines sufficiently early and commensurate to the needs of those patients who are terminally ill.


List of leaflets

Previous leaflet

Next leaflet

Medicines in Europe Forum
Click here
We call "terminal illness" the situation where patients suffer from serious illness for which there is no possibility of treatment. This could mean that the medicines available to a seriously ill patient are not effective, that the patient cannot follow a course of treatment because of serious adverse effects or patients who have an illness, for which no treatment is available.

For these people whose life is threatened in the short term by a lack of treatment, the only hope is to have access, as soon as possible, to new medicines being developed or close to being authorised whenever they are sufficiently promising. This is justification for resorting to compassionate use.

The early use of these medicines is not without risk: undesirable effects are not completely known and their frequency is poorly evaluated. But for terminally ill patients, more so than for others, it is the balance between potential benefits and the risks of the treatment that must be taken into account, not just the isolated risks.

In France, the medical products agency has shown that compassionate use of new anti HIV drugs, (delivered through Temporary Authorised Use or ATU) has saved lives before being granted marketing authorisation.

But the European regulatory framework on this subject is at the moment insufficient: the compassionate use of some medicines to combat situations of terminal illness is often restrictive and tardy. Let us take for example the T-20, a medicine developed by the firm Roche representing some hope for those terminally ill with AIDS. In spite of the great need, Roche chose to allow compassionate use at a very late date and then limited its use to only 450 patients throughout the world, when thousands more still need the medicine as a matter of urgency. Those patients unable to enter into the clinical trials are still waiting for this medicine even though no medical reason has been given to justify the restriction of access to T-20.

At the moment, compassionate use programmes depend solely on the good will of pharmaceutical companies. Health authorities have not taken the necessary steps to get drug companies to facilitate access of medicines that could be the answer to public health emergencies.

The proposed mechanism for compassionate access, as described in Article 73 of the European Regulation, does not provide for constraint or incentive towards the firms. Therefore it will not allow a better response to the needs of the patients who do not have access to medicines, until these firms find it to their own interest.