Optimalisatie van de werkingsprocessen van het Bijzonder ... - KCE

78 Special Solidarity Fund KCE Reports 133
7.3.2 Access to medication in special situations
Next to the creation of the reference centers, at national level new legislation has been
passed to improve access to medication in ‘special situations’. 42
This ruling provides access to investigational drugs to patients who have no successful
treatment and suffer from a serious disease or have a life-threatening situation. The
authorization is given by the Spanish Agency of Medicines (AEMPS).
7.3.2.1 Use of medication in pre-market authorization stage
The Spanish Agency of Medicines and Health Products (AEMPS) may authorize the
prescription and the use of drugs that have not yet been accepted and authorized in
Spain, for individual patients that suffer from a chronic or a serious disease or are in a
life threatening situation without any satisfactory therapeutic alternative available. It
concerns patients which are not part of a clinical trial and who are in a clinical situation
that cannot wait till the investigation on the new treatment will be completed. These
drugs have to be subject of a procedure for acceptance or be part of a clinical trial
procedure (but meant for patients that are no part of the clinical trial). Access to these
drugs may be authorized individually for a patient (compassionate use), or relying on a
temporary authorization for use for a group of patients. If an individual authorization is
asked, the hospital where the patient is treated will have to enter the request. The
AEMPS has to decide on the petition within a period of 10 days. If the decision is
negative the hospital has a period of 10 days to add information and argue the case.
Next to the individual authorization, a temporary authorization procedure has been
foreseen for drugs that are not accepted and authorized yet and that are meant for a
(homogeneous) group of patients. The pharmaceutical company (or the promoter of a
clinical trial) can ask to obtain such authorization if the drug is subject to an acceptance
procedure that has not been accomplished yet or if the drug is subject to a clinical trial
procedure. The authorization can be given for the use of this drug to a specified group
of patients (that, if a clinical trial is running, are not included in this trial). The hospital
where the drug will be administered has to guarantee the patient meets the conditions
that have been specified by the AEMPS. Prescribing and using these drugs is restricted
to the specialized care level (hospital care but also ambulatory treatments in hospitals).
More detailed information on the evaluation criteria by the AEMPS could not be
obtained.
7.3.2.2 Off label use
The new regulation also includes the possibility for the use of drugs for different
indications as those for which they have been authorized (off label use). The
authorization for off label use is not an individual authorization (not for one specific
patient) but for homogeneous groups of patients. The prescribing doctor will have to
justify the off label use in the individual medical file of the patient but does not have to
ask for an individual authorization from the AEMPS. The AEMPS may issue
recommendations to be taken into account in the therapeutic-care protocols developed
by health centers. This may be especially relevant in therapeutic areas in which research
activity is intense and the pace of evolution of scientific knowledge may precede the
steps necessary to incorporate such changes in the technical file of the drug. It allows
the use of drugs for conditions that have not been established in the authorization of
the medicine, often by absence of commercial interest for the pharmaceutical
companies to accomplish the studies necessary to obtain the authorization of the
AEMPS.