Compassionate use programs can be requested from the FAMHP for medicines that do not yet have a marketing authorisation but fall into either of the following categories: (i) an application for marketing authorisation has been submitted; or (ii) a clinical trial for the relevant indication is ongoing. These medicines are only used in cases where a patient has a chronic, disabling or life-threatening disease that cannot be adequately treated with a licensed and reimbursed medicine available on the Belgian market for that condition.
Medical need programs can be requested from the FAMHP for medicines that are already authorised for another indication. For medicines authorised in Belgium, this can be done in cases where a patient has a chronic disease, a disabling disease or a life-threatening disease that cannot be adequately treated by a product authorised and reimbursed in Belgium for this indication.
Additional conditions are:
- a demand to obtain the MNP indication is being evaluated in a MAA;
- or the MA has been granted for the MNP indication but the medicinal product is not yet marketed with the MNP indication
- or clinical evidence has been generated in (ongoing) clinical trials for the MNP indication.
In both scenarios, it is the responsibility of the pharmaceutical firm to submit the program to the FAMHP (Federal Agency for Medicines and Health Products). The FAMHP may choose to seek advice from an Ethics Committee as part of their evaluation process. Therefore, there is no requirement for the treating physician to submit the program to EC Research UZ Leuven, nor does the program need to be notified via the registration form.
Exceptionally, in urgent cases, a medicine that is not on the market and cannot be imported can be used outside the framework of a CUP. In this case, it concerns an urgent medical need. his exception is intended for a limited number of patients. If one wishes to treat a larger group of patients, a CUP programme should be requested.
This exception is only possible for a patient in whom, without appropriate treatment, death can be expected in the short term or the risk of consequences due to the absence of treatment will be greater than the risk of consequences due to starting treatment. The prescribing physician takes into account this procedure and the need for its timely initiation.
Additionally the following conditions should be met:
- The medicinal product in question is not a drug used in a CUP, in a clinical trial (or if the patient could not be enrolled within such a CUP or clinical trial) or/and is not a drug for which a registration or a marketing authorization is not required
- No submission can be made for a CUP, unless such an application has been made or if you have the intention to submit such a request.
- The patient cannot be treated with a marketed medicinal product, a product under hospital exemption or with a magisterial preparation
- It is impossible to import a marketed medicinal product with the same qualitative and quantitative composition of active drug substance and the same pharmaceutical form (as stated in article 105 of the Royal Decree of 14/12/2006
The treating physician should send a notification of the application of art. 107/1 RD 14/12/2006 to EC Research (ec@uzleuven.be) and pharmacy (apo_studies@uzleuven.be) for each patient he wishes to treat according to the article described above. One should not wait for confirmation of this notification to start treatment.
EC Research will assign an S number to be used in subsequent communication. If necessary, the Clinical Trial Center (CTC) lawyer will assist the physician with the contract.
For further details, additional information can be accessed via the links provided below.
Compile the file for urgent medical need
Mandatory
The following documents are mandatory for a valid submission:
- Cover letter
- Information and consent form (see ICF template)
Optional
If relevant for your study, the following can also be submitted: :
- Miscellaneous
Submit application
Please submit your application to ec@uzleuven.be.
More information can be consulted on the muzlidoc procedure https://wiki.uz.kuleuven.ac.be/pages/releaseview.action?pageId=77463528.