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

12 Special Solidarity Fund KCE Reports 133
Compassionate use / medical urgency programs
Compassionate use of drugs concerns the treatment of patients with drugs that are not
yet reimbursed or available in Belgium. 6
Compassionate use can be applied in two cases:
• Programmes of compassionate use: making available, for compassionate
reasons, of a medicinal product that can qualify for the centralized
procedure to a group of patients with a chronically or seriously
debilitating disease or whose disease is considered to be life-threatening,
and who cannot be treated satisfactorily by an authorised medicinal
product. The medicinal product concerned must either be the subject of
an application for a Marketing Authorisation in accordance with Article 6
of the European Regulation laying down Community procedures for the
authorisation and supervision of medicinal products for human and
veterinary use and establishing a European Medicines Agency 7 or must be
undergoing clinical trials. 8
• The Medical Need Programmes: making available a medicinal product to a
group of patients with a chronically or seriously debilitating disease or
whose disease is considered to be life-threatening, and who cannot be
treated satisfactorily by an authorised medicinal product. The medicinal
product concerned must have a Marketing Authorisation but
o either the given indication has not been authorised yet, or
o although authorised, the medicinal product is not yet available on the
market in this indication. 8
The essential difference between the two programmes is that Compassionate Use
concerns medicinal products which do not yet have obtained a Marketing Authorisation
in Belgium, unlike the Medical Need Programme, which concerns medicinal products
which have a Marketing Authorisation in Belgium for a given indication. In order for a
medicinal product to be considered for compassionate use, the market authorization
holder will have to introduce a demand that will be reviewed and approved by one of
the Belgian ethics committees. The compassionate use treatment will be prescribed by a
physician: the hospital can require approval for the individual patient by the local ethics
committee. In both cases the pharmaceutical companies bear the costs for the medicinal
products.
Keypoints: The SSF in the Belgian health care system
• The Belgian health care system is organized at federal and regional level.
The federal level is responsible for the regulation and financing of the
compulsory health insurance that is organized by the NIHDI.
• The SSF is a part of the NIHDI as a complement to the compulsory health
insurance system.
• Reimbursement of orphan drugs is organized separately from the SSF but
interacts. The “College of medical directors on orphan drugs” mostly
decides on the individual right of the patient to the reimbursement of an
orphan drug.
• The compulsory health insurance has systems as the OMNIO status and the
MAB to protect vulnerable groups of patients with high medical costs.