On 13 December 2020, Belgium amended its rules regarding compassionate use and medical need programs to confirm that authorized programs can continue to operate after the marketing authorization for the concerned product has been granted but while the decision on reimbursement is still pending.
The Law of 25 March 1964 (“Medicines Law”) regulates the use of medicinal products that have not (yet) received a marketing authorization (“compassionate use”, “CU”) and for off-label use (“medical need”, “MNP”). The Belgian Federal Agency for Medicines and Health Products (“FAMHP”) must grant an authorization to allow the use of medicinal products in a compassionate or medical need context
The grant of authorization for both CU and MN is subject to the following requirements, as amended:
- the patient suffers from a chronically or seriously debilitating disease or disease considered to be life‐threatening,
- such disease cannot be satisfactorily treated by a medicinal product “that is reimbursed” and that is authorized and marketed, and
- for compassionate use programs:
- a market authorization application for the medicinal product has been submitted, or
- the medicinal product is subject to ongoing clinical trials,
- for medical need programs:
- a market authorization application has been submitted for the indication for which the medicinal product will be used,
- the market authorization has been granted for this indication but the medicinal product is not yet on the market, or
- the medicinal product is subject to clinical trials that are ongoing or have demonstrated that the use of the medicinal product is relevant to treat the disease in question.
The Compassionate Use Law amended the second condition by adding the reference to reimbursement.
The amendment ensures that it is possible for a company to (continue to) operate a CU or MN program during the period between authorization and reimbursement. The preparatory works give the example of a terminal cancer patient who cannot afford to cover the cost of a treatment that has been authorized, but whose reimbursement status is pending.
The Compassionate Use Law does not amend the other requirements.