Compassionate use - Medical need
The Royal Decree of 25 April 2014 (RD in French version) amending the Royal Decree of 14 December 2006 on medicinal products for human and veterinary use (RD in french version) was published in the Belgian Official Journal on 12 June 2014 and comes into effect on 1 July 2014.
All compassionate use programs and medical need programs submitted from that date must follow the procedure described in that text.
Each submitted file must be accompanied by payment of a fee. Each complete file (cfr. article 106, § 6 and article 108, § 6 of the aforementioned Royal Decree of 14th December 2006) requires payment of € 4728,95 and each modification requires payment of € 4728,95. Payment must be made on the following account : 679-0001514-59
Contact details of the bank :
Chaussée d'Anvers 59
B-1100, Bruxelles (Belgique)
SWIFT code : PCHQBEBB
IBAN code : BE84 6790 0015 1459
Please mention “UMN” on the bank statement followed by the name of the products object of the application. This also applies for an amendment with the additional mention “amendment”.
The annual or semi-annual report of suspected unexpected serious adverse reactions (cfr. article 106, § 5, paragraph 3 and article 108, §5, paragraph 3 of the aforementioned Royal Decree of 14th December 2006) requires payment of € 2940,67 which is to be paid to the FAMHP bank account mentioned here above with the communication “UMN” and the name of the product object of the application followed by the additional mention “ASR/SSR”.
The guidance describing, among others, the process to submit a compassionate use program and medical need program is here available. You will also find below, the appendices of this new guidance :
- CUP-UMN guidance
- Annex I : Royal Decree of 25 April 2014 amending the Royal Decree of 14 December 2006 (french and dutch version)
- Annex II : Application form to request a Compassionate Use Program or a Medical Need Program
- Annex III : Template of Compassionate Use Program protocol
- Annex IV : Summarized information for publication (EN-FR-NL)
- Annex V : Labeling
- Annex VI : Template of Medical Need Program protocol
- Annex VII : CUP Physician Declaration
- Annex VIII : MNP Physician Declaration
Please submit any specific questions via e-mail at firstname.lastname@example.org.
A list of frequently asked questions regarding the application for UMN can be found below and will be regularly updated :
|Commercial name||Active substance||Indication|
|Raxone®||Idebenone||Patients with Duchenne Muscular Dystrophy (DMD) who completed DELOS study (SNT-III-003/ EudraCT
|Lenvima®||Lenvatinib||Radioiodine-refractory differentiated thyroid cancer.|
|Mylotarg ®||Gemtuzumab Ozogamicin||CD33-positive relapsed or refractory acute myeloid leukemia (AML) or CD33-positive relapsed or refractory acute promyelocytic leukemia (APL).|
|Signifor LAR ®||Pasireotide||Patients with acromegaly who are inadequately controlled with 1st generation somatostatin analogues|
|Raxone ®||Idebenone||Leber’s hereditary optic neuropathy (LHON)|
|Blincyto®||Blinatumomab||Adults with Philadelphia chromosome negative relapsed or refractory B-precursor acute lymphoblastic leukaemia (ALL)|
|Blincyto®||Blinatumomab||Adults with B-precursor acute lymphoblastic leukemia (ALL) in complete hematological remission defined as less than or equal to 5% blasts in the bone marrow after at least three intense chemotherapy blocks and presence of minimal residual disease (MRD) at a level≥10-4|
|Vimpat®||Lacosamide||Partial-onset or generalized tonic clonic seizures in patients ≥ 16 years of age coming from the SP0994 clinical trial|
|Metycor®||Metyparone||Endogenous Cushing’s syndrome in patients who have completed the study extension period of the PROMPT clinical trial with metyrapone|
|Adjunctive treatment of partial-onset seizures with or without secondary generalization in patients with epilipsy aged 16 years and older|
|Halaven®||Eribulin||Treatment of adult patients with unresectable liposarcoma who have received prior anthracycline containing therapy (unless unsuitable) for advanced or metastatic disease.|
|Ilaris®||Canakinumab||Hereditary periodic fever|
|Ibrance®||palbociclib||patients suffering from advanced/metastatic breast cancer hormone receptor (HR)‑positive, human epidermal growth factor receptor 2 (HER2)‑negative with endocrine therapy|
|COR-003 (2S,4R-ketoconazole)®||COR-003 (2S,4R-ketoconazole)||Endogenous Cushing’s Syndrome|
|RoActemra®||tocilizumab||Giant cell arteritis (GCA or temporal arteritis)|
|Wakix®||Pitolisant||Second-line treatment of adult patients with narcolepsy with or without cataplexy|
|Orkambi®||lumacaftor 100mg / ivacaftor 125mg||Treatment of cystic fibrosis (CF) in patients 6 through 11 years of age who are homozygous for the F508del mutation in the CFTR gene.|
|Nusinersen®||nusinersen||Infantile-onset (Type I) Spinal Muscular Atrophy (SMA)|
|Besponsa®||inotuzumab ozogamicin||relapsed or refractory CD22-positive B-Cell precursor acute lymphoblastic leukemia (ALL) in adult patients|
|Tecentriq®||atezolizumab||locally advanced or metastatic urothelial carcinoma in adult, previously untreated patients who are considered cisplatin ineligible|
Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, inambulatory patients aged 5 years and older for patients who have been treated with this medicinal product as part of the clinical trials (studies 019 and 020E) that are currently in the close-out process.
Primary progressive multiple sclerosis.
|Xalkori®||Crizotinib||ROS1-positive non-small cell lung cancer (NSCLC)|
|Rydapt®||Midostaurin (PKC412)||Newly diagnosed FLT3 mutated acute myeloid leukemia patients eligible for standard induction and consolidation chemotherapy.|
|Bavencio||Avelumab||Treatment of adult patients with metastatic Merkel cell carcinoma whose disease has progressed after receiving at least one prior chemotherapy or who are ineligible for chemotherapy|
long-term enzyme replacement therapy in patients with - hypophosphatasia (HPP) in whom the first symptoms presented before the age of 18, to treat the bone manifestations of the disease.
|Revlimid®||Lenalidomide||Diffuse large B cell lymphoma in patients who already received at least two prior treatment lines|
|Alectinib||First-line treatment of adult patients with anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC)|
patients who suffer from pulmonary hypertension associated with COPD and who participated in the PULSE-COPD-007 study
Treatment of newly diagnosed with high risk Metastatic hormone sensitive prostate cancer (mHSPC) in adult men in combination with androgen deprivation therapy (ADT) and prednisone or prednisolone.
|In combination with alltrans retinoic acid (ATRA) for the first‐line treatment of low or intermediate risk acute promyelocytic leukemia (APL)|