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 € 4.635,82 and each modification requires payment of € 4.635,82. Payment must be made on the following account : 679-0001514-59

Contact details of the bank :

Poste financière
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 € 2.882,76 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 umn@afmps-fagg.be.

FAQ

A list of frequently asked questions regarding the application for UMN can be found below and will be regularly updated :

FAQ

Authorised programs

Commercial name Active substance Indication
Raxone® Idebenone Patients with Duchenne Muscular Dystrophy (DMD) who completed DELOS study (SNT-III-003/ EudraCT 2009-012037-30)
Ofev® Nintedanib Idiopathic pulmonary fibrosis (IPF) in adult patients
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 level10-4
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
Briviact®
Brivaracetam
Adjunctive treatment of partial-onset seizures with or without secondary generalization in patients with epilipsy aged 16 years and older
Midostaurin® Midostaurin (PKC412) soft gelatin capsules of 25 mg Treatment of adult, newly diagnosed FLT3 mutated acute myeloid leukemia patients eligible for standard induction and consolidation chemotherapy.
Halaven® Eribulin Treatment of adult patients with unresectable liposarcoma who have received prior anthracycline containing therapy (unless unsuitable) for advanced or metastatic disease.
Humira® Adalimumab
Treatment of non-infectious intermediate, posterior and pan-uveitis
Stelara® Ustekinumab Treatment of moderate to severe active Crohn’s disease who have failed TNFα antagonists (infliximab/infliximab biosimilar, adalimumab) and the integrin inhibitor vedolizumab, or who are intolerant to, or have a contraindication to these treatments.
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
Alprolix® Eftrenacog alfa Treatment and prophylaxis of bleedings in hemophilia B patients (deficiency= Factor IX coagulation factor)
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) 
Cabometyx® cabozatinib

advanced renal cell carcinoma in adults following prior vascular endothelial growth factor (VEGF)- targeted therapy

Venclyxto® venetoclax treatment of chronic lymphocytic leukaemia in adult patients who are unsuitable for or have failed other treatments.
Besponsa® inotuzumab ozogamicin relapsed or refractory CD22-positive B-Cell precursor acute lymphoblastic leukemia (ALL) in adult patients

On hold programs

Closed programs

Last updated on 31/07/2017