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 € 4.550,79 and each modification requires payment of € 4.550,79. 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 € 2.829,88 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)
Revolade® Eltrombopag Thrombocytopenia in patients with severe aplastic anemia
Cobimetinib® Cobimetinib In combination with Zelboraf® (vemurafenib) in patients with unresectabel or metastatic BRAFV600 mutation-positive melanoma
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)  
Humira ® Adalimumab Active moderate to severe Hidradenitis Suppurativa (HS) in adult patients with an inadequate response to conventional systemic HS therapy
Opdivo® Nivolumab locally advanced and metastatic Non-Small-Cell Lung Cancer (NSCLC) in patients who have documented progression on or after prior chemotherapy
Pixuvri® Pixantrone monotherapy for the 3rd or 4th line treatment of adult patients with multiply relapsed or refractory aggressive NonHodgkin Bcell Lymphomas (NHL) (DLBCL and transformed indolent lymphoma)
Repatha® Evolocumab Severe uncontrolled familial hypercholesterolemia (FH), despite treatment with a maximum tolerated dose of a statin in combination with ezetimibe, or with ezetimibe without a statin in case of statin intolerance or contraindication
Keytruda® Pembrolizumab – MK 3475 Unresectable or metastatic melanoma in patients who are refractory to treatment with ipilimumab
Blincyto® Blinatumumab Adults with Philadelphia chromosome negative relapsed or refractory B-precursor acute lymphoblastic leukaemia (ALL)
Blincyto® Blinatumumab 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
Humira® Adalimumab Active enthesitis-related arthritis in patients, 6 years of age and older, who have had an inadequate response to, or who are intolerant to, conventional therapy
Keytruda® Pembrolizumab – MK 3475 Advanced (unresectable or metastatic) melanoma in first line or after BRAF inhibitor 
Zykadia® Ceritinib ALK positive NSCLC patients that have received prior therapy with crizotinib
Orkambi® lumacaftor/ivacaftor Cystic fibrosis in patients 12yrs and older who are homozygous for the F508del CFTR mutation
Kyprolis® carfilzomib

in combination with lenalidomide and dexamethasone (KRd) for the treatment of adult patients with relapsed multiple myeloma who did receive at least 1 prior therapy

Kyprolis® carfilzomib in combination with dexamethasone (Kd) for the treatment of adult patients with relapsed multiple myeloma who did receive 3 or more prior therapies
Opdivo® Nivolumab Medical Need Program with Opdivo (nivolumab) for the treatment of anti-PD-1-treatment naïve patients with advanced renal clear-cell carcinoma who have progressed after or failed at least one prior treatment with an anti-angiogenic agent
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
 

On hold programs

Closed programs

Last updated on 17/05/2016