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 18960,32€ for a compassionate use program (CUP) and 11561,45€ for a medical need program (MNP). Each modification and 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 2733,77€ for a compassionate use program and 2466,63€ for a medical need program.  Payment must be made on the FAMHP bank account mentioned here above with the communication “CUP” or “MNP” and the name of the product object of the application followed by the additional mention “ASR/SSR”.

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 “CUP” or “MNP”  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 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
  • Annex IX : e-submission through the CESP

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)
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 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
COR-003 (2S,4R-ketoconazole)® COR-003 (2S,4R-ketoconazole) Endogenous Cushing’s Syndrome
RoActemra® tocilizumab Giant cell arteritis (GCA or temporal arteritis)
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.
Besponsa® inotuzumab ozogamicin relapsed or refractory CD22-positive B-Cell precursor acute lymphoblastic leukemia (ALL) in adult patients
Translarna® ataluren

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.

RoACTEMRA® Tocilizumab Systemic sclerosis

Strensiq®

Asfotase alfa

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
iNOmax® iNOmax

patients who suffer from pulmonary hypertension associated with COPD and who participated in the PULSE-COPD-007 study

Alpelisib® Alpelisib In combination with fulvestrant or letrozole for postmenopausal women and men with endocrine resistant hormone receptor-positive (HR+) HER2-negative (HER2-) metastatic breast cancer, who have recurrence or progressed after at least 3 lines of systemic treatment for advanced or metastatic disease, and who harbor specific PIK3CA hotspot mutations.
Dupixent Dupilumab Treatment of moderate-to-severe atopic dermatitis in adult patients coming from the Open Label Extension study (OLE)R668-AD-1225 and who are candidates for systemic therapy

Velmanase alfa®

Velmanase alfa

Treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis
Dupixent® Dupilumab Treatment of severe atopic dermatitis in adult patients, uncontrolled with immunosuppressive systemic therapy during the last year.
Apalutamide® Apalutamide in combination with androgen deprivation therapy (ADT) for the treatment of adult patients having castration resistant prostate cancer with PSADT ≤ 10 months and no detected metastases using imaging as per physician’s clinical practice.
Gilteritinib (ASP2215) gilteritinib (ASP2215) treatment of patients with FMS-like tyrosine kinase 3 (FLT3)-mutated relapsed or refractory acute myeloid leukemia (AML) without access to comparable or alternative therapy
Tafinlar + Mekinist Dabrafenib  + Trametinib adjuvant treatment of high-risk BRAF V600 mutation-positive stage III melanoma after complete surgical resection.
Lynparza® Olaparib Treatment of patients with deleterious or suspected deleterious germline BRCA1 or BRCA2 mutation and who have previously been treated with chemotherapy in the neoadjuvant, adjuvant or metastatic setting for TNBC or HER2-/ HR+ breast cancer unsuitable for further anti-hormonal therapy.
Tecentriq® Atezolizumab

In combination with bevacizumab, paclitaxel and carboplatin, for the first-line treatment of adult patients with stage IV or recurrent metastatic non-squamous non-small cell lung cancer (NSCLC) and whose tumors express PD-L1 < 50% with no EGFR or ALK positive tumors.

Jakavi Ruxolitinib Treatment of patients with corticoid-refractory chronic graft vs. host disease after allogeneic stem cell transplantation, who cannot be adequately treated with commercially available alternatives.
Ocaliva® Obeticholic acid

Treatment of patients with Primary Biliary cholangitis who completed the Long-Term Safety Extension of the POISE phase 3 trial (747-301 / EudraCT 2011-004728-36)

Jakavi Ruxolitinib Treatment of patients with corticoid-refractory acute graft vs. host disease after allogeneic stem cell transplantation, who cannot be adequately treated with commercially available alternatives.
Privigen Human immunoglobuline Treatment of bleeds in patients with acquired von Willebrand syndrome.

Xeljanz®

Tofacitinib citrate Treatment of adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to either conventional therapy or a biologic agent.
Aimovig® erenumab Treatment of patients diagnosed with migraine, who are in need of prophylactic treatment and for whom no acceptable alternative prophylactic treatment is available.

Libtayo

Cemiplimab Treatment of patients with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for curative surgery or curative radiation.
Olorofim F901318 Treatment of invasive fungal infections due to Lomentospora prolificans, Scedosporium spp., Aspergillus spp., and other resistant fungi in patients lacking suitable alternative treatment.
Xarelto Rivaroxaban Co-administered with acetylsalicylic acid (ASA), for the prevention of atherothrombotic events in adult patients with coronary artery disease (CAD) or symptomatic peripheral artery disease (PAD) at high risk of ischaemic events.
Perjeta® pertuzumab

in combination with Herceptin® (trastuzumab) and chemotherapy for the adjuvant treatment of adult patients with HER2 positive early breast cancer at high risk of recurrence defined as lymph node positive disease.

Gilenya®

Fingolimod

Relapsing multiple sclerosis (RMS) in for pediatric patients aged 10 to less than 18 years old of 40 kg or above.

Keytruda® Pembrolizumab - MK 3475

In combination with chemotherapy (carboplatin + paclitaxel) for the first-line treatment of metastatic squamous non-small cell lung cancer (NSCLC) in adults whose tumours express PD-L1 with a tumour proportion score (TPS) between l%-49% or no PD-L1 (TPS <1%).

Hemlibra Emicizumab Routine prophylaxis of bleeding episodes in patients with hemophilia A with factor VIII inhibitors. Hemlibra can be used in all age groups.

Yescarta®

Axicabtagene ciloleucel Treatment of adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) and primary mediastinal large B cell lymphoma (PMBCL), after two or more lines of systemic therapy.
Myalepta Metreleptine Treatment of patients with generalized lipodystrophy or patients with partial lipodystrophy and uncontrollable metabolic disorders.

Tecentriq

Atezolizumab In combination with carboplatin and etoposide, for the first-line treatment of adult patients with extensive-stage small-cell lung cancer (ES-SCLC).
Esbriet Pirfenidon Treatment of adult patients with unclassifiable interstitial lung disease (uILD) and who exit trial MA39189.

On hold programs

Closed programs

Last updated on 19/02/2019