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 :
Authorised programs
| Commercial name | Active substance | Indication |
| Raxone® | Idebenone | Patients with Duchenne Muscular Dystrophy (DMD) who completed DELOS study (SNT-III-003/ EudraCT |
| 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 level≥10-4 |
| 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 generalisation in adults, adolescents and children from 4 years of age with epilepsy |
| 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 |
|
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. |
|
Velmanase alfa |
Treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis | |
| 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. |
| 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. |
| 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. |
| Fingolimod |
Relapsing multiple sclerosis (RMS) in for pediatric patients aged 10 to less than 18 years old of 40 kg or above. |
|
| Hemlibra | Emicizumab | Routine prophylaxis of bleeding episodes in patients with hemophilia A with factor VIII inhibitors. Hemlibra can be used in all age groups. |
| 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. |
| 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. |
| Lorlatinib | Lorlatinib (PF-06463922) | Metastatic NSCLC (Stage IV, AJCC v7.0) that carries an ALK rearrangement or that carries a ROS1 rearrangement |
| Yervoy® | Ipilimumab | Treatment-Naïve Intermediate and Poor Risk Patients with Advanced or Metastatic Renal Cell Carcinoma in combination with Opdivo® (nivolumab) |
| Entresto | sacubitril / valsartan | heart failure with preserved fraction (HFpEF) for patients who participated in study CLCZ696D2301 (PARAGON-HF) |
| Onpattro | Patisiran-LNP | hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy |
| Vitrakvi | Larotrectinib | Treatment of adult and paediatric patients with locally advanced or metastatic solid tumors with a Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusion that cannot be treated satisfactorily with the available treatment options |
| Esbriet | pirfenidone | treatment of patients with advanced idiopathic pulmonary fibrosis and risk of group 3 pulmonary hypertension (i.e. pulmonary hypertension occurring secondary to lung disease and/or hypoxia) and exiting study MA29957 |
| Fremanezumab (TEV-48125) | Fremanezumab (TEV-48125) | Patients with episodic migraine or chronic migraine who have successfully completed (per protocol) Study TV48125‐CNS‐30051 or the treatment phase of Study TV48125‐CNS‐30068. |
| Humira® | adalimumab | moderately to severely active Ulcerative Colitis in paediatric patients. |
| Opdivo | nivolumab | Adult patients with unresectable Malignant Pleural Mesothelioma who have progressed after at least one line of treatment including pemetrexed in combination with platinum agent. |