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.
The FAMHP will request a contribution for each file submitted. The FAMHP requests no longer to make advance payments, but to wait for the invoice (or invitation to pay) with structured notice for the payment. More information about the new method can be found here. The amount of the contribution for Unmet Medical Need files can be found here. Please indicate in the cover letter to whom the invoice should be sent.
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
- Annex X : Application form re-evaluation
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 |
| 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 |
| 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. |
| 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. |
|
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 | |
| Erleada® | 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 |
| 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. |
| 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. |
| Axicabtagene ciloleucel | Treatment of aggressive conditions in adults with primary mediastinal B-cell lymphoma (PMBCL) affecting your lymph tissue (part of the immune system) that affects a type of white blood cell called B lymphocytes and other organs in your body. | |
| Myalepta | Metreleptine | Treatment of patients with generalized lipodystrophy or patients with partial lipodystrophy and uncontrollable metabolic disorders. |
| 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 |
| 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. |
| Tecentriq | Atezolizumab | Treatment of first line for adult patients with locally advanced or metastatic, positive PD-L1, triple negative breast cancer, in combination with nab-paclitaxel. |
| Siponimod | BAF312 | Treatment for adult patients with Secondary Progressive Multiple Sclerosis (SPMS). |
| Xarelto | Rivaroxaban |
Co-administered with acetylsalicylic acid (ASA), for the treatment of patients with coronary artery disease (CAD) at high risk of ischaemic events |
| Fycompa® | Perampanel | Partial onset seizures for patients who have previously participated in Study 311 and are aged <12 years. |
| Keytruda® | Pembrolizumab | In combination with platinum and 5-fluorouracil (5-FU) chemotherapy for the first-line treatment of recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) in adults with a PDL-1 CPS score=0. |
| Erleada® | apalutamide |
in combination with androgen deprivation therapy (ADT) for the treatment of adult patients with metastatic hormone-sensitive prostate cancer (mHSPC). |
| Adcetris |
Brentuximab vedotin |
In combination with cyclophosphamide, doxorubicin and prednisone (CHP) for the treatment of adult patients with previously untreated CD30-positive peripheral T-cell lymphoma (PTCL). |
| Ipatasertib (MO40153) | Ipatasertib (MO40153) | in combination with paclitaxel in PIK3CA/AKT1/PTEN altered, locally advanced or metastatic, triple-negative breast cancer or hormone-receptor-positive, HER2-negative breast cancer. |
| Aubagio® | teriflunomide | Relapsing Remitting Multiple Sclerosis in the pediatric population. |
| Entrectinib | Entrectinib | treatment for adult patients, with NTRK fusion-positive, locally advanced or metastatic solid tumors. |
| RoActemra | tocilizumab | treatment of patients 50 years or older with active Giant Cell Arteritis who cannot be adequately treated with the current standard GC therapy. |
| Kadcyla | trastuzumab emtansine | as a single agent for the adjuvant treatment of adult patients with HER2 positive early breast cancer who have residual invasive disease, in the breast and/or lymph nodes, after neoadjuvant taxane-based and HER2-targeted therapy. |
| Givlaari® | givosiran | treatment of acute hepatic porphyria (AHP) in adults and adolescents (≥12 years of age) |
| trifluridine/tipiracil | monotherapy for the treatment of adult patients with metastatic gastric cancer including adenocarcinoma of the gastroesophageal junction, who have been previously treated with at least two prior systemic treatment regimens for advanced disease. | |
| fremanezumab | Prophylaxis of migraine in adult patients who suffer from at least 8 monthly migraine days and who previously failed on at least 2 preventive treatment classes and cannot be satisfactorily treated with the other approved and commercially available alternative treatments, in accordance with clinical guidelines, because of efficacy and/or safety issues. | |
| Tecentriq® | atezolizumab | in combination with bevacizumab, is indicated for the first-line treatment of adult patients with advanced or unresectable hepatocellular carcinoma (HCC) who have received no prior systemic therapy |
| Spravato® | Esketamine |
Indicatie (EN): In combination with a SSRI or SNRI, for treatment-resistant depression (Major Depressive Disorder) in adults who have not responded to at least two different treatments with antidepressants in the current moderate to severe depressive episode |
| Cyramza® | ramucirumab | treatment of adult patients with advanced or unresectable hepatocellular carcinoma who have a serum alpha fetoprotein (AFP) of ≥ 400 ng/ml and who have been previously treated with systemic therapy. |
| Risdiplam® | risdiplam | treatment of patients who suffer from Type 1 SMA |
| Zejula® | niraparib |
maintenance treatment of adult female patients with advanced high-grade ovarian, fallopian tube, or primary peritoneal cancer who are in response (partial or complete) following completion of firstline platinum-based chemotherapy |
| VX-445/TEZ/IVA | elexacaftor / tezacaftor / ivacaftor | Cystic fibrosis (CF) patients in critical need who are 12 years of age and older, heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes) |