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: this procedure must be followed exactly
- 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 |
| 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. |
|
| iNOmax® | iNOmax |
patients who suffer from pulmonary hypertension associated with COPD and who participated in the PULSE-COPD-007 study |
|
Velmanase alfa |
Treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis | |
| 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. |
| 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. |
| 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. |
| 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 |
| Humira® | adalimumab | moderately to severely active Ulcerative Colitis in paediatric patients of 2 to 5 years old. |
| esketamine | Esketamine | ESK is intended 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). ESK must be given in combination with an SSRI or SNRI. |
| Vamorolone |
Delta-1,4,9(11)-pregnatriene-17,21-dihydroxy-16-alpha-methyl-3,20-dione |
Duchenne Muscular Dystrophy in boys who are completing the VBP15-004 trial |
| Lumasiran | lumasiran |
Treatment of primary hyperoxaluria type 1 (PH1) in patients who have reached at least 37 weeks estimated gestational age (full‐term infant) at consent (or assent) |
| Perampanel |
Seizures associated with Lennox Gastaut Syndrome (LGS) for pediatric patients under 12 years of age. |
|
| Tolvaptan | Tolvaptan | Treatment of Children and Adolescent Patients until they reach 18 years of age with Autosomal Dominant Polycystic Kidney Disease Following Successful Participation and Completion in the 156-12-298 Clinical Trial |
| Verquvo® | vericiguat | Adult patients with symptomatic chronic heart failure (New York Heart Association (NYHA) Class II-III-IV) with a reduced ejection fraction who are stabilized after a previous decompensation event requiring IV therapy. |
| Rinvoq® | upadacitinib | the treatment of adolescents ≥ 12 years old with severe atopic dermatitis who have no other suitable treatment option and are not eligible to participate in a clinical study |
| Selinexor | selinexor | Adult patients with relapsed or refractory multiple myeloma (RRMM), who have have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy. |
| Soliris® | eculizumab | patients with refractory generalized myasthenia gravis (rgMG) who completed the study ECU-MG-302 (Open label extension) and, in the opinion and the clinical judgement of the treating physician would continue to benefit from a treatment with Eculizumab (Soliris®) which is not yet commercially available for this indication in Belgium. |
| Rybrevant | amivantamab | treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who have epidermal growth factor receptor (EGFR) Exon 20 insertion (Exon20ins), and whose disease has progressed during or after current standard of care platinum-based chemotherapy. |
| Gilenya® | fingolimod | treatment of patients with relapsing-remitting multiple sclerosis (RRMS) who are included in medical need program CFTY720DBE02_C_IPS |
| Enhertu® | trastuzumab deruxtecan | adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens. |
| Kanuma | sebelipase alfa |
Treatment of paediatric and adult patients with lysosomal acid lipase deficiency (LAL-D) who participated to the open label phase 2 study (LAL-C06). |
| Norditropin® | somatotropin | growth failure in children with achondroplasia/hypochondroplasia |
| Inrebic® | fedratinib | treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis have been treated with ruxolitinib. |
| Selumetinib | Selumetinib | neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas in paediatric patients with neurofibromatosis type 1 (NF1) aged ≥ 3 years and ≤ 18 years |
| Tebentafusp | tebentafusp | HLA-A*02:01 positive patients with metastatic uveal melanoma |
| Nexviadyme | avalglucosidase alfa | treatment of adult Pompe disease patients who have participated and exit the clinical studies COMET (EFC14028) or NEO-EXT (LTS13769) |
| Xenpozyme | olipudase alfa | acid sphingomyelinase deficiency for patients who have participated and exit the clinical studies DFI-12712-ASCEND or LTS-13632 |
| asciminib | asciminib | Patients with chronic myeloid leukemia (CML) in chronic phase previously treated with ≥ 2 TKIs |
| tucatinib (ONT-380) | in combination with trastuzumab and capecitabine for the treatment of adult patients with HER2 positive locally advanced or metastatic breast cancer who have received at least 2 prior anti HER2 treatment regimens. | |
| tepotinib | tepotinib | Treatment of adult patients with advanced non-small cell lung cancer harboring mesenchymal-epithelial transition factor gene METexon14 (METex14) skipping alterations. |
| Leriglitazone | Leriglitazone | Treatment of Friedreich’s Ataxia |
| Darzalex | daratumumab | in combination with cyclophosphamide, bortezomib and dexamethasone for the treatment of adult patients with newly diagnosed systemic amyloid light chain (AL) amyloidosis |
| Evrenzo® | roxadustat | Treatment of symptomatic anaemia of Chronic Kidney Disease in non-dialysis and dialysis patients, and where use of available treatment (IV iron or injectable ESAs) is not appropriate or is expected not to be appropriate, or patients who have a known contraindication for the currently available treatment or who do not tolerate the currently available treatment, or are expected not to tolerate this product |
| Jardiance® | empagliflozin | treatment of adult patients with heart failure with preserved ejection fraction (ejection fraction >40%) |
| Opdivo + Yervoy | nivolumab + ipilimumab | Treatment of adult patients with unresectable malignant pleural mesothelioma that have progressed after at least one line of treatment, including platinumbased doublet chemotherapy. |
| Trodelvy® | sacituzumab govitecan | treatment in monotherapy of adult patients with unresectable, locally advanced or metastatic triple-negative breast cancer (mTNBC) who have received two or more prior systemic therapies, including at least one of them for advanced disease and with no adequate treatment options |
| LUMYKRAS | sotorasib | Treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with KRAS G12C mutation and who have progressed after at least one prior line of systemic therapy |
| Kerendia | finerenone | Adult patients with chronic kidney disease (CKD) (stage 3 & 4 with albuminuria) associated with type 2 diabetes (T2D) who cannot be satisfactorily treated with the standard of care. |
| Aspaveli® | pegcetacoplan | Treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic despite treatment with a C5 inhibitor for at least 3 months. |
| Kaftrio | ELX/TEZ/IVA and IVA alone | Cystic fibrosis (CF) in patients from 6 to 11 years old who are homozygous for F508del (F/F genotype) or heterozygous for F508del and a minimal function (F/MF genotype) mutation. |
| Verzenios | abemaciclib | in combination with Endocrine Therapy as the adjuvant treatment of adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, nodepositive early breast cancer at high risk of recurrence |
| Pritelivir | Pritelivir | treatment of acyclovir-resistant mucocutaneous HSV (Herpes Simplex Virus) infections in immunocompromised patient |
| Adtralza | tralokinumab | severe atopic dermatitis in adolescent patients (12- <18 years) who are candidates for systemic treatment (including patients coming from the Open Label Extension Study ECZTEND – LP0162-1337 with EudraCT reference 2018-000746-19) |
| teclistamab | teclistamab | monotherapy, for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior lines of therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and who have exhausted all commercially approved and clinically appropriate treatment options, are ineligible for a clinical trial and have evidence of disease progression after the last therapy |
| Padcev | enfortumab vedotin | locally advanced or metastatic urothelial cancer in patients who have previously received a platinum-containing chemotherapy and a programmed death receptor 1 (PD-1) or programmed death ligand 1 (PD-L1) inhibitor |
| Avacopan | Avacopan | in combination with a rituximab or cyclophosphamide regimen, is indicated for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) |
| Imfinzi | durvalumab | First-line Treatment for Patients with Locally Advanced or Metastatic Biliary Tract Cancers in combination with Gemcitabine Plus Cisplatin Therapies |
| Bylvay | odevixibat | treatment of cholestatic liver disease in patients with Alagille Syndrome. |
| Imfinzi + tremelimumab | durvalumab + tremelimumab | first-line treatment in patients with unresectable hepatocellular carcinoma. |
| Nubeqa | darolutamide | treatment of adult men with metastatic hormone sensitive prostate cancer (mHSPC) in combination with ADT and docetaxel. |
| pitolisant | to improve wakefulness and reduce excessive daytime sleepiness (EDS) in adult patients with obstructive sleep apnoea (OSA) whose EDS has not been satisfactorily treated by, or who have not tolerated, OSA primary therapy, such as continuous positive airway pressure (CPAP) | |
| dupilumab | add-on therapy with intranasal corticosteroids for treatment of severe Chronic Rhinosinusitis with Nasal Polyps (CRSwNP) and comorbid asthma patients who have participated and exited the phase 4 clinical study EVEREST (LPS16747) | |
| Kapruvia | difelikefalin | treatment of moderate to severe chronic kidney disease-associated pruritus (CKD-aP) in adults undergoing hemodialysis (HD) |
| Minjuvi | Tafasitamab | the treatment of adult patients who have Relapsed or Refractory Diffuse Large B-Cell Lymphoma (R/R DLBCL) and who are not eligible for autologous stem cell transplantation (ASCT) |