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 email@example.com.
A list of frequently asked questions regarding the application for UMN can be found below and will be regularly updated :
|Commercial name||Active substance||Indication|
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.
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|
patients who suffer from pulmonary hypertension associated with COPD and who participated in the PULSE-COPD-007 study
|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.|
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.|
|Siponimod||BAF312||Treatment for adult patients with Secondary Progressive Multiple Sclerosis (SPMS).|
|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.|
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
|Risdiplam®||risdiplam||treatment of patients who suffer from Type 1 SMA|
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
|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.|
treatment of patients with active Crohn's Disease who have no other suitable treatment option and are not eligible to participate in a clinical study for their serious or life-threatening condition.
|Remimazolam||Remimazolam||Sedation of Covid-19 patients under artificial ventilation in the Intensive Care Unit.|
|Remimazolam||Remimazolam||Induction and Maintenance of General Anesthesia.|
|Duchenne Muscular Dystrophy in boys who are completing the VBP15-004 trial|
|Braftovi®||encorafenib||in combination with cetuximab, for the treatment of adult patients with metastatic colorectal carcinoma with a BRAFV600E mutation, who have received prior systemic therapy|
In combination with bendamustine and rituximab for the treatment of adult patients with relapsed/refractory diffuse large B‐cell lymphoma (DLBCL) who are not candidates for haematopoietic stem cell transplant.
|Entrectinib||Entrectinib||adult patients with locally advanced or metastatic Non-Small Cell Lung Cancer (NSCLC) with Central Nervous System (CNS) metastases, harbouring a ROS1 gene fusion, who have not received prior ROS1 inhibitor.|
|moderate to severe atopic dermatitis (AD), defined as EASI score ≥16 before start of treatment, in adult patients that qualify for systemic treatment|
|Nubeqa®||darolutamide||treatment of adult men with non-metastatic castration resistant prostate cancer (nmCRPC) who are at high risk of developing metastatic disease|
Treatment of adult patients with Primary Immune thrombocytopenia (ITP) within the first 12 months of diagnosis who are refractory to other treatments (e.g. corticosteroids, immunoglobulins)
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)
patients with metastatic castration resistant prostate cancer who have progressed on prior NHA and have a BRCA mutation (germline or somatic)
|risdiplam||risdiplam||Spinal Muscular Atrophy Type 2 for patients ≥2 months.|
Seizures associated with Lennox Gastaut Syndrome (LGS) for pediatric patients under 12 years of age.
|Pemazyre||pemigatinib||adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that is relapsed or refractory after at least one line of systemic therapy|
|Blenrep||belantamab mafodin||multiple myeloma in adult patients, who have received at least four prior therapies and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy|
|Forxiga®||dapagliflozin||patients with chronic kidney disease regardless the presence of Type 2 Diabetes|
|Venclyxto®||venetoclax||in combination with azacitidine for the treatment of newly diagnosed acute myeloid leukemia (AML) in adult patients who are ineligible for intensive chemotherapy and who have no other suitable treatment option and are not eligible to participate in a clinical study for their serious condition.|
|Retsevmo||Selpercatinib||adults with advanced RET fusion-positive non-small cell lung cancer (NSCLC) following prior systemic therapy|
|Retsevmo®||Selpercatinib||treatment of adults with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require
systemic treatment following prior treatment
|Opdivo and Yervoy||nivolumab and ipilimumab||Indication First line treatment of adult patients with unresectable malignant pleural mesothelioma.|
|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||patients (adults and 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.|
|Xtandi®||enzalutamide||treatment of adult men with metastatic hormone‐sensitive prostate cancer (mHSPC) in combination with androgen deprivation therapy (pending final marketing authorisation)|
|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.|
Treatment of paediatric and adult patients with lysosomal acid lipase deficiency (LAL-D) who participated to the open label phase 2 study (LAL-C06).
|Rinvoq®||upadacitinib||adult patients with severe Ankylosing Spondylitis who have no other suitable treatment option and are not eligible to participate in a clinical study for their serious condition|
|Norditropin®||somatotropin||growth failure in children with achondroplasia/hypochondroplasia|
|Inrebic®||fedratinib||disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential|
|Opdivo®||nivolumab||first line treatment of adult patients with HER2 negative advanced or metastatic gastric, gastro oesophageal junction or oesophageal adenocarcinoma whose tumours express PD-L1 with a CPS ≥ 5 in combination with fluoropyrimidine- and platinum-based chemotherapy|