Overzicht van goedgekeurde compassionate use programma's
Het CBG kan toestemming verlenen voor het in de handel brengen van een (nog) niet geregistreerd geneesmiddel in schrijnende gevallen, ook wel compassionate use programma genaamd. Een fabrikant kan deze geneesmiddelen voor de patiëntengroep beschikbaar stellen. In het overzicht staan alle goedgekeurde compassionate use programma’s vermeld inclusief de indicatie en looptijd.
Overzicht goedgekeurde compassionate use programma's
-
2023
-
Company name: GlaxoSmithKline B.V.
Indication: Omjjara is indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and moderate to severe anaemia who have previously been treated with a JAK inhibitor (ruxulotinib or fedratinib) and for whom there are no satisfactory alternative treatments.
Date of approval: 10 november 2023 -
Company name: Janssen-Cilag B.V.
Indication: Treatment of adult patients with relapsed or refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and who have exhausted all available treatment options accessible as local standards of care and who have demonstrated disease progression on the last therapy.
Date of approval: 14 juli 2023
Date of cessation: 14 juli 2024 -
Company name: Roche Nederland B.V.
Indication: Patients who have R/R DLBCL NOS (incl. HGL), tFL or PMBCL who have exhausted all other viable treatment options and who fulfil the patient profile criteria.
Date of approval: 1 juni 2023
Date of cessation: 1 juni 2024
-
-
2022
-
Company name: Boehringer Ingelheim B.V.
Indication: Treatment of patients with generalized pustular psoriasis (GPP) presenting with a flare.
Date of approval: 27 oktober 2022
Date of cessation: 01 august 2023 -
Company name: Janssen-Cilag B.V.
Indication:
Adult patients with relapsed and refractory multiple myeloma who have exhausted all available treatment options accessible as local standard of care (e.g. PIs, IMIDs, anti CD38 mAbs and therapies targeting XPO1 and BCMA) and who have evidence of disease progression after the last therapy.Date of approval: 22 juli 2022
-
-
2021
-
Company name: Novartis Pharma B.V.
Indication:
Adult patients with Philadelphia chromosome positive chronic myeloid leukaemia in chronic phase (Ph+ CML CP) after failure to or intolerance of two or more tyrosine kinase inhibitors, and for whom no alternative treatment options are available.
Date of approval: 2 November 2021
Date of cessation: 2 November 2022 -
Company name: Janssen-Cilag B.V.
Indication:
As monotherapy for treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with activating epidermal growth factor receptor (EGFR) Exon 20 insertion mutations, after failure of platinum-based therapy.
Date of approval: 29 October 2021
Date of cessation: 1 april 2023 -
Company name: Boehringer Ingelheim B.V.
Indication: Treatment of patients with generalized pustular psoriasis (GPP) presenting with a flare.
Date of approval: 14 October 2021
Date of cessation: 1 August 2023 -
Company name: Celgene Europe B.V.
Indication: Maintenance therapy in newly diagnosed adult FLT3 mutation negative patients with acute myelogenous leukemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive chemotherapy induction with or without consolidation treatment, and who are not candidates for, including those who choose not to proceed to, hematopoietic stem cell transplantation (HSCT) and who are not eligible for a clinical trial.
Date of approval: 12 March 2021
Date of cessation: 11 March 2022 -
Company name: GlaxoSmithKline B.V.
Indication: Adult patients with recurrent or advanced mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) endometrial cancer (EC) that has progressed on or following prior treatment with a platinum-containing regimen.
Date of approval: 4 March 2021
Date of cessation: 3 March 2023
-
-
2020
-
Naam firma: Roche Nederland B.V.
Indicatie:
- Confirmed of 5q autosomal recessive SMA (Spinal muscular atrophy)
- Clinical history, signs or symptoms of Type 1 or Type 2 SMA
- ≥ 2 months of age
- not eligible for clinical trials
- not eligible, have an unsatisfactory response or have exhausted all other viable treatment options – including Spinraza and Zolgensma.
Datum goedkeuring: 23 oktober 2020
Datum stopzetting: 21 oktober 2023, tenzij vergoeding van het geneesmiddel eerder is toegekend door ZiN en het middel daardoor eerder op de Nederlandse markt beschikbaar komt. -
Naam firma: Gilead Sciences Netherlands BV
Indicatie: Treatment of adult and paediatric patients from 12 years of age weighing at least 40 kg requiring invasive mechanical ventilation, due to COVID-19 confirmed by polymerase chain reaction (PCR) or who have known contact with confirmed case of COVID-19, with PCR pending.
Datum goedkeuring: 7 April 2020
Datum stopzetting: 14 December 2020
-
-
2019
-
Naam firma: Novartis Pharma BV
Indicatie: Postmenopausal women and men with HR-positive, HER2-negative advanced breast cancer with mutated phosphoinositide 3-kinase in combination with fulvestrant who progressed on or after any other Aromatase Inhibitor treatment.
Datum goedkeuring: 7 October 2019
Datum stopzetting: 7 October 2020 -
Naam firma: Bayer B.V.
Indicatie: Vitrakvi als monotherapie is geïndiceerd voor de behandeling van volwassen en pediatrische patiënten met solide tumoren die een neurotrofe tyrosinereceptorkinase (NTRK) genfusie vertonen,
- die een ziekte hebben die lokaal gevorderd of gemetastaseerd is of waarbij de kans groot is dat chirurgische resectie leidt tot ernstige morbiditeit, en
- die geen bevredigende behandelopties hebben.
Datum goedkeuring: 5 september 2019
Datum stopzetting: 16 september 2021
-
Naam firma: Janssen-Cilag B.V.
Indicatie: behandelresistente depressie (treatment resistent depression - TRD). Alle andere behandelopties dienen te zijn uitgeput.
Datum goedkeuring: 26 augustus 2019
Datum stopzetting: 1 september 2021
-
Naam firma: Pfizer Limited
Indicatie: As monotherapy for the treatment of adult patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) whose disease has progressed on crizotinib and at least one other ALK inhibitor for metastatic disease.
Datum van goedkeuring: 4 februari 2019
Datum van stopzetting: 1 augustus 2019
-
Naam firma: Roche NL
Indicatie: In combination with bendamustine and rituximab is indicated for the treatment of previously treated adult patients with diffuse large B-cell lymphoma (DLBCL) who are not candidates for haematopoietic stem cell transplant and who fulfil the eligibility criteria outlined in the patient profile. Inclusion Criteria: Patient has exhausted all therapeutic options for r/r DLBCL and has been treated with at least 2 prior lines of therapy including R-CHOP (or similar regimen for 1L DLBCL).
Datum goedkeuring: 28 januari 2019
Datum stopzetting: 1 april 2020
-
-
2018
-
Naam firma: Alnylam UK limited
Indicatie: Treatment of adults with stage I-III hATTR amyloidosis polyneuropathy (PN)
Datum van goedkeuring: 21 juni 2018
Datum van stopzetting: 11 januari 2019
-
Naam firma: Jazz Pharmaceuticals, Ireland Limited
Indicatie: Patients 18 years of age and older with therapy related AML (t-AML) or AML with myelodysplasia-related changes.
Datum van goedkeuring: 18 april 2018
Datum van stopzetting: 21 maart 2019
-
-
2017
-
Naam firma: AstraZeneca
Indicatie: Locally advanced, unresectable NSCLC (Stage III) in adults whose tumours express PD-L1 on > 1% of tumour cells and whose disease has not progressed following platinum based chemoradiation therapy.
Datum van goedkeuring: 21 december 2017
Datum van stopzetting: 1 september 2019 -
Naam firma: Roche
Indicatie: Adult patients with anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC), and who fulfill the eligibility criteria outlined in the patient profile.
Datum van goedkeuring: januari 2017
Datum van stopzetting: maart 2018
-
-
2016
-
Naam firma: Pfizer B.V.
Indicatie: Patients with hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative locally advanced or metastatic breast cancer:- in combination with an aromatase inhibitor
- in combination with fulvestrant in patients who have received prior endocrine therapy
In pre- or perimenopausal women the endocrine therapy should be combined with a luteinizing hormone releasing hormone (LHRH) agonist.
Datum van goedkeuring: 19 juli 2016
Datum van stopzetting: 1 juli 2017 -
Naam firma: AbbVie B.V.
Indicatie: Treatment of adult patients with chronic lymphocytic leukaemia (CLL) in the presence of 17p deletion or TP53 mutations who are insuitable for or have failed a B-cell receptor pathway inhibitor.
Datum van goedkeuring: 21 juni 2016
Datum van stopzetting: 21 juni 2017 -
Naam firma: Janssen-Cilag
Indicatie: Multiple myeloma. Patients who have relapsed and refractory multiple myeloma (MM), who have received at least 3 prior lines of therapy and who are unsuitable for retreatment with a proteasome (PI) and an immunomodulatory agent (IMiD), or whose disease is double refractory to both a PI and an IMiD.
Datum van goedkeuring: 3 februari 2016
Datum van stopzetting: 2 februari 2017 -
Naam firma: AstraZeneca
Indicatie: Adult patients with locally advanced or metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small-cell lung cancer (NSCLC) who have progressed on or after EGFR TKI therapy.
Datum van goedkeuring: 14 januari 2016
Datum van stopzetting: 30 mei 2016
-
-
2015
-
Naam firma: Servier Nederland Farma B.V.
Indicatie: Adult patients with metastatic colorectal cancer (CRC) who have been previously treated with, or are not considered candidates for, available therapies including fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapies, anti-VEGF agents, and anti-EGFR agents.
Datum van goedkeuring: 17 december 2015
Datum van stopzetting: 17 december 2016 -
Naam firma: Bristol-Myers Squibb B.V.
Indicatie: Patients with histologically- or cytologically-documented SqNSCLC and non-SqNSCLC who have relapsed after systemic pretreatment with a minimum of 1 prior systemic treatment for stage IIIB/stage IV disease.
Datum van goedkeuring: 18 mei 2015
Datum van stopzetting: 18 mei 2016 -
Naam firma: Roche Nederland B.V.
Indicatie: For use in combination with Zelboraf (vemurafenib) for treatment-naïve adult patients with BRAF V600 mutation-positive unresectable or metastatic melanoma.
Datum van goedkeuring: 9 maart 2015
Datum van stopzetting: 9 maart 2016
-
-
2014
-
Naam firma: AstraZeneca
Indicatie: Monotherapy for the maintenance treatment of adult patients with platinum sensitive relapsed BRCA mutated (germline and/or somatic) high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete response or partial response) to platinum-based chemotherapy.
Datum van goedkeuring: 15 december 2014
Datum van stopzetting: 22 mei 2015 -
Naam firma: Merck Sharp & Dohme
Indicatie: Patients with a diagnosis of unresectable (Stage III) or metastatic melanoma (Stage IV) who have failed or progressed on standard-of-care systemic therapy including ipilimumab, and when indicated a BRAF/MEK inhibitor.
Datum van goedkeuring: 3 november 2014
Datum van stopzetting: 3 november 2015 -
Naam firma: Boehringer Ingelheim
Indicatie: Treatment of patients with Idiopathic Pulmonary Fibrosis (IPF).
Datum van goedkeuring: 5 juni 2014
Datum van stopzetting: 1 maart 2015 -
Naam firma: Bristol-Myers Squibb B.V.
Indicatie: Subjects chronically infected with hepatitis C who are at a high risk of liver decompensation or death within 12 months if left untreated and who have no available therapeutic options.
Datum van goedkeuring: 14 april 2014
Datum van stopzetting: 1 maart 2015 -
Naam firma: Johnson & Johnson / Janssen-Cilag B.V.
Indicatie: Treatment of adult patients with relapsed or refractory: mantle cell lymphoma (MCL), chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
Datum van goedkeuring: 10 april 2014
Datum van stopzetting: 3 november 2014 -
Naam firma: Lilly Nederland B.V.
Indicatie: Cyramza in combination with paclitaxel is indicated for the treatment of adult patients with advanced gastric cancer or gastro-oesophageal junction adenocarcinoma with disease progression after prior platinum and fluoropyrimidine chemotherapy. Cyramza monotherapy is indicated for the treatment of adult patients with advanced gastric cancer or gastro-oesophageal junction adenocarcinoma with disease progression after prior platinum or fluoropyrimidine chemotherapy, for whom treatment in combination with paclitaxel is not appropriate.
Datum van goedkeuring: 3 maart 2014
Datum van stopzetting: 5 februari 2015
-
-
2013
-
Naam firma: GlaxoSmithKline
Indicatie: Unresectable or metastatic melanoma with BRAF V600 mutation who did not receive previous treatment with BRAF- and/or MEK inhibitors.
Datum van goedkeuring: 7 juni 2013
Datum van stopzetting: 7 juni 2014 -
Naam firma: Roche
Indicatie: Metastasised breast cancer. Have received at least two lines of Herceptin-containing regimens in combination with two different forms of chemotherapy (anthracyclines, taxanes and capecitabine) for the metastasised disease in addition to a minimum of one line of lapatinib.
Datum van goedkeuring: 7 januari 2013
Datum van stopzetting: 14 februari 2014
-
-
2010
-
Naam firma: Janssen-Cilag
Indicatie: Treatment of metastatic castration resistant prostate cancer in adult men whose disease has progressed on or after a docetaxel-based chemotherapy regimen.
Datum van goedkeuring: 25 november 2010
Datum van stopzetting: 15 december 2011 -
Naam firma: Roche Nederland B.V.
Indicatie: Patients with an influenza infection who are unable to take, tolerate or absorb oral formulations and for whom intravenous administration of an intravenous neuraminidase inhibitor is indicated.
Datum van goedkeuring: 12 oktober 2010
Datum van stopzetting: 12 oktober 2011 -
Naam firma: GlaxoSmithKline
Indicatie: Treatment of severe influenza in critically ill hospitalised patients.
Datum van goedkeuring: 12 oktober 2010
Datum van stopzetting: 28 juni 2014 -
Naam firma: Sanofi-Aventis
Indicatie: Treatment of patients with hormone refractory metastatic prostate cancer previously treated with a docetaxel-containing regimen.
Datum van goedkeuring: 5 juni 2010
Datum van stopzetting: 5 juni 2011
-