Overview of approved compassionate use programmes
The MEB can give permission for bringing a medicinal product on the market that has not (yet) been granted a marketing authorisation, known as a compassionate use programme. A company can make this medicinal product available to the patient group. The overview lists all the approved compassionate use programmes including the indication and the duration.
Overview of approved compassionate use programmes
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2021
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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: 29 October 2022 -
Company name: Roche Nederland B.V.
Indication:
- Confirmed of 5q autosomal recessive SMA
- Clinical history, signs or symptoms of Type 1 or Type 2 SMA
- ≥ 2 months of age
And for patients that
b) are not eligible for clinical trials;
c) are not eligible, have an unsatisfactory response or have exhausted all other viable treatment options –including Spinraza and Zolgensma.
Date of approval: 21 October 2021
Date of cessation: 21 October 2022, unless reimbursement of the medicinal product has been assigned by ZiN earlier and the product is available on the Dutch market earlier. -
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: 14 October 2022 -
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 maart 2021
Date of cessation: 11 maart 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 maart 2021
Date of cessation: 3 maart 2022
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2020
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Company name: Roche Nederland B.V.
Indication:- 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.
Date of approval: 23 October 2020
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Company name: Gilead Sciences Netherlands BV
Indication: 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.
Date of approval: 7 April 2020
Date of cessation: 14 December 2020
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2019
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Company name: Novartis Pharma BV
Indication: 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.
Date of approval: 7 October 2019
Datum of cessation: 7 October 2020 -
Company name: Bayer B.V.
Indication: Vitrakvi as monotherapy is indicated for the treatment of adult and paediatric patients with solid tumours that display a Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusion,
- who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and
- who have no satisfactory treatment options.The compassionate use programme is only permitted for patients within the above indication who cannot participate in a study with this medicine in progress in the Netherlands.
Date of approval: 5 September 2019
Date of cessation: expires after 1 year based on this approval from the MEB, unless the medicine becomes available on the Dutch market earlier. At that time this compassionate use program will be terminated.
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Company name: Janssen-Cilag B.V.
Indication: treatment resistent depression - TRD. All other treatment options should have been exhausted.
Date of approval: 26 August 2019
Date of cessation:
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Company name: Pfizer Limited
Indication: 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.
Date of approval: 4 February 2019
Date of cessation: 1 August 2019
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Company name: Roche NL
Indication: 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).
Date of approval: 28 January 2019
Date of cessation:
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2018
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Company name: Alnylam UK limited
Indication: Treatment of adults with stage I-III hATTR amyloidosis polyneuropathy (PN)
Date of approval: 21 June 2018
Date of cessation: 11 January 2019
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Company name: Jazz Pharmaceuticals, Ireland Limited
Indication: Patients 18 years of age and older with therapy related AML (t-AML) or AML with myelodysplasia-related changes.
Date of approval: 18 April 2018
Date of cessation: 21 March 2019
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2017
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Company name: AstraZeneca
Indication: 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.
Date of approval: 21 December 2017
Date of cessation: 1 September 2019 -
Company name: Roche
Indication: 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.
Date of approval: January 2017
Date of cessation: March 2018
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2016
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Company name: Pfizer B.V.
Indication: 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.
Date of approval: 19 July 2016
Date of cessation: 1 July 2017 -
Company name: AbbVie B.V.
Indication: 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.
Date of approval: 21 June 2016
Date of cessation: 21 June 2017 -
Company name: Janssen-Cilag
Indication: 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.
Date of approval: 3 February 2016
Date of cessation: -
Company name: AstraZeneca
Indication: 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.
Date of approval: 14 January 2016
Date of cessation: 30 May 2016
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2015
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Company name: Servier Nederland Farma B.V.
Indication: 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.
Date of approval: 17 December 2015
Date of cessation: 17 December 2016 -
Company name: Bristol-Myers Squibb B.V.
Indication: 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.
Date of approval: 18 May 2015
Date of cessation: 18 May 2016 -
Company name: Roche Nederland B.V.
Indication: For use in combination with Zelboraf (vemurafenib) for treatment-naïve adult patients with BRAF V600 mutation-positive unresectable or metastatic melanoma.
Date of approval: 9 March 2015
Date of cessation: 9 March 2016
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2014
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Company name: AstraZeneca
Indication: 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.
Date of approval: 15 December 2014
Date of cessation: 22 May 2015 -
Company name: Merck Sharp & Dohme
Indication: 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.
Date of approval: 3 November 2014
Date of cessation: 3 November 2015 -
Company name: Boehringer Ingelheim
Indication: Treatment of patients with Idiopathic Pulmonary Fibrosis (IPF).
Date of approval: 5 June 2014
Date of cessation: 1 March 2015 -
Company name: Bristol-Myers Squibb B.V.
Indication: 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.
Date of approval: 14 April 2014
Date of cessation: 1 March 2015 -
Company name: Johnson & Johnson / Janssen-Cilag B.V.
Indication: Treatment of adult patients with relapsed or refractory: mantle cell lymphoma (MCL), chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
Date of approval: 10 April 2014
Date of cessation: 3 November 2014 -
Company name: Lilly Nederland B.V.
Indication: 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.
Date of approval: 3 March 2014
Date of cessation: 5 February 2015
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2013
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Company name: GlaxoSmithKline
Indication: Unresectable or metastatic melanoma with BRAF V600 mutation who did not receive previous treatment with BRAF- and/or MEK inhibitors.
Date of approval: 7 June 2013
Date of cessation: 7 June 2014 -
Company name: Roche
Indication: 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.
Date of approval: 7 January 2013
Date of cessation: 14 February 2014
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2010
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Company name: Janssen-Cilag
Indication: Treatment of metastatic castration resistant prostate cancer in adult men whose disease has progressed on or after a docetaxel-based chemotherapy regimen.
Date of approval: 25 November 2010
Date of cessation: 15 December 2011 -
Company name: Roche Nederland B.V.
Indication: 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.
Date of approval: 12 October 2010
Date of cessation: 12 October 2011 -
Company name: GlaxoSmithKline
Indication: Treatment of severe influenza in critically ill hospitalised patients.
Date of approval: 12 October 2010
Date of cessation: 28 June 2014 -
Company name: Sanofi-Aventis
Indication: Treatment of patients with hormone refractory metastatic prostate cancer previously treated with a docetaxel-containing regimen.
Date of approval: 5 June 2010
Date of cessation: 5 June 2011
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