Prix Galien

Referenties Ajovy ® 1. https://www.lumc.nl/org/hoofdpijnonderzoek/informatie/ migraine/#:~:text=Migraine%20is%20een%20van%20 de,%C3%A9%C3%A9n%20of%20twee%20aanvallen%20krijgen 2. Samenvatting van de productkenmerken AJOVY: EU/1/19/1358 op https://www.cbg-meb.nl 3. Sacco S et al. J Headache Pain 2019; 20(1): 6. 4. Hepp Z et al. J Manag Care Pharm 2014; 20(1) 22–33 5. Dodick DWet al. JAMA 2018; 319(19):1999–2008. 6. Silberstein SD et al. N Engl J Med 2017; 377(22): 2113–2122 7. Ferrari MD et al. Lancet 2019; doi:10.1016/S0140-6736(19)31946-4 Referenties Arikayce ® liposomal 1. Falkingham JO. Current Epidemiologic Trends of the Nontuberculous Mycobacteria (NTM). 2016; Curr Environ Health Rep, 3(2):161-7 2. Awuh JA and Flo TH. Molecular basis of mycobacterial survival in macrophages. 2017; Cell Mol Life Sci 74, 1625–1648 3. Qvist T et al. Chronic pulmonary disease withMycobacterium abscessus complex is a biofilm infection. 2015; Eur Respir J, 46(6):1823-1826 4. LipmanMet al. Current and future management of non-tuberculous mycobacterial pulmonary disease (NTM-PD) in the UK. 2020; BMJ Open Respiratory Research, 7:e000591 5. Doyle OMet al. Identification of potentially undiagnosed patients with nontuberculous mycobacterial lung disease using machine learning applied to primary care data in the UK. 2020; Eur Respir J, 56(4):2000045 6. Ringshausen FC et al. Predictive modeling of nontuberculous mycobacterial pulmonary disease epidemiology using German health claims data. 2021; Int J Infect Dis, 11;104:398-406 7. Kotilianen H et al. Clinical findings in relation to mortality in nontuberculous a. mycobacterial infections: Patients withMycobacterium avium complex have better survival than patients with other mycobacteria. 2015; Eur J ClinMicrobiol Infect Dis, 34(9):1909-19182015 8. Wagner D et al. Annual prevalence and treatment estimates of nontuberculous mycobacterial pulmonary disease in Europe: ANTM- NET collaborative study. 2014; Eur Respir J, 44(Suppl 58):P1067 9. MethaMandMarras TK. Impaired health-related quality of life in pulmonary nontuberculous mycobacterial disease. 2011; Respir Med, 105(11):1718-1725 10. Diel R et al. High mortality in patients withMycobacterium avium complex lung disease: a systematic review. 2018; BMC Infect Dis, 18(1):1-102016 11. Park HY et al. Lung function decline according to clinical course in nontuberculous mycobacterial lung disease. 2016; Chest, 150(6):1222-12322016 12. Park HY et al. Natural course of the nodular bronchiectatic form of Mycobacterium avium complex lung disease: Long-term radiologic change without treatment. 2017; PLoS One, 12(10):1-11.2019 13. Goring SMet al. The cost of Mycobacterium avium complex lung disease in Canada, France, Germany, and the United Kingdom: a nationally representative observational study2018; BMCHealth Services Research, 18:700 14. Dayley C et al. Treatment of nontuberculous Mycobacterial Pulmonary Disease: an official ATS/ERS/ESCMID/IDSA Clinical Practice Guideline. 2020; Eur Respir J, 56:2000535 15. Expertise centrum https://www.radboudumc.nl/expertisecentra/ expertisecentra-zeldzame-aandoeningen/tuberculose-en- nontuberculeuze-mycobacteriele-infectieziekten 16. Zhang J et al. Amikacin liposome inhalation suspension (ALIS) penetrates non-tuberculous mycobacterial biofilms and enhances amikacin uptake into macrophages. 2018; Front Microbiol, 9:1- 122018 17. Griffith DE et al. Amikacin liposome inhalation suspension for treatment-refractory lung disease caused byMycobacterium avium complex (CONVERT): A prospective, open-label, randomized study. 2081; Am J Respir Crit CareMed, 198(12):1559-15692018 Referenties Comirnaty ® 1. Polack FP, Thomas SJ, Kitchin N, Absalon J, Gurtman A, Lockhart S, Perez JL, Pérez Marc G, Moreira ED, Zerbini C, Bailey R, Swanson KA, Roychoudhury S, Koury K, Li P, KalinaWV, Cooper D, Frenck RWJr, Hammitt LL, Türeci Ö, Nell H, Schaefer A, Ünal S, Tresnan DB, Mather S, Dormitzer PR, Sahin U, Jansen KU, Gruber WC; C4591001 Clinical Trial Group. Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine. N Engl J Med. 2020 Dec 31;383(27):2603-2615. doi: 10.1056/NEJMoa2034577. Epub 2020 Dec 10. PMID: 33301246; PMCID: PMC7745181 2. Samenvatting van productkenmerken en EPAR te raadplegen via https://www.ema.europa.eu/en/medicines/human/EPAR/comirnaty 3. Noa Dagan, M.D., NoamBarda, M.D., Eldad Kepten, Ph.D., Oren Miron, M.A., Shay Perchik, M.A., Mark A. Katz, M.D., Miguel A. Hernán, M.D., Marc Lipsitch, D.Phil., Ben Reis, Ph.D., and Ran D. Balicer, M.D. BNT162b2 mRNA Covid-19 Vaccine in a Nationwide Mass Vaccination Setting | NEJMFebruary 24, 2021 DOI: 10.1056/ NEJMoa2101765 Referenties COVID-19 vaccineModerna 1. https://www.undp.org/content/undp/en/home/coronavirus/socio- economic-impact-of-covid-19.html 2. Johns Hopkins University &Medicine, Coronavirus Resource Center - Global Covid Death confirmed and probable where reported https:// coronavirus.jhu.edu/map.html 3. https://www.cdc.gov/coronavirus/2019-ncov/vaccines/different- vaccines/mrna.html 4. https://www.nejm.org/doi/full/10.1056/NEJMoa2035389 5. https://www.ema.europa.eu/en/news/ema-recommends-covid-19- vaccine-moderna-authorisation-eu Referenties Givlaari ® 1. Te Velde, K. Et al. Een familie-onderzoek bij de eerste Nederlandse patiënten met porfyrie NTVG; 1989;13,51:2547-2551. Te Velde, K. Nederlandse families met acute porfyrie; 2010 2. Neeleman, RA, et al. Diagnostic and therapeutic strategies for porphyrias; Neth. J. Med. 2020;78,4:149-160 3. Balwani, M, et al. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria; N Engl J Med 2020;382:2289-301 4. AS1-NLD-00037 Version: April 2020 Referenties Jyseleca ® 1. SmPC Jyseleca ® . September 2020. 2. https://www.volksgezonheiden zorg.info/onderwerp/reumatoïde- artritis-ra bezocht op 22 februari 2021 3. Prince FH, et al. Arthritis Res Ther 2012; 14:R68 4. Hamann PDH, et al. Rheumatology (Oxford) 2019; pii: kez188 5. Yu C, et al. Clin Rheumatol 2019; 38:727-738 Referenties Kaftrio ® 1. European Cystic Fibrosis Society. 2017 ECFS Patient Registry Annual Data Report. Karup, Denmark: EuropeanCystic Fibrosis Society; 2019 2. Heijerman H, De Jonge H. Expressie van het cystische-fibrose-gen in de longen. Ned Tijdschr Geneesk 2004;148:816-819 3. Van de Belt E, Kraan J, Bouman K, et al. Klinische manifestaties cystische fibrose bij volwassen patiënten. Ned Tijdschr Geneeskd 2009;153:B155 4. Rommens JM, Iannuzzi MC, KeremB, et al. Identification of the cystic fibrosis gene: chromosome walking and jumping. Science 1989;245:1059-1065 5. Nederlandse Cystic Fibrosis Registratie. Rapportage van Het Jaar 2019. https://ncfs.nl/wp-content/uploads/2021/01/CF-Registratie- 2019-rapport-NL.pdf. Geraadpleegd januari 2021 6. Heijerman HGM, McKone EF, Downey DG, et al. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet 2019;394:1940-1948 7. Middleton PG, Mall MA, Drevínek P, et al. Elexacaftor-tezacaftor- ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med 2019;381:1809-1819 Referenties Libtayo ® 1. SmPC Libtayo. Website CBG, Geneesmiddeleninformatiebank. 2021. Bezocht op 9 feb 2021. Beschikbaar op: https://www. geneesmiddeleninformatiebank.nl/nl/ 2. Integraal Kanker CentrumNederland. Rapport Huidkanker in Nederland. 2019. Bezocht op 9 feb 2021. Beschikbaar op: https:// www.iknl.nl/docs/default-source/PDF_Docs/iknl_huidkanker-in- nl_rapport_nkr.pdf?sfvrsn=2 3. EuropeanMedicines Agency. EPAR - Public Assessment Report Libtayo. 2019. Bezocht op 16 feb 2021. Beschikbaar op: https:// www.ema.europa.eu/en/documents/assessment-report/libtayo-epar- public-assessment-report_en.pdf 4. MigdenM, Khushalani N, Chang A, et al. Cemiplimab in locally advanced cutaneous squamous cell carcinoma: results from an open- label, phase 2, single-arm trial. The Lancet Oncology. 2020; 21(2) 5. Rischin D, MigdenM, LimA, et al. Phase 2 study of cemiplimab in patients with metastatic cutaneous squamous cell carcinoma: primary analysis of fixed-dosing, long-term outcome of weight-based dosing. Journal for ImmunoTherapy of Cancer. 2020;(8:e000775) 6. NVMO. Website Nederlandse Vereniging voor Medische Oncologie. 2021. Bezocht op 17 feb 2021. Beschikbaar op: https://www.nvmo. org/2021/01/drug-access-protocol-gestart/ 42 | PRIX GALIEN NEDERLAND 2020-2021