{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,12,15]],"date-time":"2025-12-15T04:28:30Z","timestamp":1765772910716,"version":"3.37.3"},"reference-count":57,"publisher":"Informa UK Limited","issue":"5","funder":[{"DOI":"10.13039\/501100002701","name":"Ministry of Education","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100002701","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["www.tandfonline.com"],"crossmark-restriction":true},"short-container-title":["Expert Review of Pharmacoeconomics &amp; Outcomes Research"],"published-print":{"date-parts":[[2023,5,28]]},"DOI":"10.1080\/14737167.2023.2197213","type":"journal-article","created":{"date-parts":[[2023,3,30]],"date-time":"2023-03-30T11:55:48Z","timestamp":1680177348000},"page":"561-570","update-policy":"https:\/\/doi.org\/10.1080\/tandf_crossmark_01","source":"Crossref","is-referenced-by-count":4,"title":["Economic analysis of cerliponase alfa for treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2)"],"prefix":"10.1080","volume":"23","author":[{"given":"Medo","family":"Guti\u0107","sequence":"first","affiliation":[{"name":"Department of Pharmacology and Toxicology, Faculty of Medical Sciences, University of Kragujevac, Kragujevac, Serbia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1786-1507","authenticated-orcid":false,"given":"Milo\u0161 N.","family":"Milosavljevi\u0107","sequence":"additional","affiliation":[{"name":"Department of Pharmacology and Toxicology, Faculty of Medical Sciences, University of Kragujevac, Kragujevac, Serbia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9297-8449","authenticated-orcid":false,"given":"Teodora","family":"Safiye","sequence":"additional","affiliation":[{"name":"Department of Pharmacology and Toxicology, Faculty of Medical Sciences, University of Kragujevac, Kragujevac, Serbia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ardea","family":"Milidrag","sequence":"additional","affiliation":[{"name":"Department of Pharmacology and Toxicology, Faculty of Medical Sciences, University of Kragujevac, Kragujevac, Serbia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Slobodan M.","family":"Jankovic","sequence":"additional","affiliation":[{"name":"Department of Pharmacology and Toxicology, Faculty of Medical Sciences, University of Kragujevac, Kragujevac, Serbia"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"301","published-online":{"date-parts":[[2023,4,3]]},"reference":[{"key":"cit0001","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2016.07.011"},{"key":"cit0002","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-016-0414-2"},{"key":"cit0003","doi-asserted-by":"publisher","DOI":"10.1111\/jpc.14890"},{"key":"cit0004","doi-asserted-by":"publisher","DOI":"10.1080\/21678707.2020.1856654"},{"key":"cit0005","doi-asserted-by":"publisher","DOI":"10.1177\/0883073819895694"},{"key":"cit0006","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2020.04.018"},{"key":"cit0007","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30368-5"},{"key":"cit0008","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2017.01.034"},{"key":"cit0009","unstructured":"Clinical review report: cerliponase alfa (brineura): (BioMarin Pharmaceutical (Canada) Inc.): indication: for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2019 Jun. Introduction. [cited 1 Oct 2022]. Available from: https:\/\/www.ncbi.nlm.nih.gov\/books\/NBK544816\/."},{"key":"cit0010","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1712649"},{"key":"cit0011","doi-asserted-by":"publisher","DOI":"10.1590\/s1413-81232010000900019"},{"key":"cit0012","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0731-0"},{"key":"cit0013","doi-asserted-by":"publisher","DOI":"10.1007\/s11096-022-01491-8"},{"key":"cit0014","unstructured":"WDI \u2013 Classifying countries by income. [Internet]. [cited 1 Oct 2022]. Available from: https:\/\/datatopics.worldbank.org\/world-development-indicators\/stories\/the-classification-of-countries-by-income.html."},{"key":"cit0015","unstructured":"Parliament of Republic of Serbia. Health Insurance Law. Off Gaz RS. 2019. [Internet]. [cited 1 Oct 2022]. Available from: https:\/\/www.paragraf.rs\/propisi\/zakon_o_zdravstvenom_osiguranju.html."},{"key":"cit0016","unstructured":"National Centre for Pharmacoeconomics. Cost-effectiveness of cerliponase alfa (Brineura\u00ae) for the treatment of patients with CLN2 disease. [Internet]. [cited 1 Oct 2022]. Available from:https:\/\/www.ncpe.ie\/wp-content\/uploads\/2017\/11\/Summary-CLN-alfa-26.03.2019.pdf."},{"key":"cit0017","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-4642(18)30179-2"},{"key":"cit0018","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.10660"},{"key":"cit0019","doi-asserted-by":"publisher","DOI":"10.1177\/0883073812448459"},{"key":"cit0020","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-01829-x"},{"key":"cit0021","doi-asserted-by":"publisher","DOI":"10.1002\/epi4.12365"},{"key":"cit0022","doi-asserted-by":"publisher","DOI":"10.3390\/ijerph17093102"},{"key":"cit0023","doi-asserted-by":"crossref","first-page":"1476","DOI":"10.1002\/ajmg.a.61197","volume":"179","author":"Varni JW","year":"2019","journal-title":"Am J Med Genet A"},{"key":"cit0024","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpurol.2018.07.017"},{"key":"cit0025","doi-asserted-by":"publisher","DOI":"10.1177\/0272989X14557178"},{"key":"cit0026","doi-asserted-by":"publisher","DOI":"10.1111\/j.1464-5491.2008.02430.x"},{"key":"cit0027","doi-asserted-by":"publisher","DOI":"10.1016\/j.vhri.2021.05.007"},{"key":"cit0028","doi-asserted-by":"publisher","DOI":"10.1007\/s00198-015-3350-4"},{"key":"cit0029","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2020-046609"},{"key":"cit0030","doi-asserted-by":"publisher","DOI":"10.1002\/gps.2610"},{"key":"cit0031","doi-asserted-by":"publisher","DOI":"10.2165\/11633900-000000000-00000"},{"key":"cit0032","doi-asserted-by":"publisher","DOI":"10.1016\/j.eclinm.2022.101354"},{"key":"cit0033","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1097\/01.NT.0000521171.91100.84","volume":"17","author":"Shaw G","year":"2017","journal-title":"Neurology Today"},{"key":"cit0034","unstructured":"Statistical Office of the Republic of Serbia 2022. [Internet]. [cited 1 Oct 2022]. Available from: https:\/\/data.stat.gov.rs\/Home\/Result\/09020101?languageCode=sr-Cyrl."},{"key":"cit0035","unstructured":"National Bank of Serbia. Interest rates. [Internet]. 2021. [Internet]. [cited 1 Oct 2022]. Available from: https:\/\/www.nbs.rs\/sr\/ciljevi-i-funkcije\/monetarna-politika\/kamatne-stope\/."},{"key":"cit0036","unstructured":"Republic Health Insurance Fund of Serbia. Rulebook on the prices of health services at the secondary and tertiary levels of health care. Off Gaz RS. 55\/2019, 53\/2021"},{"key":"cit0037","unstructured":"Republic Health Insurance Fund of Serbia. Rulebook on the list of medicines prescribed and issued at the expense of compulsory health insurance funds. Off Gaz RS. 2019. 43\/19, 55\/19, 56\/19-correction, 73\/19, 87\/19, 18\/20, 43\/20, 108\/20, 49\/21, 51\/21-correction and 60\/21"},{"journal-title":"Off Gaz RS","year":"2021","author":"The Government of the Republic of Serbia","key":"cit0038"},{"key":"cit0039","doi-asserted-by":"publisher","DOI":"10.1007\/s40273-014-0147-9"},{"key":"cit0040","unstructured":"Jankovic SM Discrete-event simulation model for comparison of cost-effectiveness of cerliponase alfa vs. symptomatic therapy in patients with late neuronal lipofuscinosis type 2. 2022. [Internet]. [cited 1 Oct 2022]. Available from: https:\/\/docs.google.com\/spreadsheets\/d\/1VP2S9IagPnvvjrUxkVhn_YruZLa2O6Vt\/edit#gid=1401080500."},{"key":"cit0041","unstructured":"Food, Administration D. FDA news release. FDA approves first treatment for a form of batten disease. [Internet]. [cited 1 Oct 2022]. ymptome from: https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-first-treatment-form-batten-disease."},{"key":"cit0042","unstructured":"European Medical Agency. EPAR summary for the public. Brineura (cerliponase alfa). [Internet]. [cited 1 Oct 2022]. Available from: https:\/\/www.ema.europa.eu\/en\/documents\/overview\/brineura-epar-summary-public_en.pdf."},{"key":"cit0043","unstructured":"Food and Drug Administration. Brineura- highlights of prescribing information. [Internet]. [cited 1 Oct 2022]. Available from: https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2017\/761052lbl.pdf."},{"key":"cit0044","unstructured":"Pharmacoeconomic review report: cerliponase Alfa (Brineura): (BioMarin Pharmaceutical (Canada) Inc.): indication: for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2019 Jun. Appendix 1, Cost Comparison. [cited 1 Oct 2022]. Available from: https:\/\/www.ncbi.nlm.nih.gov\/books\/NBK544832\/."},{"key":"cit0045","unstructured":"CADTH Canadian Drug Expert Committee Recommendation: Cerliponase Alfa (Brineura \u2014 BioMarin Pharmaceutical [Canada] Inc.): indication: for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2019 May. [cited 1 Oct 2022]. Available from: https:\/\/www.ncbi.nlm.nih.gov\/books\/NBK543392\/."},{"key":"cit0046","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-02147-y"},{"key":"cit0047","unstructured":"National Centre for Pharmacoeconomics. NCPE Ireland. Cost-effectiveness of cerliponase alfa (Brineura\u00ae) for the treatment of patients with CLN2 disease. [Internet]. [cited 1 Oct 2022]. Available at: https:\/\/www.ncpe.ie\/wp-content\/uploads\/2017\/11\/\/Summary-CLN-alfa-26.03.2019.pdf"},{"key":"cit0048","doi-asserted-by":"publisher","DOI":"10.1080\/13696998.2020.1815026"},{"volume-title":"Experiencia cl\u00ednica con medicamentos hu\u00e9rfanos para enfermedades raras metab\u00f3licas [Clinical experience with orphan drugs for rare metabolic diseases]","year":"2020","author":"Caso-Gonz\u00e1lez A","key":"cit0049"},{"key":"cit0050","doi-asserted-by":"publisher","DOI":"10.1007\/s10198-016-0842-x"},{"key":"cit0051","unstructured":"Statista. Health, Pharma & Medtech. Health professionals & hospitals. Average cost of inpatient day at U.S. hospitals in 2019, by hospital type. [Internet]. [cited 1 Oct 2022]. Available from: https:\/\/www.statista.com\/statistics\/630443\/inpatient-day-hospital-costs-in-us-by-nonprofit-or-profit\/."},{"key":"cit0052","doi-asserted-by":"publisher","DOI":"10.1136\/rmdopen-2018-000794"},{"key":"cit0053","doi-asserted-by":"publisher","DOI":"10.2478\/sjecr-2021-0049"},{"key":"cit0054","doi-asserted-by":"publisher","DOI":"10.1016\/j.healthpol.2009.12.001"},{"key":"cit0055","doi-asserted-by":"publisher","DOI":"10.5152\/balkanmedj.2015.15185"},{"key":"cit0056","unstructured":"NICE. Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2. 2019. [Internet]. [cited 1 Oct 2022]. Available from: https:\/\/www.nice.org.uk\/guidance\/hst12."},{"key":"cit0057","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23672"}],"container-title":["Expert Review of Pharmacoeconomics &amp; Outcomes Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.tandfonline.com\/doi\/pdf\/10.1080\/14737167.2023.2197213","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,12,9]],"date-time":"2023-12-09T22:51:56Z","timestamp":1702162316000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.tandfonline.com\/doi\/full\/10.1080\/14737167.2023.2197213"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,4,3]]},"references-count":57,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2023,5,28]]}},"alternative-id":["10.1080\/14737167.2023.2197213"],"URL":"https:\/\/doi.org\/10.1080\/14737167.2023.2197213","relation":{},"ISSN":["1473-7167","1744-8379"],"issn-type":[{"type":"print","value":"1473-7167"},{"type":"electronic","value":"1744-8379"}],"subject":[],"published":{"date-parts":[[2023,4,3]]},"assertion":[{"value":"The publishing and review policy for this title is described in its Aims & Scope.","order":1,"name":"peerreview_statement","label":"Peer Review Statement"},{"value":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=ierp20","URL":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=ierp20","order":2,"name":"aims_and_scope_url","label":"Aim & Scope"},{"value":"2022-10-25","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-03-27","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-04-03","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}