dc.contributor.author | Kırmızı, Neriman İpek | |
dc.contributor.author | Aydın, Volkan | |
dc.contributor.author | Akıcı, Narin | |
dc.contributor.author | Bayar, Banu | |
dc.contributor.author | Akıcı, Ahmet | |
dc.date.accessioned | 2021-09-09T06:16:43Z | |
dc.date.available | 2021-09-09T06:16:43Z | |
dc.date.issued | 2021 | en_US |
dc.identifier.citation | Kırmızı, N. İ., Aydın, V., Akıcı, N., Bayar, B. ve Akıcı, A. (2021). Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey. Turkish Journal of Medical Sciences, 51(4), 1791-1799. https://dx.doi.org/10.3906/sag-2012-355 | en_US |
dc.identifier.issn | 1300-0144 | |
dc.identifier.issn | 1303-6165 | |
dc.identifier.uri | https://dx.doi.org/10.3906/sag-2012-355 | |
dc.identifier.uri | https://hdl.handle.net/20.500.12511/8061 | |
dc.description.abstract | Background/aim: Pediatric patients, especially those with rare diseases, represent a population that has a high tendency towards off label drug use (OLDU) and needs a more careful practice of pharmacotherapy than in adults. We aimed to investigate biotechnological drug use in children with rare diseases requiring OLDU. Materials and methods: This retrospective study examined all single-diagnosed OLDU applications (n = 5792) for 4992 children (<18 year) in Turkey. Applications of rare diseases were selected, and their descriptive characteristics were examined, including demographic features of patients, biotechnological drug utilization status, and disease categories. The off-label statuses of the drugs at the end of 2020 were also examined. Results: In total, 77.7% (n = 4501) of OLDU applications were made for rare diseases. Biotechnological drug use was higher in rare disease applications than in nonrare diseases (37.9% vs. 19.2%, respectively; p < 0.0001). Canakinumab was the top applied biotechnological drug (73.2%). Compared to that in small-molecule drugs, the mean age of patients was higher in biotechnological drug-containing applications (8.1 +/- 5.3 vs. 9.7 +/- 4.9, respectively; p < 0.0001). Biotechnological drug use was higher in nonneoplastic rare diseases (40.3%) than in neoplastic rare diseases (26.4%), (p < 0.0001). At the end of 2020, the approval status of the off-label indications covered in 2016 was significantly higher for rare (24.4%) vs. nonrare (5.2%, p < 0.0001) diseases and for biotechnological (32.3%) vs. small molecule (13.9%, p < 0.0001) drugs. In total, 87.7% of the drugs would have to be still used in the off-label setting at the end of 2020. Conclusion: It was seen that more than three-quarters of the pediatric OLDU applications are for rare diseases, and the need for biotechnological OLDU in this group is almost 2-fold of small-molecule drug use. While further projected findings imply a higher approval tendency for rare diseases and biotechnological drugs, there seems to be more room for improvement for pediatric drug use. | en_US |
dc.description.sponsorship | Marmara University | en_US |
dc.language.iso | eng | en_US |
dc.publisher | TÜBİTAK Scientific and Technological Research Council of Turkey | en_US |
dc.rights | info:eu-repo/semantics/openAccess | en_US |
dc.rights | Attribution 4.0 International | * |
dc.rights.uri | https://creativecommons.org/licenses/by/4.0/ | * |
dc.subject | Off-Label Drug Use | en_US |
dc.subject | Pediatrics | en_US |
dc.subject | Rare Disease | en_US |
dc.subject | Biotechnological Drugs | en_US |
dc.subject | Canakinumab | en_US |
dc.title | Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey | en_US |
dc.type | article | en_US |
dc.relation.ispartof | Turkish Journal of Medical Sciences | en_US |
dc.department | İstanbul Medipol Üniversitesi, Tıp Fakültesi, Dahili Tıp Bilimleri Bölümü, Tıbbi Farmakoloji Ana Bilim Dalı | en_US |
dc.department | İstanbul Medipol Üniversitesi, Uluslararası Tıp Fakültesi, Dahili Tıp Bilimleri Bölümü, Tıbbi Farmakoloji Ana Bilim Dalı | en_US |
dc.authorid | 0000-0001-8294-0802 | en_US |
dc.authorid | 0000-0002-8511-6349 | en_US |
dc.identifier.volume | 51 | en_US |
dc.identifier.issue | 4 | en_US |
dc.identifier.startpage | 1791 | en_US |
dc.identifier.endpage | 1799 | en_US |
dc.relation.publicationcategory | Makale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanı | en_US |
dc.identifier.doi | 10.3906/sag-2012-355 | en_US |
dc.identifier.wosquality | Q4 | en_US |
dc.identifier.scopusquality | Q3 | en_US |