Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey
Yıl: 2021 Cilt: 51 Sayı: 4 Sayfa Aralığı: 1791 - 1799 Metin Dili: İngilizce DOI: 10.3906/sag-2012-355 İndeks Tarihi: 12-01-2022
Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey
Öz: Background/aim: Pediatric patients, especially those with rare diseases, represent a population that has a high tendency towards offlabel 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. smallmolecule (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.Key words: Off-label drug use, pediatrics, rare disease, biotechnological drugs, canakinumab
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| APA | Kirmizi Sonmez N, Aydin V, Akıcı N, BAYAR B, Akici A (2021). Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey. , 1791 - 1799. 10.3906/sag-2012-355 |
| Chicago | Kirmizi Sonmez N. Ipek,Aydin Volkan,Akıcı Narin,BAYAR BANU,Akici Ahmet Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey. (2021): 1791 - 1799. 10.3906/sag-2012-355 |
| MLA | Kirmizi Sonmez N. Ipek,Aydin Volkan,Akıcı Narin,BAYAR BANU,Akici Ahmet Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey. , 2021, ss.1791 - 1799. 10.3906/sag-2012-355 |
| AMA | Kirmizi Sonmez N,Aydin V,Akıcı N,BAYAR B,Akici A Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey. . 2021; 1791 - 1799. 10.3906/sag-2012-355 |
| Vancouver | Kirmizi Sonmez N,Aydin V,Akıcı N,BAYAR B,Akici A Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey. . 2021; 1791 - 1799. 10.3906/sag-2012-355 |
| IEEE | Kirmizi Sonmez N,Aydin V,Akıcı N,BAYAR B,Akici A "Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey." , ss.1791 - 1799, 2021. 10.3906/sag-2012-355 |
| ISNAD | Kirmizi Sonmez, N. Ipek vd. "Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey". (2021), 1791-1799. https://doi.org/10.3906/sag-2012-355 |
| APA | Kirmizi Sonmez N, Aydin V, Akıcı N, BAYAR B, Akici 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. 10.3906/sag-2012-355 |
| Chicago | Kirmizi Sonmez N. Ipek,Aydin Volkan,Akıcı Narin,BAYAR BANU,Akici Ahmet Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey. Turkish Journal of Medical Sciences 51, no.4 (2021): 1791 - 1799. 10.3906/sag-2012-355 |
| MLA | Kirmizi Sonmez N. Ipek,Aydin Volkan,Akıcı Narin,BAYAR BANU,Akici Ahmet Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey. Turkish Journal of Medical Sciences, vol.51, no.4, 2021, ss.1791 - 1799. 10.3906/sag-2012-355 |
| AMA | Kirmizi Sonmez N,Aydin V,Akıcı N,BAYAR B,Akici A Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey. Turkish Journal of Medical Sciences. 2021; 51(4): 1791 - 1799. 10.3906/sag-2012-355 |
| Vancouver | Kirmizi Sonmez N,Aydin V,Akıcı N,BAYAR B,Akici A Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey. Turkish Journal of Medical Sciences. 2021; 51(4): 1791 - 1799. 10.3906/sag-2012-355 |
| IEEE | Kirmizi Sonmez N,Aydin V,Akıcı N,BAYAR B,Akici A "Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey." Turkish Journal of Medical Sciences, 51, ss.1791 - 1799, 2021. 10.3906/sag-2012-355 |
| ISNAD | Kirmizi Sonmez, N. Ipek vd. "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 (2021), 1791-1799. https://doi.org/10.3906/sag-2012-355 |
