Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey

• Published in: Turkish journal of medical sciences Vol. 51; no. 4; pp. 1791 - 1799
• Main Authors: Kırmızı, N. İpek, Aydın, Volkan, Akıcı, Narin, Bayar, Banu, Akıcı, Ahmet
• Format: Journal Article
• Language: English
• Published: Turkey The Scientific and Technological Research Council of Turkey 30.08.2021
• Subjects: Antibodies, Monoclonal, Humanized - therapeutic use; Child; Drug Utilization - statistics & numerical data; Female; Humans; Male; Off-Label Use - statistics & numerical data; Pediatrics; Rare Diseases - drug therapy; Rare Diseases - epidemiology; Retrospective Studies; Turkey - epidemiology; Turkey; biotechnological drugs; canakinumab; rare disease; Off-label drug use; pediatrics
• ISSN: 1300-0144; 1303-6165
• DOI: 10.3906/sag-2012-355

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. 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. 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. 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.