Kirmizi, N. Ipek; Aydin, Volkan; Akici, Narin; Bayar, Banu; Akici, Ahmet published the artcile< Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey>, Quality Control of 1492-18-8, the main research area is biotechnol drug rare disease OLDU child; biotechnological drugs; canakinumab; pediatrics; rare disease; Off-label drug use.
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 biotechnol. drug use in children with rare diseases requiring OLDU. This retrospective study examined all single-diagnosed OLDU applications (n = 5792) for 4992 children (<18-yr) in Turkey. Applications of rare diseases were selected, and their descriptive characteristics were examined, including demog. features of patients, biotechnol. 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. Biotechnol. drug use was higher in rare disease applications than in nonrare diseases (37.9% vs. 19.2%, resp.; p < 0.0001). Canakinumab was the top applied biotechnol. drug (73.2%). Compared to that in small-mol. drugs, the mean age of patients was higher in biotechnol. drug-containing applications (8.1 ± 5.3 vs. 9.7 ± 4.9, resp.; p < 0.0001). Biotechnol. 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 biotechnol. (32.3%) vs. smallmol. (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 biotechnol. OLDU in this group is almost 2-fold of small-mol. drug use. While further projected findings imply a higher approval tendency for rare diseases and biotechnol. drugs, there seems to be more room for improvement for pediatric drug use. Turkish Journal of Medical Sciences published new progress about Blood. 1492-18-8 belongs to class alcohols-buliding-blocks, and the molecular formula is C20H21CaN7O7, Quality Control of 1492-18-8.
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