Slučajevi sumnji na primjenu izvan odobrenja (Off-label primjena) u djece prijavljeni Agenciji za lijekove i medicinske proizvode

master's thesis

• Title (english): Cases of suspected off-label use in children reported to the Agency for Medicinal Products and Medical Devices
• Author: Nikolina Šnajcer
• Mentor: Iva Mucalo (mentor)
• Mentor: Nikica Mirošević Skvrce (mentor)
• Committee member: Iva Mucalo (predsjednik povjerenstva)
• Committee member: Nikica Mirošević Skvrce (član povjerenstva)
• Committee member: Jasna Jablan (član povjerenstva)
• Granter: University of Zagreb; Faculty of Pharmacy and Biochemistry; (Centre for applied pharmacy); Zagreb
• Defense date and country: 2022-07-20, Croatia
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Pharmacy; Pharmacy

Abstract

Cilj ovog diplomskog rada je opisati slučajeve sumnji na off-label primjenu lijeka spontano prijavljene Hrvatskoj agenciji za lijekove i medicinske proizvode u pedijatrijskoj populaciji u razdoblju do 31.12.2021. godine. Identificirani su slučajevi u kojima je prijavljena sumnja na off-label primjenu lijeka u osoba mlađih od 18 godina, zaprimljeni zaključno s 31.12.2021. godine. Primarno su analizirane prijave sumnji na nuspojave koje pripadaju High Level Term: Off-label use prema MedDRA. Prijave su analizirane prema dobu i spolu pacijenta, vrsti prijavitelja, djelatnoj tvari, ATK klasifikaciji lijekova, PT klasifikaciji MedDRA i prema ozbiljnosti nuspojava. Ukupno je zaprimljeno 36 prijava sumnji na off-label primjenu lijekova za pacijente mlađe od 18 godina. Najviše prijava je zaprimljeno za djecu starosti od 2 do 11 godina. Najmanje prijava je zaprimljeno za novorođenčad, od 0 do 27 dana starosti. Prema ATK klasifikaciji lijekova, najčešće su bili uključeni lijekovi s djelovanjem na živčani sustav, lijekovi s djelovanjem na sustavne infekcije, lijekovi s djelovanjem na respiratorni sustav i lijekovi s djelovanjem na osjetila. Najzastupljenije djelatne tvari pod sumnjom bile su paliperidon, diklofenak, pantoprazol i mikafungin. Od ukupno 36 zaprimljenih prijava, u tri slučaja se nisu razvile nuspojave na primijenjen lijek, u 21 slučaju su se javile nuspojave koje ubrajamo u neozbiljne, a u 12 slučajeva došlo je do razvoja ozbiljnih nuspojava od čega je u tri slučaja došlo do smrti. Ovim radom uočena je potreba za unapređenjem farmakovigilancijskog sustava te potreba za edukacijom zdravstvenih djelatnika i pacijenata o prijavljivanju korištenja lijekova izvan odobrenih uvjeta te nuspojava koje nastaju primjenom istih.

Abstract (english)

This thesis aims to describe cases of suspected off-label use of drugs spontaneously reported to the Croatian Agency for Medicinal Products and Medical Devices in the pediatric population in the period until the end of 2021 (December 31st). By the end of the same year, there have been identified cases of reporting the suspicion of off-label use of the drug by patients younger than 18 years old. Primary analyses were made for the reports of suspected adverse reactions belonging to High-Level Term: Off-label uses according to MedDRA. Reports were investigated according to the age and sex of the patient, the reporter qualification, the active ingredients, the ATK classification of drugs, the PT classification of MedDRA, and the severity of side effects. A total of 36 reports of suspected off-label use of drugs by patients younger than 18 years old were received. Most applications were received for children between 2 and 11 years old. The lowest number of applications was received for newborns, from 0 to 27 days of age. According to the ATK classification of drugs, drugs with effects on the nervous system, drugs with effects on systemic infections, drugs with effects on the respiratory system, and drugs with effects on the senses were the ones that were the most often included. The most commonly suspected active ingredients were paliperidone, diclofenac, pantoprazole, and micafungin. Out of a total of 36 received reports, there were 3 cases with no side effects developed, 21 cases with side effects we consider to be frivolous, and 12 cases with severe side effects developed (including 3 cases in which death occurred). This thesis identified the need for improvements in the pharmacovigilance system and the need to educate healthcare professionals and patients about reporting the off-label drug use and the side effects that arise from using them.

• Keywords: primjena lijekova; off-label primjena lijekova; nuspojave lijekova; farmakovigilancija; djeca
• Keywords (english): use of drugs; off-label use of drugs; drug side effects; pharmacovigilance; children
• Language: croatian
• URN:NBN: urn:nbn:hr:163:940299
• Study programme: Title: Pharmacy; Study programme type: university; Study level: integrated undergraduate and graduate; Academic / professional title: magistar/magistra farmacije (magistar/magistra farmacije)
• Type of resource: Text
• File origin: Born digital
• Access conditions: Open access
• Created on: 2022-07-22 12:39:07