Title Koristi i rizici off-label primjene lijekova Title (english) Benefits and risks of off-label use of drugs Author Karla Petričević Mentor Diana Gujinović (mentor)Committee member Ana Šešelja Perišin (predsjednik povjerenstva)Committee member Josipa Bukić (član povjerenstva)Committee member Diana Gujinović (član povjerenstva)Granter University of Split Defense date and country 2024-10-29, Croatia Scientific / art field, BIOMEDICINE AND HEALTHCARE Abstract Cilj istraživanja: Primjena lijekova izvan odobrenih uvjeta važan je dio legitimne medicinske prakse u cijelom svijetu, s obzirom da učinkoviti odobreni lijekovi ponekad mogu biti rijetki ili nedostupni. Budući da off-label primjena implicira važna pitanja o učinkovitosti i sigurnosti; cilj ovog istraživanja je, pregledom relevantnih izvora, identificirati najnovija saznanja o svim koristima i rizicima ovakve primjene lijekova, kao i njenoj prevalenciji. Materijal i metode: Mrežne stranice regulatornih agencija (HALMED, EMA i FDA), kao i elektronska bibliografska baza podataka PubMed, pregledane su u kolovozu 2024. godine. Pretraga na PubMed-u ograničena je na petogodišnje razdoblje (2019.-2024.) kako bi se osigurala što veća aktualnost podataka Rezultati: Unatoč sve kompleksnijim regulatornim zahtjevima za odobrenje lijeka, prevalencija primjene lijekova izvan odobrenih uvjeta vrlo je visoka i izvan regulative regulatornih agencija. Međutim, naš pregled objava na mrežnim stranicama agencija za lijekove pokazao je kako se podatci vezani uz upitnu sigurnost pojedinih lijekova u off-label primjeni redovito evaluiraju od strane takvih agencija. Pregledom dostupne literature utvrđeno je kako je indikacija najčešće promijenjeni odobreni uvjet, nakon čega slijedi skupina pacijenata, zatim način primjene, doza te farmaceutski oblik. Zaključak: Svijet off-label upotrebe lijekova pun je potencijala, ali još uvijek dosta neistražen. Potrebna je bolja regulacija, kao i više poticaja za klinička ispitivanja, kako bi se pacijentima pružila što sigurnija terapija, a smanjila odgovornost samih liječnika. Glavna je zadaća regulatornih agencija pratiti nuspojave i nadzirati takvu upotrebu lijekova te na temelju toga procijeniti nadmašuju li koristi rizike primjene. Idealno bi bilo da se osmisle službene smjernice koje bi olakšale liječnicima propisivanje u takvim slučajevima.
Abstract (english) Objective: The off-label use of medicine is an important part of legitimate medical practice worldwide, considering that effective, approved drugs could sometimes be scarce or not available. Since off-label use raises important questions about efficacy and safety; the aim of this study is, by reviewing relevant sources, to identify the latest knowledge about all the benefits and risks of this type of drug use, as well as its prevalence. Methods: The websites of the regulatory agencies (HALMED, EMA and FDA), as well as the electronic bibliographic database PubMed, were reviewed in August 2024. The search on PubMed was limited to a five-year period (2019-2024) in order to ensure that the data is as upto- date as possible. Results: Despite the increasingly complex regulatory requirements for drug approval, the prevalence of drug use outside of its approved labeling is very high and excluded from the regulation of regulatory agencies. However, our review of different entries on the websites of medicines agencies showed that data related to the questionable safety of certain drugs in offlabel use were regularly evaluated. A review of the available literature found that the indication was the most frequently changed approved condition, followed by the patient group, then the method of administration, the dose and the pharmaceutical form. Conclusion: The world of off-label use of drugs is full of potential, but still largely unexplored. Better regulation is needed, as well as more incentives for clinical trials, in order to provide patients with safer therapies and to reduce the responsibility of the doctors. The primary task of regulatory agencies is to monitor side effects and oversee such use of drugs, determining whether the benefits outweigh the risks. Ideally, official guidelines should be created to assist doctors in prescribing in such cases.
Keywords
off-label
odobreni uvjeti
regulatorne agencije
djeca
trudnice
starije osobe
rijetke bolesti
onkologija
psihijatrija
prevalencija
Keywords (english)
off-label
approved conditions
regulatory agencies
children
pregnant women
elderly people
rare diseases
oncology
psychiatry
Language croatian URN:NBN urn:nbn:hr:171:773144 Study programme Title: Pharmacy Type of resource Text File origin Born digital Access conditions Open access Terms of use Created on 2024-10-29 12:25:19
