FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W4205249249> ?p ?o ?g. }

• W4205249249 abstract "Abstract Background Information about the specific regulatory environment of orphan drugs is scarce and inconsistent. Uncertainties surrounding the postmarketing long-term safety of orphan drugs remain. This study aimed to evaluate the labelling changes of orphan drugs and to identify postmarketing safety-associated approval factors. Methods This retrospective cohort study includes all drugs with orphan drug designation approved by the Center for Drug Evaluation and Research of the US Food and Drug Administration between 1999 and 2018. Main outcomes are safety-related labelling changes up to 31 December 2019. We defined any safety-related labelling changes as postmarketing safety events (PMSE). Safety-related withdrawals, suspensions, and boxed warnings were further categorised as severe postmarketing safety events (SPSE). Outcome measurements include frequencies of PMSE, SPSE, and association between approval factors and the occurrence of safety events. Results Amongst the 214 drugs identified with orphan drug designation (25.7% biologics), 83.6% were approved through at least one expedited programme, and 29.4% were approved with boxed warnings. During a median follow-up of 6.74 years since approval, 69.2% and 14.5% of the analysed orphan drugs had PMSE and SPSE, respectively. Safety-related withdrawal (0%, 0/214), suspended marketing (0.46%, 1/214) and new boxed warnings are uncommon (3.7%, 8/214). The safety-related labelling changes were more frequent in the drugs approved with boxed warnings [Incidence rate ratio (IRR): 1.95 (1.02–3.73)] and approved for long-term use [IRR: 2.76 (1.52–5.00)]. Conclusions and Relevance In this long-term postmarketing analysis, approximately 70% of FDA-approved orphan drugs had safety-related labelling changes although severe safety events were rare. While maintaining early access to orphan drugs, the drug regulatory body has taken timely regulatory action with postmarketing surveillance to ensure patient safety." @default.
• W4205249249 created "2022-01-25" @default.
• W4205249249 creator A5013298747 @default.
• W4205249249 creator A5016334509 @default.
• W4205249249 creator A5018381716 @default.
• W4205249249 creator A5022905161 @default.
• W4205249249 creator A5029284712 @default.
• W4205249249 creator A5029330906 @default.
• W4205249249 creator A5030112494 @default.
• W4205249249 creator A5062694116 @default.
• W4205249249 creator A5064479308 @default.
• W4205249249 creator A5066384550 @default.
• W4205249249 creator A5072041259 @default.
• W4205249249 date "2022-01-04" @default.
• W4205249249 modified "2023-10-16" @default.
• W4205249249 title "Postmarketing safety of orphan drugs: a longitudinal analysis of the US Food and Drug Administration database between 1999 and 2018" @default.
• W4205249249 cites W1489821983 @default.
• W4205249249 cites W1930543669 @default.
• W4205249249 cites W2030883675 @default.
• W4205249249 cites W2103044501 @default.
• W4205249249 cites W2108093005 @default.
• W4205249249 cites W2118894060 @default.
• W4205249249 cites W2157584018 @default.
• W4205249249 cites W2212454316 @default.
• W4205249249 cites W2507351290 @default.
• W4205249249 cites W2602824263 @default.
• W4205249249 cites W2612600212 @default.
• W4205249249 cites W2777434883 @default.
• W4205249249 cites W2793007221 @default.
• W4205249249 cites W2885739590 @default.
• W4205249249 cites W2909995413 @default.
• W4205249249 cites W2971814634 @default.
• W4205249249 cites W2982466744 @default.
• W4205249249 cites W2996690001 @default.
• W4205249249 cites W3017195870 @default.
• W4205249249 cites W3035992474 @default.
• W4205249249 cites W4210779690 @default.
• W4205249249 doi "https://doi.org/10.1186/s13023-021-02166-9" @default.
• W4205249249 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/34983612" @default.
• W4205249249 hasPublicationYear "2022" @default.
• W4205249249 type Work @default.
• W4205249249 citedByCount "2" @default.
• W4205249249 countsByYear W42052492492023 @default.
• W4205249249 crossrefType "journal-article" @default.
• W4205249249 hasAuthorship W4205249249A5013298747 @default.
• W4205249249 hasAuthorship W4205249249A5016334509 @default.
• W4205249249 hasAuthorship W4205249249A5018381716 @default.
• W4205249249 hasAuthorship W4205249249A5022905161 @default.
• W4205249249 hasAuthorship W4205249249A5029284712 @default.
• W4205249249 hasAuthorship W4205249249A5029330906 @default.
• W4205249249 hasAuthorship W4205249249A5030112494 @default.
• W4205249249 hasAuthorship W4205249249A5062694116 @default.
• W4205249249 hasAuthorship W4205249249A5064479308 @default.
• W4205249249 hasAuthorship W4205249249A5066384550 @default.
• W4205249249 hasAuthorship W4205249249A5072041259 @default.
• W4205249249 hasBestOaLocation W42052492491 @default.
• W4205249249 hasConcept C197636746 @default.
• W4205249249 hasConcept C197934379 @default.
• W4205249249 hasConcept C2780035454 @default.
• W4205249249 hasConcept C3018359032 @default.
• W4205249249 hasConcept C3018890749 @default.
• W4205249249 hasConcept C57658597 @default.
• W4205249249 hasConcept C60644358 @default.
• W4205249249 hasConcept C71924100 @default.
• W4205249249 hasConcept C75480439 @default.
• W4205249249 hasConcept C86803240 @default.
• W4205249249 hasConcept C98274493 @default.
• W4205249249 hasConceptScore W4205249249C197636746 @default.
• W4205249249 hasConceptScore W4205249249C197934379 @default.
• W4205249249 hasConceptScore W4205249249C2780035454 @default.
• W4205249249 hasConceptScore W4205249249C3018359032 @default.
• W4205249249 hasConceptScore W4205249249C3018890749 @default.
• W4205249249 hasConceptScore W4205249249C57658597 @default.
• W4205249249 hasConceptScore W4205249249C60644358 @default.
• W4205249249 hasConceptScore W4205249249C71924100 @default.
• W4205249249 hasConceptScore W4205249249C75480439 @default.
• W4205249249 hasConceptScore W4205249249C86803240 @default.
• W4205249249 hasConceptScore W4205249249C98274493 @default.
• W4205249249 hasIssue "1" @default.
• W4205249249 hasLocation W42052492491 @default.
• W4205249249 hasLocation W42052492492 @default.
• W4205249249 hasLocation W42052492493 @default.
• W4205249249 hasLocation W42052492494 @default.
• W4205249249 hasLocation W42052492495 @default.
• W4205249249 hasLocation W42052492496 @default.
• W4205249249 hasLocation W42052492497 @default.
• W4205249249 hasLocation W42052492498 @default.
• W4205249249 hasOpenAccess W4205249249 @default.
• W4205249249 hasPrimaryLocation W42052492491 @default.
• W4205249249 hasRelatedWork W1777403110 @default.
• W4205249249 hasRelatedWork W1995674909 @default.
• W4205249249 hasRelatedWork W2011444787 @default.
• W4205249249 hasRelatedWork W2065911667 @default.
• W4205249249 hasRelatedWork W2400214312 @default.
• W4205249249 hasRelatedWork W2890735111 @default.
• W4205249249 hasRelatedWork W2981971963 @default.
• W4205249249 hasRelatedWork W4205249249 @default.
• W4205249249 hasRelatedWork W4300283912 @default.
• W4205249249 hasRelatedWork W2398119912 @default.
• W4205249249 hasVolume "17" @default.

• next