FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W4206431395> ?p ?o ?g. }

• W4206431395 endingPage "e057744" @default.
• W4206431395 startingPage "e057744" @default.
• W4206431395 abstract "Objectives Drug and biological products that treat rare, serious or life-threatening conditions can receive US Food and Drug Administration (FDA) orphan designation and expedited programme designations (accelerated approval, breakthrough therapy, fast track or priority review) meant to incentivise development. Timely recommendations from guidance documents may encourage more rapid and appropriate use and access to these medicines for serious conditions. We sought to determine time between FDA approval and inclusion in guidance documents for non-oncological orphan products overall and by number and type of expedited programme designations. Design and setting Retrospective survival analysis of non-oncological orphan products with ≥1 expedited designation approved since 1992. In June 2020, PubMed, Turning Research into Practice and Guideline Central databases were searched to identify guidance documents influencing US practice that included each product. Main outcomes and measures The primary outcome was time to guidance inclusion, defined as any recommendation on use provided within the recommendation framework used by the guidance document. Results Among 135 included non-oncological orphan products, 97.0% (n=131) were designated with priority review, 49.6% (n=67) fast track, 16.3% (n=22) breakthrough therapy and 14.1% (n=19) accelerated approval. Sixty per cent of products (n=81) received ≥2 designations. Overall, 74.1% (n=100) were included in a guidance document. The median time to inclusion was 2.87 years (IQR 2.21–4.18) for the entire cohort. In survival analyses, guidance inclusion was more likely to occur earlier for products with ≥2 designations (HR, 1.84; 95% CI 1.21 to 2.79) and for those with fast-track designation compared with priority review (HR 1.40; 95% CI 1.02 to 2.0). Of 35 products not included in a guidance document, 54.3% (n=19) were approved in 2018 or later. Conclusions Among non-oncological orphan products with priority designations, nearly 3 years had passed between FDA approval and inclusion in any guidance document. These findings suggest that despite efforts to expedite availability, appropriate access to these treatments may be delayed because of the lack of timely guidance on their use in clinical practice." @default.
• W4206431395 created "2022-01-25" @default.
• W4206431395 creator A5000661806 @default.
• W4206431395 creator A5020189738 @default.
• W4206431395 creator A5060786197 @default.
• W4206431395 creator A5064599368 @default.
• W4206431395 date "2021-12-01" @default.
• W4206431395 modified "2023-10-06" @default.
• W4206431395 title "Time to inclusion in clinical guidance documents for non-oncological orphan drugs and biologics with expedited FDA designations: a retrospective survival analysis" @default.
• W4206431395 cites W1533447420 @default.
• W4206431395 cites W1579507411 @default.
• W4206431395 cites W1788182075 @default.
• W4206431395 cites W1938547050 @default.
• W4206431395 cites W2109314733 @default.
• W4206431395 cites W2554491985 @default.
• W4206431395 cites W2619545114 @default.
• W4206431395 cites W2623751563 @default.
• W4206431395 cites W2750636690 @default.
• W4206431395 cites W2758111666 @default.
• W4206431395 cites W2803429027 @default.
• W4206431395 cites W2861428399 @default.
• W4206431395 cites W2884486468 @default.
• W4206431395 cites W2892049139 @default.
• W4206431395 cites W2899026268 @default.
• W4206431395 cites W2901313771 @default.
• W4206431395 cites W3015792696 @default.
• W4206431395 cites W3128444525 @default.
• W4206431395 cites W3130309548 @default.
• W4206431395 cites W3209231238 @default.
• W4206431395 doi "https://doi.org/10.1136/bmjopen-2021-057744" @default.
• W4206431395 hasPublicationYear "2021" @default.
• W4206431395 type Work @default.
• W4206431395 citedByCount "2" @default.
• W4206431395 countsByYear W42064313952023 @default.
• W4206431395 crossrefType "journal-article" @default.
• W4206431395 hasAuthorship W4206431395A5000661806 @default.
• W4206431395 hasAuthorship W4206431395A5020189738 @default.
• W4206431395 hasAuthorship W4206431395A5060786197 @default.
• W4206431395 hasAuthorship W4206431395A5064599368 @default.
• W4206431395 hasBestOaLocation W42064313951 @default.
• W4206431395 hasConcept C107993555 @default.
• W4206431395 hasConcept C109359841 @default.
• W4206431395 hasConcept C141071460 @default.
• W4206431395 hasConcept C142724271 @default.
• W4206431395 hasConcept C144024400 @default.
• W4206431395 hasConcept C167135981 @default.
• W4206431395 hasConcept C187212893 @default.
• W4206431395 hasConcept C188313352 @default.
• W4206431395 hasConcept C204787440 @default.
• W4206431395 hasConcept C2777436895 @default.
• W4206431395 hasConcept C2780035454 @default.
• W4206431395 hasConcept C2780182762 @default.
• W4206431395 hasConcept C2992293740 @default.
• W4206431395 hasConcept C3018890749 @default.
• W4206431395 hasConcept C512399662 @default.
• W4206431395 hasConcept C545542383 @default.
• W4206431395 hasConcept C60644358 @default.
• W4206431395 hasConcept C71924100 @default.
• W4206431395 hasConcept C75480439 @default.
• W4206431395 hasConcept C86803240 @default.
• W4206431395 hasConcept C98274493 @default.
• W4206431395 hasConceptScore W4206431395C107993555 @default.
• W4206431395 hasConceptScore W4206431395C109359841 @default.
• W4206431395 hasConceptScore W4206431395C141071460 @default.
• W4206431395 hasConceptScore W4206431395C142724271 @default.
• W4206431395 hasConceptScore W4206431395C144024400 @default.
• W4206431395 hasConceptScore W4206431395C167135981 @default.
• W4206431395 hasConceptScore W4206431395C187212893 @default.
• W4206431395 hasConceptScore W4206431395C188313352 @default.
• W4206431395 hasConceptScore W4206431395C204787440 @default.
• W4206431395 hasConceptScore W4206431395C2777436895 @default.
• W4206431395 hasConceptScore W4206431395C2780035454 @default.
• W4206431395 hasConceptScore W4206431395C2780182762 @default.
• W4206431395 hasConceptScore W4206431395C2992293740 @default.
• W4206431395 hasConceptScore W4206431395C3018890749 @default.
• W4206431395 hasConceptScore W4206431395C512399662 @default.
• W4206431395 hasConceptScore W4206431395C545542383 @default.
• W4206431395 hasConceptScore W4206431395C60644358 @default.
• W4206431395 hasConceptScore W4206431395C71924100 @default.
• W4206431395 hasConceptScore W4206431395C75480439 @default.
• W4206431395 hasConceptScore W4206431395C86803240 @default.
• W4206431395 hasConceptScore W4206431395C98274493 @default.
• W4206431395 hasIssue "12" @default.
• W4206431395 hasLocation W42064313951 @default.
• W4206431395 hasLocation W42064313952 @default.
• W4206431395 hasOpenAccess W4206431395 @default.
• W4206431395 hasPrimaryLocation W42064313951 @default.
• W4206431395 hasRelatedWork W1481094626 @default.
• W4206431395 hasRelatedWork W1891615569 @default.
• W4206431395 hasRelatedWork W2035802792 @default.
• W4206431395 hasRelatedWork W2134589367 @default.
• W4206431395 hasRelatedWork W2208683947 @default.
• W4206431395 hasRelatedWork W2792201647 @default.
• W4206431395 hasRelatedWork W2966778919 @default.
• W4206431395 hasRelatedWork W4206431395 @default.
• W4206431395 hasRelatedWork W4225507164 @default.
• W4206431395 hasRelatedWork W4230240971 @default.
• W4206431395 hasVolume "11" @default.

• next