FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W3100813408> ?p ?o ?g. }

• W3100813408 endingPage "285" @default.
• W3100813408 startingPage "277" @default.
• W3100813408 abstract "Background: Clinical research and drug development in orphan diseases are challenging, since large-scale randomized studies are difficult to conduct. Formally synthesizing the evidence is therefore of great value, yet this is rarely done in the drug-approval process. Phase III designs that make better use of phase II data can facilitate drug development in orphan diseases. Methods: A Bayesian meta-analytic approach is used to inform the phase III study with phase II data. It is particularly attractive, since uncertainty of between-trial heterogeneity can be dealt with probabilistically, which is critical if the number of studies is small. Furthermore, it allows quantifying and discounting the phase II data through the predictive distribution relevant for phase III. A phase III design is proposed which uses the phase II data and considers approval based on a phase III interim analysis. The design is illustrated with a non-inferiority case study from a Food and Drug Administration approval in herpetic keratitis (an orphan disease). Design operating characteristics are compared to those of a traditional design, which ignores the phase II data. Results: An analysis of the phase II data reveals good but insufficient evidence for non-inferiority, highlighting the need for a phase III study. For the phase III study supported by phase II data, the interim analysis is based on half of the patients. For this design, the meta-analytic interim results are conclusive and would justify approval. In contrast, based on the phase III data only, interim results are inconclusive and require further evidence. Conclusion: To accelerate drug development for orphan diseases, innovative study designs and appropriate methodology are needed. Taking advantage of randomized phase II data when analyzing phase III studies looks promising because the evidence from phase II supports informed decision-making. The implementation of the Bayesian design is straightforward with public software such as R." @default.
• W3100813408 created "2020-11-23" @default.
• W3100813408 creator A5007871238 @default.
• W3100813408 creator A5039141235 @default.
• W3100813408 creator A5057325416 @default.
• W3100813408 creator A5069688964 @default.
• W3100813408 date "2017-04-07" @default.
• W3100813408 modified "2023-10-14" @default.
• W3100813408 title "Using phase II data for the analysis of phase III studies: An application in rare diseases" @default.
• W3100813408 cites W1536497620 @default.
• W3100813408 cites W1741011578 @default.
• W3100813408 cites W1756081881 @default.
• W3100813408 cites W1857542727 @default.
• W3100813408 cites W1972543205 @default.
• W3100813408 cites W1988432737 @default.
• W3100813408 cites W1997530226 @default.
• W3100813408 cites W2010622913 @default.
• W3100813408 cites W2010995130 @default.
• W3100813408 cites W2021609141 @default.
• W3100813408 cites W2041098432 @default.
• W3100813408 cites W2041685717 @default.
• W3100813408 cites W2045573131 @default.
• W3100813408 cites W2046899821 @default.
• W3100813408 cites W2100950823 @default.
• W3100813408 cites W2107836148 @default.
• W3100813408 cites W2134318552 @default.
• W3100813408 cites W2143109076 @default.
• W3100813408 cites W2145695460 @default.
• W3100813408 cites W2149219496 @default.
• W3100813408 cites W2149965232 @default.
• W3100813408 cites W2150847344 @default.
• W3100813408 cites W2166992787 @default.
• W3100813408 cites W2271307894 @default.
• W3100813408 cites W2345227881 @default.
• W3100813408 cites W2400374725 @default.
• W3100813408 cites W2438033049 @default.
• W3100813408 cites W2491633544 @default.
• W3100813408 cites W2547923310 @default.
• W3100813408 cites W2900300149 @default.
• W3100813408 cites W3101259738 @default.
• W3100813408 cites W3103359439 @default.
• W3100813408 doi "https://doi.org/10.1177/1740774517699409" @default.
• W3100813408 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/5833035" @default.
• W3100813408 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/28387537" @default.
• W3100813408 hasPublicationYear "2017" @default.
• W3100813408 type Work @default.
• W3100813408 sameAs 3100813408 @default.
• W3100813408 citedByCount "12" @default.
• W3100813408 countsByYear W31008134082017 @default.
• W3100813408 countsByYear W31008134082018 @default.
• W3100813408 countsByYear W31008134082019 @default.
• W3100813408 countsByYear W31008134082020 @default.
• W3100813408 countsByYear W31008134082021 @default.
• W3100813408 countsByYear W31008134082022 @default.
• W3100813408 crossrefType "journal-article" @default.
• W3100813408 hasAuthorship W3100813408A5007871238 @default.
• W3100813408 hasAuthorship W3100813408A5039141235 @default.
• W3100813408 hasAuthorship W3100813408A5057325416 @default.
• W3100813408 hasAuthorship W3100813408A5069688964 @default.
• W3100813408 hasBestOaLocation W31008134081 @default.
• W3100813408 hasConcept C105795698 @default.
• W3100813408 hasConcept C126322002 @default.
• W3100813408 hasConcept C148482608 @default.
• W3100813408 hasConcept C166957645 @default.
• W3100813408 hasConcept C178790620 @default.
• W3100813408 hasConcept C185592680 @default.
• W3100813408 hasConcept C205649164 @default.
• W3100813408 hasConcept C2776957806 @default.
• W3100813408 hasConcept C2779318504 @default.
• W3100813408 hasConcept C2780035454 @default.
• W3100813408 hasConcept C33923547 @default.
• W3100813408 hasConcept C41008148 @default.
• W3100813408 hasConcept C44280652 @default.
• W3100813408 hasConcept C535046627 @default.
• W3100813408 hasConcept C61943457 @default.
• W3100813408 hasConcept C64903051 @default.
• W3100813408 hasConcept C71924100 @default.
• W3100813408 hasConcept C98274493 @default.
• W3100813408 hasConceptScore W3100813408C105795698 @default.
• W3100813408 hasConceptScore W3100813408C126322002 @default.
• W3100813408 hasConceptScore W3100813408C148482608 @default.
• W3100813408 hasConceptScore W3100813408C166957645 @default.
• W3100813408 hasConceptScore W3100813408C178790620 @default.
• W3100813408 hasConceptScore W3100813408C185592680 @default.
• W3100813408 hasConceptScore W3100813408C205649164 @default.
• W3100813408 hasConceptScore W3100813408C2776957806 @default.
• W3100813408 hasConceptScore W3100813408C2779318504 @default.
• W3100813408 hasConceptScore W3100813408C2780035454 @default.
• W3100813408 hasConceptScore W3100813408C33923547 @default.
• W3100813408 hasConceptScore W3100813408C41008148 @default.
• W3100813408 hasConceptScore W3100813408C44280652 @default.
• W3100813408 hasConceptScore W3100813408C535046627 @default.
• W3100813408 hasConceptScore W3100813408C61943457 @default.
• W3100813408 hasConceptScore W3100813408C64903051 @default.
• W3100813408 hasConceptScore W3100813408C71924100 @default.
• W3100813408 hasConceptScore W3100813408C98274493 @default.
• W3100813408 hasIssue "3" @default.
• W3100813408 hasLocation W31008134081 @default.

• next