FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2098724822> ?p ?o ?g. }

• W2098724822 endingPage "761" @default.
• W2098724822 startingPage "757" @default.
• W2098724822 abstract "BackgroundThe designation of a disease as “rare” is associated with some substantial benefits for companies involved in new drug development, including expedited review by regulatory authorities and relaxed criteria for reimbursement. How “rare disease” is defined therefore has major financial implications, both for pharmaceutical companies and for insurers or public drug reimbursement programs. All existing definitions are based, somewhat arbitrarily, on disease incidence or prevalence.ObjectivesWhat is proposed here is a functional definition of rare based on an assessment of the feasibility of measuring the efficacy of a new treatment in conventional randomized controlled trials, to inform regulatory authorities and funding agencies charged with assessing new therapies being considered for public funding.MethodsIt involves a five-step process, involving significant negotiations between patient advocacy groups, pharmaceutical companies, physicians, and public drug reimbursement programs, designed to establish the feasibility of carrying out a randomized controlled trial with sufficient statistical power to show a clinically significant treatment effect.Results and ConclusionsThe steps are as follows: 1) identification of a specific disease, including appropriate genetic definition; 2) identification of clinically relevant outcomes to evaluate efficacy; 3) establishment of the inherent variability of measurements of clinically relevant outcomes; 4) calculation of the sample size required to assess the efficacy of a new treatment with acceptable statistical power; and 5) estimation of the difficulty of recruiting an adequate sample size given the estimated prevalence or incidence of the disorder in the population and the inclusion criteria to be used." @default.
• W2098724822 created "2016-06-24" @default.
• W2098724822 creator A5009242761 @default.
• W2098724822 creator A5035665285 @default.
• W2098724822 creator A5042123586 @default.
• W2098724822 creator A5046694154 @default.
• W2098724822 creator A5068505292 @default.
• W2098724822 date "2014-12-01" @default.
• W2098724822 modified "2023-10-18" @default.
• W2098724822 title "Toward a Functional Definition of a “Rare Disease” for Regulatory Authorities and Funding Agencies" @default.
• W2098724822 cites W1981632300 @default.
• W2098724822 cites W2003470122 @default.
• W2098724822 cites W2011363763 @default.
• W2098724822 cites W2034022408 @default.
• W2098724822 cites W2034981978 @default.
• W2098724822 cites W2035802792 @default.
• W2098724822 cites W2040077351 @default.
• W2098724822 cites W2042609729 @default.
• W2098724822 cites W2056048699 @default.
• W2098724822 cites W2063326100 @default.
• W2098724822 cites W2063752042 @default.
• W2098724822 cites W2093589847 @default.
• W2098724822 cites W2093722439 @default.
• W2098724822 cites W2097672482 @default.
• W2098724822 cites W2100199292 @default.
• W2098724822 cites W2114577841 @default.
• W2098724822 cites W2117030035 @default.
• W2098724822 cites W2117239948 @default.
• W2098724822 cites W2136033660 @default.
• W2098724822 cites W2139517025 @default.
• W2098724822 cites W2165663095 @default.
• W2098724822 cites W1984612750 @default.
• W2098724822 doi "https://doi.org/10.1016/j.jval.2014.08.2672" @default.
• W2098724822 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/25498770" @default.
• W2098724822 hasPublicationYear "2014" @default.
• W2098724822 type Work @default.
• W2098724822 sameAs 2098724822 @default.
• W2098724822 citedByCount "19" @default.
• W2098724822 countsByYear W20987248222015 @default.
• W2098724822 countsByYear W20987248222016 @default.
• W2098724822 countsByYear W20987248222017 @default.
• W2098724822 countsByYear W20987248222018 @default.
• W2098724822 countsByYear W20987248222019 @default.
• W2098724822 countsByYear W20987248222020 @default.
• W2098724822 countsByYear W20987248222021 @default.
• W2098724822 countsByYear W20987248222022 @default.
• W2098724822 countsByYear W20987248222023 @default.
• W2098724822 crossrefType "journal-article" @default.
• W2098724822 hasAuthorship W2098724822A5009242761 @default.
• W2098724822 hasAuthorship W2098724822A5035665285 @default.
• W2098724822 hasAuthorship W2098724822A5042123586 @default.
• W2098724822 hasAuthorship W2098724822A5046694154 @default.
• W2098724822 hasAuthorship W2098724822A5068505292 @default.
• W2098724822 hasBestOaLocation W20987248221 @default.
• W2098724822 hasConcept C100001284 @default.
• W2098724822 hasConcept C105795698 @default.
• W2098724822 hasConcept C116834253 @default.
• W2098724822 hasConcept C126322002 @default.
• W2098724822 hasConcept C129848803 @default.
• W2098724822 hasConcept C141071460 @default.
• W2098724822 hasConcept C144133560 @default.
• W2098724822 hasConcept C160735492 @default.
• W2098724822 hasConcept C162118730 @default.
• W2098724822 hasConcept C162324750 @default.
• W2098724822 hasConcept C168563851 @default.
• W2098724822 hasConcept C2779134260 @default.
• W2098724822 hasConcept C2779703844 @default.
• W2098724822 hasConcept C2908647359 @default.
• W2098724822 hasConcept C33923547 @default.
• W2098724822 hasConcept C50522688 @default.
• W2098724822 hasConcept C59822182 @default.
• W2098724822 hasConcept C60644358 @default.
• W2098724822 hasConcept C71924100 @default.
• W2098724822 hasConcept C75480439 @default.
• W2098724822 hasConcept C86803240 @default.
• W2098724822 hasConcept C99454951 @default.
• W2098724822 hasConceptScore W2098724822C100001284 @default.
• W2098724822 hasConceptScore W2098724822C105795698 @default.
• W2098724822 hasConceptScore W2098724822C116834253 @default.
• W2098724822 hasConceptScore W2098724822C126322002 @default.
• W2098724822 hasConceptScore W2098724822C129848803 @default.
• W2098724822 hasConceptScore W2098724822C141071460 @default.
• W2098724822 hasConceptScore W2098724822C144133560 @default.
• W2098724822 hasConceptScore W2098724822C160735492 @default.
• W2098724822 hasConceptScore W2098724822C162118730 @default.
• W2098724822 hasConceptScore W2098724822C162324750 @default.
• W2098724822 hasConceptScore W2098724822C168563851 @default.
• W2098724822 hasConceptScore W2098724822C2779134260 @default.
• W2098724822 hasConceptScore W2098724822C2779703844 @default.
• W2098724822 hasConceptScore W2098724822C2908647359 @default.
• W2098724822 hasConceptScore W2098724822C33923547 @default.
• W2098724822 hasConceptScore W2098724822C50522688 @default.
• W2098724822 hasConceptScore W2098724822C59822182 @default.
• W2098724822 hasConceptScore W2098724822C60644358 @default.
• W2098724822 hasConceptScore W2098724822C71924100 @default.
• W2098724822 hasConceptScore W2098724822C75480439 @default.
• W2098724822 hasConceptScore W2098724822C86803240 @default.
• W2098724822 hasConceptScore W2098724822C99454951 @default.

• next