FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2788702966> ?p ?o ?g. }

• W2788702966 abstract "It has been suggested that ultra-rare diseases should be recognized as distinct from more prevalent rare diseases, but how drugs developed to treat ultra-rare diseases (DURDs) might be distinguished from drugs for 'other' rare diseases (DORDs) is not clear. We compared the characteristics of DURDs to DORDs from a health technology assessment (HTA) perspective in submissions made to the CADTH Common Drug Review. We defined a DURD as a drug used to treat a disease with a prevalence ≤ 1 patient per 100,000 people, a DORD as a drug used to treat a disease with a prevalence > 1 and ≤ 50 patients per 100,000 people. We assessed differences in the level and quantity of evidence supporting each HTA submission, the molecular basis of treatment agents, annual treatment cost per patient, type of reimbursement recommendation made by CADTH, and reasons for negative recommendations.We analyzed 14 DURD and 46 DORD submissions made between 2004 and 2016. Compared to DORDs, DURDs were more likely to be biologic drugs (OR = 6.06, 95%CI 1.25 to 38.58), to have been studied in uncontrolled clinical trials (OR = 23.11, 95%CI 2.23 to 1207.19), and to have a higher annual treatment cost per patient (median difference = CAN$243,787.75, 95%CI CAN$83,396 to CAN$329,050). Also, submissions for DURDs were associated with a less robust evidence base versus DORDs, as DURD submissions were less likely to include data from at least one double-blinded randomized controlled trial (OR = 0.13, 95%CI 0.02 to 0.70) and have smaller patient cohorts in clinical trials (median difference = -108, 95%CI -234 to -50). Furthermore, DURDs are less likely to receive a positive reimbursement recommendation (OR = 0.22, 95%CI 0.05 to 0.91), and low level of evidence was the major contributor for a negative recommendation.The results suggest that DURDs could be viewed as distinct category from an HTA perspective. Applying the same HTA decision-making framework to DURDs and DORDs might have contributed the higher rate of negative reimbursement recommendations made for DURDs. Recognition of DURDs as a distinct subgroup of DRDs by explicitly defining DURDs based on objective criteria may facilitate the implementation of HTA assessment process that accounts for the issues associated with DURD." @default.
• W2788702966 created "2018-03-06" @default.
• W2788702966 creator A5004540130 @default.
• W2788702966 creator A5020594263 @default.
• W2788702966 creator A5040377035 @default.
• W2788702966 creator A5051070613 @default.
• W2788702966 date "2018-02-01" @default.
• W2788702966 modified "2023-10-12" @default.
• W2788702966 title "Characteristics of drugs for ultra-rare diseases versus drugs for other rare diseases in HTA submissions made to the CADTH CDR" @default.
• W2788702966 cites W1579507411 @default.
• W2788702966 cites W1981652493 @default.
• W2788702966 cites W1999697554 @default.
• W2788702966 cites W2019310367 @default.
• W2788702966 cites W2038500552 @default.
• W2788702966 cites W2053894818 @default.
• W2788702966 cites W2065306083 @default.
• W2788702966 cites W2096145249 @default.
• W2788702966 cites W2145695460 @default.
• W2788702966 cites W2151716129 @default.
• W2788702966 cites W2158820614 @default.
• W2788702966 cites W2165663095 @default.
• W2788702966 cites W2557309514 @default.
• W2788702966 cites W2600539686 @default.
• W2788702966 doi "https://doi.org/10.1186/s13023-018-0762-1" @default.
• W2788702966 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/5793441" @default.
• W2788702966 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/29386040" @default.
• W2788702966 hasPublicationYear "2018" @default.
• W2788702966 type Work @default.
• W2788702966 sameAs 2788702966 @default.
• W2788702966 citedByCount "24" @default.
• W2788702966 countsByYear W27887029662018 @default.
• W2788702966 countsByYear W27887029662020 @default.
• W2788702966 countsByYear W27887029662021 @default.
• W2788702966 countsByYear W27887029662022 @default.
• W2788702966 countsByYear W27887029662023 @default.
• W2788702966 crossrefType "journal-article" @default.
• W2788702966 hasAuthorship W2788702966A5004540130 @default.
• W2788702966 hasAuthorship W2788702966A5020594263 @default.
• W2788702966 hasAuthorship W2788702966A5040377035 @default.
• W2788702966 hasAuthorship W2788702966A5051070613 @default.
• W2788702966 hasBestOaLocation W27887029661 @default.
• W2788702966 hasConcept C126322002 @default.
• W2788702966 hasConcept C160735492 @default.
• W2788702966 hasConcept C162324750 @default.
• W2788702966 hasConcept C168563851 @default.
• W2788702966 hasConcept C177713679 @default.
• W2788702966 hasConcept C187212893 @default.
• W2788702966 hasConcept C2779134260 @default.
• W2788702966 hasConcept C2779701055 @default.
• W2788702966 hasConcept C2779703844 @default.
• W2788702966 hasConcept C2780035454 @default.
• W2788702966 hasConcept C50522688 @default.
• W2788702966 hasConcept C535046627 @default.
• W2788702966 hasConcept C60644358 @default.
• W2788702966 hasConcept C71924100 @default.
• W2788702966 hasConcept C75480439 @default.
• W2788702966 hasConcept C86803240 @default.
• W2788702966 hasConcept C98274493 @default.
• W2788702966 hasConceptScore W2788702966C126322002 @default.
• W2788702966 hasConceptScore W2788702966C160735492 @default.
• W2788702966 hasConceptScore W2788702966C162324750 @default.
• W2788702966 hasConceptScore W2788702966C168563851 @default.
• W2788702966 hasConceptScore W2788702966C177713679 @default.
• W2788702966 hasConceptScore W2788702966C187212893 @default.
• W2788702966 hasConceptScore W2788702966C2779134260 @default.
• W2788702966 hasConceptScore W2788702966C2779701055 @default.
• W2788702966 hasConceptScore W2788702966C2779703844 @default.
• W2788702966 hasConceptScore W2788702966C2780035454 @default.
• W2788702966 hasConceptScore W2788702966C50522688 @default.
• W2788702966 hasConceptScore W2788702966C535046627 @default.
• W2788702966 hasConceptScore W2788702966C60644358 @default.
• W2788702966 hasConceptScore W2788702966C71924100 @default.
• W2788702966 hasConceptScore W2788702966C75480439 @default.
• W2788702966 hasConceptScore W2788702966C86803240 @default.
• W2788702966 hasConceptScore W2788702966C98274493 @default.
• W2788702966 hasIssue "1" @default.
• W2788702966 hasLocation W27887029661 @default.
• W2788702966 hasLocation W27887029662 @default.
• W2788702966 hasLocation W27887029663 @default.
• W2788702966 hasLocation W27887029664 @default.
• W2788702966 hasOpenAccess W2788702966 @default.
• W2788702966 hasPrimaryLocation W27887029661 @default.
• W2788702966 hasRelatedWork W1842590226 @default.
• W2788702966 hasRelatedWork W1993780631 @default.
• W2788702966 hasRelatedWork W2038375976 @default.
• W2788702966 hasRelatedWork W2274162763 @default.
• W2788702966 hasRelatedWork W2905957873 @default.
• W2788702966 hasRelatedWork W3031221386 @default.
• W2788702966 hasRelatedWork W3031628127 @default.
• W2788702966 hasRelatedWork W3105771742 @default.
• W2788702966 hasRelatedWork W4238728006 @default.
• W2788702966 hasRelatedWork W4283512297 @default.
• W2788702966 hasVolume "13" @default.
• W2788702966 isParatext "false" @default.
• W2788702966 isRetracted "false" @default.
• W2788702966 magId "2788702966" @default.
• W2788702966 workType "article" @default.