Matches in SemOpenAlex for { <https://semopenalex.org/work/W4286008596> ?p ?o ?g. }
Showing items 1 to 79 of
79
with 100 items per page.
- W4286008596 endingPage "410" @default.
- W4286008596 startingPage "407" @default.
- W4286008596 abstract "In the past few decades, the regulatory system has changed its approach to speed up the assessment and approval of drugs for the treatment of serious and orphan diseases. However, too early assessments may fail to provide solid evidence to define the therapeutic value of drugs and their fair price. As for rare diseases, patients' expectations amplify such criticalities due to the lack of therapeutic alternatives. The revision of the EU regulation on orphan medicinal products represents an opportunity to promote greater integration between the development and approval of drugs with their subsequent access. For this reason, we propose: 1) establishing a process for evaluating the comparative efficacy and quantification of the therapeutic benefit of a drug at a European level, while addressing incentives towards rarer diseases to support their development and marketing; 2) downsizing the impact of orphan drugs on general sustainability, considering that incentives supporting their development and marketing cannot transform them into new 'blockbusters'. In this perspective, a European procurement - initially intended for drugs for ultra-rare diseases - could improve access by all EU countries through simplifying the burdensome procedures for the pharmaceutical industry; 3) enhancing the contribution of post-authorization research from a public-private partnership perspective to steer the development of drugs already approved towards rarer pathologies or mutations of little commercial interest." @default.
- W4286008596 created "2022-07-21" @default.
- W4286008596 creator A5004497604 @default.
- W4286008596 creator A5054500534 @default.
- W4286008596 date "2022-07-20" @default.
- W4286008596 modified "2023-10-12" @default.
- W4286008596 title "[Harmonizing development, approval and access of drugs for rare diseases in Europe.]" @default.
- W4286008596 doi "https://doi.org/10.1701/3850.38333" @default.
- W4286008596 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/35852074" @default.
- W4286008596 hasPublicationYear "2022" @default.
- W4286008596 type Work @default.
- W4286008596 citedByCount "1" @default.
- W4286008596 crossrefType "journal-article" @default.
- W4286008596 hasAuthorship W4286008596A5004497604 @default.
- W4286008596 hasAuthorship W4286008596A5054500534 @default.
- W4286008596 hasConcept C100001284 @default.
- W4286008596 hasConcept C10138342 @default.
- W4286008596 hasConcept C108759981 @default.
- W4286008596 hasConcept C112930515 @default.
- W4286008596 hasConcept C144133560 @default.
- W4286008596 hasConcept C162324750 @default.
- W4286008596 hasConcept C162853370 @default.
- W4286008596 hasConcept C175444787 @default.
- W4286008596 hasConcept C201650216 @default.
- W4286008596 hasConcept C2777589142 @default.
- W4286008596 hasConcept C2779652045 @default.
- W4286008596 hasConcept C2780035454 @default.
- W4286008596 hasConcept C29122968 @default.
- W4286008596 hasConcept C38652104 @default.
- W4286008596 hasConcept C41008148 @default.
- W4286008596 hasConcept C60644358 @default.
- W4286008596 hasConcept C64903051 @default.
- W4286008596 hasConcept C71750763 @default.
- W4286008596 hasConcept C71924100 @default.
- W4286008596 hasConcept C75480439 @default.
- W4286008596 hasConcept C86803240 @default.
- W4286008596 hasConcept C98274493 @default.
- W4286008596 hasConceptScore W4286008596C100001284 @default.
- W4286008596 hasConceptScore W4286008596C10138342 @default.
- W4286008596 hasConceptScore W4286008596C108759981 @default.
- W4286008596 hasConceptScore W4286008596C112930515 @default.
- W4286008596 hasConceptScore W4286008596C144133560 @default.
- W4286008596 hasConceptScore W4286008596C162324750 @default.
- W4286008596 hasConceptScore W4286008596C162853370 @default.
- W4286008596 hasConceptScore W4286008596C175444787 @default.
- W4286008596 hasConceptScore W4286008596C201650216 @default.
- W4286008596 hasConceptScore W4286008596C2777589142 @default.
- W4286008596 hasConceptScore W4286008596C2779652045 @default.
- W4286008596 hasConceptScore W4286008596C2780035454 @default.
- W4286008596 hasConceptScore W4286008596C29122968 @default.
- W4286008596 hasConceptScore W4286008596C38652104 @default.
- W4286008596 hasConceptScore W4286008596C41008148 @default.
- W4286008596 hasConceptScore W4286008596C60644358 @default.
- W4286008596 hasConceptScore W4286008596C64903051 @default.
- W4286008596 hasConceptScore W4286008596C71750763 @default.
- W4286008596 hasConceptScore W4286008596C71924100 @default.
- W4286008596 hasConceptScore W4286008596C75480439 @default.
- W4286008596 hasConceptScore W4286008596C86803240 @default.
- W4286008596 hasConceptScore W4286008596C98274493 @default.
- W4286008596 hasIssue "7" @default.
- W4286008596 hasLocation W42860085961 @default.
- W4286008596 hasOpenAccess W4286008596 @default.
- W4286008596 hasPrimaryLocation W42860085961 @default.
- W4286008596 hasRelatedWork W10355314 @default.
- W4286008596 hasRelatedWork W10791266 @default.
- W4286008596 hasRelatedWork W12214839 @default.
- W4286008596 hasRelatedWork W13597315 @default.
- W4286008596 hasRelatedWork W13916586 @default.
- W4286008596 hasRelatedWork W14259521 @default.
- W4286008596 hasRelatedWork W14689180 @default.
- W4286008596 hasRelatedWork W704948 @default.
- W4286008596 hasRelatedWork W8148999 @default.
- W4286008596 hasRelatedWork W828189 @default.
- W4286008596 hasVolume "113" @default.
- W4286008596 isParatext "false" @default.
- W4286008596 isRetracted "false" @default.
- W4286008596 workType "article" @default.