data mining methods enable the discovery of new or the repurposing of previously known pharmaceutical compounds in the development of treatments for new indications.
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Tissue Chip for Drug Screening program and Consortium
Published by IRDiRC
BiomarkerDrug DiscoveryDrug Screening ProgrammeHigh Throughput ScreeningLibraryNCATSResearch and Drug DevelopmentTissue ChipNCATS, in collaboration with other NIH Institutes and Centers and the Food and Drug Administration (FDA), is leading the Tissue Chip for Drug Screening program to develop human tissue chips that accurately model the structure and function of human organs — such as the lungs, liver and heart — to help predict drug Safety in humans more rapidly and effectively
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Core Outcome Measures in Effectiveness Trials
Published by IRDiRC
Clinical DevelopmentClinical Trial DesignEffectiveness TrialEndpoint COMETOutcome MeasureResearch and Drug DevelopmentThe COMET (Core Outcome Measures in Effectiveness Trials) Initiative brings together people interested in the development and application of agreed standardised sets of outcomes, known as ‘core outcome sets’ (COS). These sets represent the minimum that should be measured and reported in all clinical trials of a specific condition, and are also suitable for use in clinical audit or research other than randomised trials.
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EURORDIS Open Academy
Published by IRDiRC
Open AcademyOrphan DrugPatient InformationResearch and Drug DevelopmentToolkit for EURORDISTraining ProgrammeThe training aims to provide participants with the knowledge and skills needed to become experts in medicines research and development. Over 20 EURORDIS Summer School trainers provide the training each year.
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IRDiRC Recognized Resources
Published by IRDiRC
Consulting & Advice IRDiRC Recognised ResourceOrphan DrugOrphan Drug Development GuidelineResearch and Drug DevelopmentTranlational MedicineUse CaseIRDiRC Recognized Resources is a quality indicator, which is expected to help accelerate the pace of translating discoveries into clinical applications. It therefore lists a number of resources important for Orphan Drug Development.
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Coding of Rare Diseases: Orphanet nomenclature
Published by IRDiRC
Coding of Rare DiseaseDatabase or Tool OrphanetResearch and Drug Development
Patient surveys / Patient Preferences studies / Ethnographic research
Published by IRDiRC
Clinical DevelopmentEndpoInternationalPatientEthnographic ResearchPatient Preference StudyQualitative AssessmentResearch and Drug DevelopmentQualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions.
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Development and use of Patient-Centered Outcome Measures
Published by IRDiRC
BiomarkerClinical DevelopmentEndpointPatient-Centred Outcome MeasureResearch and Drug DevelopmentPatient-Centred Outcomes Measures (PCOMs) are questionnaires that ‘directly’ quantify the impact of a disease and treatment on health outcomes that matter to patients (as identified or affirmed by patients themselves, or their caregivers)
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New-born screening programs
Published by IRDiRC
Genetic ConditionNewborn Sreening ProgrammePatient InformationRare DiseaseResearch and Drug DevelopmentThe purpose of NBS is to detect potentially fatal or disabling conditions in newborns as early as possible and possibly before onset of symptoms. Such detection allows the early treatment which may significantly modify the natural history of the disease and potentially prevent developmental delays, physical disabilities and eventually death.
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Initiatives for undiagnosed diseases
Published by IRDiRC
Genetic ConditionGenotype and Phenotype MatchmakingPatient InformationResearch and Drug DevelopmentUndiagnosed Disease ProgrammeThe purposes of Undiagnosed Diseases Programs (UDPs) are to provide patients with an unknown genetic condition a diagnosis and to find the correlation between genotype and phenotype; to share globally the information to facilitate the diagnosis through a matchmaking for finding possible second cases
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