A companion diagnostic is a medical device, often an in vitro device, which provides information that is essential for the safe and effective use of a corresponding drug or biological product.
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FAIR principle for data use
Published by IRDiRC
Research and Drug DevelopmentRare DiseasePatient RegistryFAIRGuidelineLack of ultimate data use in rare disease created many silos slowing down development. The FAIR is coming to bridge this gap by proving essential guidelines for optimal data use.
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Initiatives for undiagnosed diseases
Published by IRDiRC
Research and Drug DevelopmentPatient InformationUndiagnosed Disease ProgrammeGenetic ConditionGenotype and Phenotype MatchmakingThe 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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New-born screening programs
Published by IRDiRC
Research and Drug DevelopmentRare DiseasePatient InformationGenetic ConditionNewborn Sreening ProgrammeThe 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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Target Patient Value Profile
Published by IRDiRC
Research and Drug DevelopmentTarget Patient Value ProfileDrug DescriptionPatient-Centric InformationA document outlining the goals, profile and potential benefit of a specific product, addressing relevant current and future patient needs in a differentiated way. It provides accurate, up-to-date information describing the expected benefit for patients and describe the drug.
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TREAT-NMD Advisory Committee for Therapeutics
Published by IRDiRC
Advisory Committee for TherapeuticsTREAT-NMDEstablished in 2009, the TREAT-NMD Advisory Committee for Therapeutics (TACT) is a unique multi-disciplinary international group of internationally recognized academic and industry drug development experts as well as representatives of patient foundations and regulatory experts
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Joint EMA-FDA Scientific Advice
Published by IRDiRC
Regulatory AffairsEarly Access SupportScientific AdviceJoint EMA-FDA Scientific AdviceThe European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services have a program to provide parallel scientific advice (PSA) to sponsors. The goal of the PSA program is to provide a mechanism for EMA assessors and FDA reviewers to concurrently exchange with sponsors their views on scientific issues during the development phase of new medicinal products (i.e., new human drugs and Biologics).
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Feasibility-Patient engagement in trial design and feasibility
Published by IRDiRC
Research and Drug DevelopmentPatient EngagementPatient InformationPatient OrganisationEurordisPatient OrganisationsEURORDIS as first point of contact; the patient organization of the specific disease
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Initiatives for undiagnosed diseases
Published by IRDiRC
Research and Drug DevelopmentPatient InformationUndiagnosed Disease ProgrammeGenetic ConditionGenotype and Phenotype MatchmakingThe 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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New-born screening programs
Published by IRDiRC
Research and Drug DevelopmentRare DiseasePatient InformationGenetic ConditionNewborn Sreening ProgrammeThe 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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