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FAIRGuidelinePatient RegistryRare DiseaseResearch and Drug DevelopmentLack 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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Clinical DevelopmentClinical TrialEndpointOutcome MeasurePatient EngagementResearch and Drug DevelopmentTrail EndpoInternationalSelectionGuidance on the pivotal clinical trials outcomes measures .The important of the patient’s feedback. Academics can then assess whether there is an outcome measure available that can measure this – if not, outcome measure(s) may need to be developed.
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EurordisPatient EngagementPatient InformationPatient OrganisationPatient OrganisationsResearch and Drug DevelopmentEURORDIS as first poInternationalof contact; the patient organization of the specific disease
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DatabasePatient RegistryRegistry for Rare DiseaseResearch and Drug DevelopmentRegistries collect information on patients afflicted by a particular disease or group of diseases. By combining data on as many patients as possible, at the regional, national, European or global level, the potency of the data increases exponentially
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Natural HistoryPatient DataResearch and Drug DevelopmentA Natural History is the understanding of a disease progression throughout the lifespan of a patient. The collection of data from a significant number of patients affected by diverse phenotypes inside the same condition contribute to describe the history of a disease. It is a major issue in Rare Diseases research.
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Bio-Analytical AssayBioassayDrug DiscoveryResearch and Drug DevelopmentMeasurement of drug concentrations in biological matrices (such as serum, plasma, blood, urine, and saliva) is an important aspect of drug development. These methods provide data to support the Safety and effectiveness of drugs, which may be needed to support applications for new drugs in regulatory pathways
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Database or ToolOrphan Drug Development GuideResearch and Drug DevelopmentThese guidelines include tools, incentives, resources and practices for developing traditional and innovative drugs/therapies for rare diseases and how to best use them. It can be used by academic, non-profit organizations, small and larger (innovative) biotechs and patient-driven drug developers.
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Clinical DevelopmentEarly Access SupportFDA Regulatory AdvicePatient AdvicePatient Affairs StaffPatient EngagementPatient SupportRegulatory AffairsResearch and Drug DevelopmentAt the FDA, the Patient Affairs Staff (PAS) is devoted to supporting cross-cutting patient engagement activities across the FDA. Its mission is to coordinate and support patient engagement activities across FDA to facilitate awareness and collaboration with patients, their advocates and the FDA
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Biomarker DevelopmentClinical DevelopmentEndpointEndpoInternationalBiomarkerOrphan DrugRegulatory SciencesResearch and Drug DevelopmentBiomarkers can be utilized as primary surrogate endpoInternationalas a basis for accelerated approval (US), and as secondary or exploratory endpoints for drug mechanism of action or aspects of Safety monitoring.
View this resource Bookmark this resourceDecentralized trials aim to improve patient access to trials by enabling patient follow-up from home or community care, increase the participation of more diverse populations, and enhancing data collection by combining the adoption of digital endpoints and telemedicine as applied to trials, decentralized trials aim
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