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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EMA Innovation Task Force
Published by IRDiRC
Regulatory AffairsEarly Access SupportScientific AdviceEMA Innovation Task ForceEarly Stage AdviceThe ITF is a multidisciplinary EMA group that includes scientific, regulatory and legal competences. It was set up to ensure coordination across the Agency and to provide a forum for early dialogue with applicants on innovative aspects in medicines development. Area of interest: innovative therapeutics and techniques, innovative development methods, borderline products.
From the first request by the applicant, the procedure lasts approximately 8-10 weeks (ITF Secretariats confirms eligibility criteria approx. within 2 weeks; when confirmed, then approx. 2 weeks about experts’ involvement + meeting date after 4-6 weeks).
European Orphan Drug Designation
Published by IRDiRC
Regulatory AffairsEarly Access SupportOrphan Drug DesignationEU orphan legislation offers important incentives to encourage the development of medicinal products for rare diseases and the success of the legislation has been demonstrate
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European Patients’ Academy (EUPATI) toolbox
Published by IRDiRC
Research and Drug DevelopmentOrphan DrugPatient InformationToolkit for PatientEUPATIToolboxPatient TrainingThe EUPATI Academy is the only in-depth training program for patient experts so far in EU that lasts 14months.
It includes an on online training program supported with two weeks of F2F training. The Academy is still operated by EPF, providing high quality training in drug development to patient from EMEA.
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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Regulatory and Ethics Toolkit, ICF guidelines
Published by IRDiRCECRINGlobal Alliance for Genomic and Health (GA4GH)
Clinical Trials ToolkitGA4GH and IRDiRC have developed model consent clauses for rare diseases research, in order to improve data interoperability, to meet the informational needs of participants, and to ensure proper ethical and legal use of data sources and participants’ overall protection
View this resource Bookmark this resourceProcedures set up by many (but not all) National Member State to offer scientific advice to developers of new medicines. The procedures can be similar but often less formal than for CHMP scientific advice/protocol assistance at the EMA; they may include written advice and/or face-to-face meetings. The procedures are not limited only to orphan product development and may vary between Member States.
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IRDiRC Building block_ Alternative designs for small population clinical trials
Clinical Trials Toolkit
General recommendations to select the most efficient study design for each medical condition or trial and on potential adaptations of conventional designs to the low sample size scenario
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