In this section you may find indication on the role and function of clinical studies in research and drug development.
Published by UNEW
AdviceClinical DevelopmentClinical Trial DesignPreclinical ResearchResearch and Drug DevelopmentTranslational ResearchTREAT-NMDThis pre-application form template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit.
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AdviceClinical DevelopmentClinical Trial DesignPreclinical ResearchResearch and Drug DevelopmentTranslational ResearchTREAT-NMDThis report to the applicant template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit.
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AdviceClinical DevelopmentPreclinical ResearchResearch and Drug DevelopmentToolkits for patientsTranslational ResearchTREAT-NMDThe Advisory Committee for Therapeutics (ACT) toolkit provides procedural advice and guidance to replicate the successful TREAT-NMD Advisory Committee for Therapeutics (TACT) model in other rare disease communities. An ACT can provide independent and objective advice to industry and academia, on a real-life therapeutic pathway of drug development programmes. An ACT review can help an applicant to position a candidate compound along a realistic and well-informed pathway to clinical trial and eventual
registration.
Published by UNEW
AdviceClinical DevelopmentClinical Trial DesignPreclinical ResearchResearch and Drug DevelopmentTranslational ResearchTREAT-NMDThis agenda template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit.
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AdviceClinical DevelopmentClinical Trial DesignPreclinical ResearchResearch and Drug DevelopmentTranslational ResearchTREAT-NMDThis applicant agreement template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit.
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AdviceClinical DevelopmentClinical Trial DesignPreclinical ResearchResearch and Drug DevelopmentTranslational ResearchTREAT-NMDThis conflict of interest form template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit.
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Clincal Trial DesignPublished by EMA
Biotechnological ProductCarcinogenicity StudyGenotoxicity StudyImmunotoxicology StudyNonclinical Safety in Paediatric MedicinePhotosafety EvaluationRepeat-Dose ToxicityReproductive ToxicologySafety Pharmacology StudyTherapeutic Area-SpecificToxicokinetics and PharmacokineticsThe European Medicines Agency publishes scientific guidelines on human medicines that are harmonised by the International Council for Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH)
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Clinical DevelopmentNCATSNetworkRDCRNThe RDCRN program is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation.
View this resource Bookmark this resourceRaDaR, formerly known as the Global Rare Diseases Registry Data Repository program, aims to define best practices for patient registries. RaDaR also strives to identify and adopt standards to support high-quality registries for rare diseases therapeutics development.
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