Clinical Development

In this section you may find indication on the role and function of clinical studies in research and drug development.

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Advisory Committee for Therapeutics (ACT) toolkit

Published by UNEW

Translational ResearchResearch and Drug DevelopmentTREAT-NMDClinical DevelopmentClinical Trial DesignPreclinical ResearchTemplatesAdvice

The 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. 

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Rare Diseases Clinical Research Network (RDCRN)

Published by NCATS

Clinical DevelopmentNetworkNCATSRDCRN

The 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.

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Procedural document: data collection and registration of clinical trials

Published by Orphanet

Patient EngagementClinical TrialOrphanet

Orphanet offers, amongst a range of expert resources on rare diseases, a directory of national and multinational clinical trials aiming to help: – clinical investigators working in the field of rare diseases find each other, establish collaborations, recruit patients; – patients and general public retrieve information on ongoing clinical trials for a particular disease, and stay informed on clinical trials that are recruiting; – experts, funding agencies and pharmaceutical industry obtain visibility on therapeutic development in the field of rare disease.

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Rare Diseases Registry Program (RaDaR)

Published by NCATS

Patient Registry

RaDaR, 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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Feasibility-Patient engagement in trial endpoint selection 

Published by IRDiRC

Research and Drug DevelopmentClinical DevelopmentEndpointPatient EngagementClinical TrialOutcome MeasureTrail Endpoint Selection

Guidance 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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Extrapolation of efficacy and Safety in medicine development

Published by IRDiRC

Research and Drug DevelopmentRegulatory AffairsClinical DevelopmentClinical Trial DesignTarget PopulationStudy DesignExtrapolation of Efficacy and SafetyAdaptative Pathways

The EMA describes extrapolation as extending information and conclusions available from studies in one or more subgroups of the patient population (source population(s)), or in related conditions or with related medicinal products, in order to make inferences for another subgroup of the population (target population), or condition or product, thus reducing the amount of, or general need for, additional evidence generation (types of studies, design modifications, number of patients required) needed to reach conclusions.

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