Clinical Trial

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Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01) Clinical Trials Required

Published by NIH

Clinical TrialRare diseasesUnmet Needs

The purpose of this funding opportunity announcement (FOA) is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions. Additionally, through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare diseases and exert a broad and positive impact on rare disease drug development.

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Clinical trials – Regulation EU No 536/2014

Published by EMA

Clinical TrialRegulation

The Regulation will ensure a greater level of harmonisation of the rules for conducting clinical trials throughout the EU. It introduces an authorisation procedure based on a single submission via a single EU portal, an assessment procedure leading to a single decision, rules on the protection of subjects and informed consent, and transparency requirements

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Procedural document: data collection and registration of clinical trials

Published by Orphanet

Clinical TrialOrphanetPatient Engagement

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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Stakeholders interactions – Professional affairs and Community Engagement

Published by IRDiRC

Clinical TrialCommunity EngagementEarly Access SupportEarly Stage InteractionPatient AdviceProfessional AffairsRegulatory AdviceRegulatory AffairsScientific Advice

Interaction of the stakeholders and multilateral education at an early stage of development will facilitate drug development (e.g. ensuring outcome measures for clinical trials are developed that are meaningful for patients, are acceptable to the regulators, and are acceptable to payers)

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Feasibility-Patient engagement in trial endpoInternationalselection 

Published by IRDiRC

Clinical DevelopmentClinical TrialEndpointOutcome MeasurePatient EngagementResearch and Drug DevelopmentTrail EndpoInternationalSelection

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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Advisory Committee for Therapeutics (ACT) Toolkit

Published by Newcastle University

Clinical TrialExpert AdviceNetworkNeuromuscularResearch and Translational Resource

The TREAT-NMD Advisory Committee for Therapeutics (TACT) is a unique multi-disciplinary model centred upon an international group of academic and industry drug development experts, including patient representatives. TACT gives advice on the translational and development pathway of therapeutic programs in rare Neuromuscular Diseases. Due to its success in the neuromuscular field, a toolkit to replicate the TACT model has been produced. The Advisory Committee for Therapeutics (ACT) toolkit is intended to support rare disease communities outside of Neuromuscular to establish their own ACT, by providing them with step-by-step guidance and template documentation.

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Exploring the new horizons of Drug Repurposing: A vital tool for turning hard work into smart work

Published by European Journal of Medicinal Chemistry

Clinical TrialDrug DiscoveryDrug RepurposingLead MoleculeResearch and Drug Development

This review provides different approaches for Drug Repurposing and addresses recent developments and future scope of drug repositioning strategies. In addition provides a list of repurposed drugs and repurposing resources.

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