Extrapolation of efficacy and Safety in medicine development

Published by IRDiRC

Adaptative Pathways Clinical Development Clinical Trial Design Extrapolation of Efficacy and Safety Regulatory Sciences Research and Drug Development Study Design Target Population

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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National programs for early access

Published by IRDiRC

Clinical Development Compassionate Use Regulatory Sciences

Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available

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Alternative designs for Small Population Clinical Trials

Published by IRDiRC

Adaptative Pathways Clinical Development Clinical Trial Clinical Trial Design Rare Disease Regulatory Sciences Small Population

Guidance and general recommendations are available to select the most efficient study design for each medical condition or trial, and on potential adaptions of conventional designs to the low sample size scenario.

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Use of Biomarkers in Orphan Drug Development

Published by IRDiRC

Biomarker Development Clinical Development Endpoint EndpoInternationalBiomarker Orphan Drug Regulatory Sciences Research and Drug Development

Biomarkers 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.

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Decentralized trials

Published by IRDiRC

Adaptative Pathway Clinical Development Clinical Trial Design Decentralised Trial Digital Endpoint Patient Data Regulatory Sciences Research and Drug Development

Decentralized 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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The Innovation Management Toolbox was originally developed through funding from the EJP RD project, which received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No. 825575. The continued development and maintenance of the Toolbox are now supported by the ERDERA partnership, funded under the European Union’s Horizon Europe Research & Innovation programme, grant agreement No. 101156595.

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