Stakeholders interactions – Patients Affairs Staff

Published by IRDiRC

Research and Drug Development Regulatory Affairs Clinical Development Early Access Support Patient Engagement Patient Advice FDA Regulatory Advice Patient Affairs Staff Patient Support

At the FDA, the Patient Affairs Staff (PAS) is devoted to supporting cross-cutting patient engagement activities across the FDA. Its mission is to coordinate and support patient engagement activities across FDA to facilitate awareness and collaboration with patients, their advocates and the FDA

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Use of Biomarkers in Orphan Drug Development

Published by IRDiRC

Research and Drug Development Regulatory Affairs Clinical Development Endpoint Biomarker Orphan Drug Biomarker Development Endpoint

Biomarkers can be utilized as primary surrogate endpoint as 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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Clinical Trial Regulation

Published by EMA

Regulatory Affairs Clinical Development Clinical Trial Design

The goal of the Clinical Trial Regulation is to create an environment that is favourable to conducting clinical trials in the EU, with the highest standards of Safety for participants and increased transparency of trial information.

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Adaptive Pathways

Published by IRDiRC

Regulatory Affairs Clinical Development Clinical Trial Design Adaptative Pathway Early Access Support

The adaptive pathways approach is part of the European Medicines Agency’s (EMA) efforts to improve timely access for patients to new medicines

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Extrapolation of efficacy and Safety in medicine development

Published by IRDiRC

Research and Drug Development Regulatory Affairs Clinical Development Clinical Trial Design Target Population Study Design Extrapolation of Efficacy and Safety Adaptative 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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Alternative designs for Small Population Clinical Trials

Published by IRDiRC

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

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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FDA Expedited Program for serious conditions – Special Protocol Assessment

Published by IRDiRC

Regulatory Affairs Clinical Development Clinical Trial Design Regulatory Advice Special Protocol Assessment

FDA-SPA is a process in which sponsors may ask to meet with FDA to reach agreement on the design and size of certain clinical trials, clinical studies, or animal studies, to determine if they adequately address scientific and regulatory requirements for a study that could support marketing approval.

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EU Compassionate Use Programs

Published by IRDiRC

Research and Drug Development Regulatory Affairs Clinical Development Orphan Drug Early Access Support European Union Compassionate Use EMA Compassionate Use

Compassionate use is a treatment option that allows the use of an unauthorised medicine. Under strict conditions, products in development can be made available to groups of patients who have a disease with no satisfactory authorised therapies and who cannot enter clinical trials.

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National programs for early access

Published by IRDiRC

Regulatory Affairs Clinical Development Compassionate Use

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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Japan Compassionate Use Programs

Published by IRDiRC

Regulatory Affairs Clinical Development Early Access Support MHLW or PMDA Regulatory Compassionate Use Programme

A system for patients with life-threatening conditions without effective a treatment to gain access to unapproved investigational medical products.

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