Japan Agency for Medical Research and Development (AMED) leads the Initiative on Rare and Undiagnosed Diseases (IRUD) program. The program provides patients across Japan suffering from undiagnosed diseases with a diagnostic strategy that involves genetic analysis

View this resource Bookmark this resource

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

View this resource Bookmark this resource

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.

View this resource Bookmark this resource

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.

View this resource Bookmark this resource

FDA Expedited Program for serious conditions – Special Protocol Assessment

Published by IRDiRC

Regulatory AffairsClinical DevelopmentClinical Trial DesignRegulatory AdviceSpecial 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.

View this resource Bookmark this resource