Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives

Published by IACMHR Co., Ltd.

Orphan DrugRegulatory Science

this paper describes the current status of the regulation of rare diseases and orphan drugs in Asia and we comparatively analyze the regulation of rare diseases and orphan drugs worldwide in order to examine the challenges to and future perspectives on promoting research on rare diseases and development of orphan drugs in China and other Asian countries.

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Qualification of novel methodologies for drug development: guidance to applicants (EMA/CHMP/SAWP/72894/2008). 2013

Published by EMA

Scientific AdviceEMA Qualification

The EMA Qualification process is a new, voluntary, scientific pathway leading to either a CHMP Qualification opinion or a qualification advice on innovative methods or drug development tools

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The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services have a program to provide parallel scientific advice (PSA) to sponsors. The goal of the PSA program is to provide a mechanism for EMA assessors and FDA reviewers to concurrently exchange with sponsors their views on scientific issues during the development phase of new medicinal products (i.e., new human drugs and Biologics).

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Study Group on Unapproved and Off-label Drugs of High Medical Need

Published by IRDiRC

Regulatory AffairsEarly Access SupportScientific AdviceMHLW or PMDA Regulatory

The objective of the Study Group is to evaluate medical need, investigate necessary studies for market approval, and request company to develop medicinal products to solve the problem of unapproved drug and off- label use with medical need. Public consultations are conducted to gather requests from public.

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