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Orphan Drugs: Worldwide Orphan Medicinal Designation Workshop

December 27, 2013

Mark your calendars !!

Plan on attending a workshop on orphan product designation and grants organized by the following three regulatory agencies :

•   European Medicines Agency (EMA)

•   US Food and Drug Administration (FDA)

•   Japanese Ministry of Health, Labour, and Welfare (MHLW) and Pharmaceuticals and Medical Devices Agency (PMDA).

The joint EMA/FDA/MHLW-PMDA Orphan Medicinal Product Workshop is scheduled for March 10, 2014, at the EMA, in London (UK). There is no registration fee for attending, but registration is required. One can register online. The meeting Agenda is available.

The purpose of the workshop is to provide information, about the EMA, FDA, and MHLW-PMDA regulatory agencies, requirements for orphan product designations, and information on grant programs available for the development of orphan products. The goal is to encourage and promote the development of new orphan products for rare diseases.

The workshop’s morning session will provide information about the EMA, FDA, and MHLW-PMDA regulatory agencies and their different orphan product regulations. It is to be broadcast live on the EMA website. The afternoon session involves one-to-one meetings with each of the three regulatory agencies’ staff, where one has an opportunity to present “a draft application for orphan designation and discuss issues with the three agencies concerning a medicine under development that holds promise for the treatment of a rare disease.”

This is the third time this workshop is being held and the first time that Japan’s WHLW-PMDA is involved as an organizer. It is an opportunity to encourage “international cooperation for orphan medicines” between three different and unique global regulatory agencies. Since 2008, the EMA and the FDA work together to encourage applications to be submitted in parallel for orphan product designation to both agencies. According to the EMA website, in 2012, 62% of applications for orphan product designation are submitted in parallel to the EMA and the FDA. Japan’s PMDA and the EMA release in July 2012, “pilot” terms for cooperation on orphan products.


Japan’s Ministry of Health, Labour, & Welfare: Overview of Orphan Drug/Medical device Designation System.

Please Note: “Writing Hand” by gnuckx (Flickr: Roma Italy – Creative Commons by gnuckx) [Public domain or CC-BY-2.0] | Wikimedia Commons.

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