Orphan Drugs: Worldwide Orphan Medicinal Designation Workshop

Orphan Druganaut Blog

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…

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FDA Breakthrough Therapy Designation: 2013 In Review

Orphan Druganaut Blog

As most of the Blog’s readers already know, FDASIA, or the FDA Safety and Innovation Act (S. 3187) is enacted after President Obama signs it in July 2012. FDASIA is a big step towards developing effective and safe treatments for rare diseases and orphan drug development in the United States. The legislation is to have an important and large impact on the future of accelerating new medical treatments for patient access.

It is in Section 902 of FDASIA, that the new FDA Breakthrough Therapy Designation (BTD) is described. To expedite the development and review of drugs for life-threatening and serious diseases, the FDA had three programs before the BTD is created :

•   Fast Track
•   Accelerated Approval
•   Priority Review.

The three Designations are in existence for at least 20 years. What is different about the BTD is the “ … requirement of early clinical data demonstrating an unprecedented…

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Update to EU GMP Chapter 2 – Personnel

Inspired Pharma Blog


A new update to EU GMP Chapter 2 has been issued and comes into operation on 16th February 2014.  Here is a summary of the main changes from the previous version Chapter 2 Personnel OLD.  A copy of the new version can be found by clicking on the link Chapter 2 Personnel NEW.

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