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Orphan Drugs: 2013 Forecast For Europe

May 3, 2013

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP), announces February 14, 2013 the committee’s accomplishments and goals for 2013.

The trend for the past several years according to COMP, is a steady increase in the granting of orphan designations over the last few years. Though there is an increase of almost 38.3 % from 2011 to 2012, there is only a slight increase that is expected of > 1.3 % from 2012 to 2013 :

  1. In 2011, 107 orphan designations
  2. In 2012, 148 orphan designations
  3. In 2013, > 150 orphan designations is expected.

There is an increase of 35.7 % in the number of applications for marketing authorizations for orphan designated products from 2011 to 2012 :

  1. In 2011, 14 applications for marketing authorization
  2. In 2012, 19 applications for marketing authorization.

The COMP February 2013 announcement outlines the following accomplishments and 2013 goals :

  1. Expansion of international cooperation
  2. Collaboration with Health-Technology Assessment (HTA) groups
  3. Integration of patient views into the decision process.

Expansion Of International Cooperation

COMP starts a collaboration with the US FDA in 2008, permitting applications for orphan drug designations to be submitted in parallel to both regulatory agencies. This action results in “ … 62% of applications submitted in parallel in the EU and the FDA in 2012.”

The Pharmaceuticals and Medical Devices Agency (PMDA) of Japan and the European Medicines Agency (EMA) on July 30, 2012, release terms to cooperate on orphan medicines. The first objective is for both groups to understand each other’s orphan drug designation submission process. The result in 2012 is an increase in the number of Japanese orphan drug designations with prior EU designations.

Also, Health Canada and COMP start discussions on orphan drugs and a close collaboration is expected for 2013.

Collaboration With Health-Technology Assessment (HTA) Groups

COMP is closely working with EUnetHTA, an organization setup to create a network for HTA across Europe to “help develop reliable, timely, transparent, and transferable information to contribute to HTAs in European countries”.

Integration Of Patient Views Into The Decision Process

COMP will request and involve expertise through patient organizations to contribute in decision making about assessing the benefits and development of orphan medicines for specific drug indications. In particular, COMP will work closer with the European Organization for Rare Disease (EURORDIS).


EMA & PMDA July 30, 2012 Report

Please Note: “Azt” by John Crawford (Photographer) [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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