Orphan Drug Cooperation between Japan and Europe

Over the past three years, there has been increased collaboration between the Pharmaceuticals and Medical Devices Agency (PMDA) of Japan and the European Medicines Agency (EMA).  One particular area of mutual interest that has been discussed is orphan drugs, as access to orphan drugs for those suffering from rare diseases is vital.  Both the PMDA and EMA individually have encouraged progress in the field of orphan drugs through several measures, and the two regulatory bodies are now looking to work together.

On July 30, 2012, the PMDA and EMA released pilot terms to a cooperation on orphan medicines.  The first goal of the collaboration is to better understand each other’s orphan drug designation submission process. More specifically, they would like to determine the ways in which the regulations overlap (or are different) when designating orphan drugs.  The flow of this kind of information has already begun, with a Japanese expert working in the EMA for several months last year.  The hope is now to create exchange systems that would report on:

  • the research and development stage of orphan drugs, where it is deemed necessary;
  • the outcome of orphan drug designations;
  • the basic argument for authorization or rejection during the licensing stage;
  • special considerations for manufacturing, supply, or distribution;
  • as well was phamacovigilance and risk management information from the post-licensing stage.

One of the additional objectives includes cooperation with respect to small and medium sized enterprises.  Although specific goals have not yet been outlined, the aim is to reduce bureaucracy for such enterprises.