Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Exceptional Japan filing of a biologic mature product

Dr. Astrid Gerhardt (Abschlußjahr: 2012)

Language: English

With an estimated total population of 128 million inhabitants in 2011 and pharmaceuticals sold for around 65 billion Euros in 2007, Japan is the second largest market for pharmaceuticals in the world.

The Pharmaceutical Affairs Law (PAL) was enacted in 1943 to assure the quality of drugs marketed in Japan and today consists of eleven chapters and 91 articles. The last major revision of the Japanese Pharmaceutical Affairs Law (JPAL) became effective in April 1st, 2005. The Japanese regulatory system has many unique features like the "drug lag", biennial price cuts, a separation of dispensing from medical practice called "Iyaku Bungyo" and a requirement for local clinical trials for approval.

As a consequence of the 2007 "Five-Year Strategy for Creation of Innovative Pharmaceutical Products and Medical Devices", in April 2010, the Ministry of Health, Labor and Welfare (MHLW) requested to obtain as soon as possible the dossier of a drug marketed since more than 15 years in the USA, in Europe and in other countries. This drug, a biologic, is indicated for the treatment of patients suffering from a genetic disease which is infrequent in Japan. It is produced in the USA.

Because of the long history of the drug, at the beginning of the project, the dossier consisted of the original submission and of a variety of documents created after the original filing in order to keep the dossier up to date.

In February 2011, an orphan drug application was made and in mid July 2011, the CTD-structured dossier was submitted to Japanese Health Authorities.
This thesis describes, after a short introduction to the Japanese regulatory environment, the update of the quality part of the dossier and the process of this exceptional Japan filing which included international cooperation.