T-13: Academia’s Challenges for Implementing an Investigator-initiated Clinical Trial Aimed at Developing A New Biological Drug

Poster Presenter

Tetsuya Kusakabe

Professor
Osaka City University, Graduate School of Medicine
Japan

Objectives

To confirm the actions necessary to implement clinical trials in Japanese academia, and to propose measures to activate open innovation based on the experience of an investigator-initiated clinical trial of a biological drug for rare disease.

Method

Information necessary to submit clinical trial notification to Japanese regulatory authorities (MHLW, PMDA) according to Japanese Law in order to carry out an investigator-initiated clinical trial is arranged. Challenges in order to accelerate medical innovation in Japan are also raised.

Results

1. Documents necessary for clinical trial notification include; (1) reason why the clinical trial is scientifically valid, (2) protocol for the trial, (3) Investigator’s Brochure, and (3) quality information of the investigational drug. After submitting these documents to PMDA, we repeatedly discuss with PMDA during 30 day’s PMDA review. PMDA consultation before submitting the notification would make the process much easier. 2. In order to accelerate medical innovation in Japan, academia has to; 1) draw concrete image for commercialization of the academia seeds. - Cooperation with a domestic pharmaceutical company in the early developmental stage. - Budget acquisition from public funding agency (AMED etc.) and private enterprises, etc. 2) respond quickly and accurately to inquiries from regulatory authorities. - Establishment of research organization scheme (ARO), securing of human resources, and accumulation of experiences. - Familiarity with pharmaceutical regulations (ex. Japanese Pharmaceutical Affairs Act, The Standard for Biological Ingredients). - “Those who master regulation achieve innovation”

Conclusion

Drugs have two aspects of efficacy and safety, and we human beings have experienced disastrous health damages by drugs in the past. In order to market new drugs, it is necessary to comply with internationally harmonized pharmaceutical regulations. It is no exaggeration to say that innovation in drug development is achieved only after compliance with regulation. In supporting an investigator-initiated clinical trial, we have confirmed the regulations necessary to use new medicines as investigational drugs, and found challenges to accelerate medical innovation. Especially, close cooperation with pharmaceutical company from the early developmental stage is necessary for the academia to fulfill pharmaceutical regulations including the standard for biological ingredients in developing biological drugs in Japan.