Unlocking Japan’s potential in rare disease drug development

Japan stands as a promising market for the development of rare disease drugs, offering a well-structured regulatory framework and an established healthcare system. Japan provides comparable opportunities to U.S. or European markets, making it a strong candidate for expanding your rare disease drug development efforts.

Join us for an in-depth exploration of Japan’s regulatory landscape, patient dynamics and market environment. Participate in discussions with officials from Japan's Pharmaceuticals and Medical Devices Agency (PMDA) and industry leaders about the practical steps and strategies to bring innovative rare disease drugs to Japan. This event will equip your organization with the essential knowledge to effectively enter Japan’s market and make informed decisions about entering this globally important healthcare space.

11:00 AM - 11:30 AM ET

Navigating regulatory pathways for rare disease drugs in Japan

A rare opportunity to understand Japan’s regulatory landscape for rare disease drug development. Officials and reviewers from PMDA will provide an overview of Japan’s orphan drug designation process and its benefits, as well as insights into rare disease patient population sizes, and an introduction to registries that could support clinical development efforts. Learn how your company can successfully navigate Japan’s regulatory pathways to bring groundbreaking therapies to patients in need.

To view slides from this session, click here.

For more information related to this session from PMDA, click here.

Yoko Aoi

Coordination Director

PMDA

Mamoru Narukawa, Ph.D.

Director of Center for Product Evaluation and Director of Consultation Center for Pediatric and Orphan Drugs Development

Pharmaceuticals and Medical Devices Agency (PMDA)

Aya Kubota

Moderator

Editor-in-Chief

Nikkei Biotechnology & Business

11:35 AM - 12:05 PM ET

Developing rare disease drugs in Japan — a biotech perspective

What makes Japan an important and viable market for the development of rare disease therapies? Join industry leaders actively developing rare disease drugs in this market as they share their perspectives, challenges, and strategies for success. Gain insights into how to enter the Japanese market for rare diseases, the opportunities Japan offers to global innovators, and the steps your company can take to maximize your potential in the field of rare diseases.