The Ultrarare Disease Drug Loss

The VC-funded biotech model that has driven pharmaceutical innovation since the 1980s is reaching its limits, particularly for the approximately 9,500 ultrarare diseases affecting fewer than 300 patients in the US. The high cost of capital now demands price tags of several million dollars per patient, creating an unsustainable situation.

In Japan, this challenge manifests as the “drug loss” problem — an increasing number of innovative therapies developed abroad that never reach Japanese patients. Launch prices in the US are now approximately three times higher than in Japan, and the gap continues to widen.

A New Model for Innovation

Innovation in science and technology must be complemented by new models of organising and financing drug development. Non-profit business models for ultrarare disease therapeutics are emerging in the US, Europe and elsewhere, but Japan has yet to establish such a framework. JFRDR is being established to fill this critical gap.

By leveraging low-cost capital in the form of government-backed grants, JFRDR aims to provide drugs that would otherwise not be available to Japanese patients — at a cost that ensures the Japanese Social Security system remains financially sustainable.

JFRDR Activities

JFRDR aims to be a key change agent in the Japanese biopharma ecosystem through several core activities:

• Patient Organisation Support — Vitalising and professionalising Japanese patient organisations to give patients a stronger voice
• Public Education — Raising awareness about the realities of living with rare diseases in Japan
• Infrastructure Building — Contributing to critical translational and CMC infrastructure
• Global Collaboration — Becoming a key player in the emerging global network of non-profit ventures along the R&D value chain

Global Vision

The ultimate goal extends beyond Japan — JFRDR strives to not only develop drugs for ultrarare diseases but to make them available to patients all over the world, wherever they may live.