Our Activities

Three Approaches

JFRDR adopts three complementary approaches to resolve current and future drug loss, for ultrarare disease operating as a professionally-managed non-profit organisation.

1. Bringing Foreign-approved Drugs to Japan

Obtaining the Japanese rights to drugs approved in the US but of no interest to commercial players, developing them at cost-plus in academic hospitals with CRO and CMO support, and out-licensing the commercial rights for distribution at “reasonable prices.”

2. Global Clinical Trial Participation

Including a small number of Japanese patients in global pivotal trials to facilitate PMDA approval and thus avoid future drug loss.

3. Supporting Academic Translational Research

Supporting selected translational projects addressing ultrarare diseases in Japanese academia financially and with coaching and mentoring by pharmaceutical experts to bring them to Proof of Concept and/or approval.

Key Approach

The key to JFRDR’s approach is to raise funds backed by government guarantees and operate on the basis of grants with very low cost of capital, enabling development of drugs which would otherwise remain unavailable. JFRDR operates complementary to commercial entities — it only works on high-quality projects which are commercially infeasible.

Five-Year Strategy

Phase 1 (Years 1-5): Focus on projects with relatively low risk and short timelines — drug loss projects and projects in clinical development outside Japan. With a five-year budget of approximately $60 million, five to six approvals are a realistic goal.

Phase 2 (Years 6-10): Extend scope to higher-risk projects with longer timelines, including translational and clinical projects in Japanese academia. JFRDR aims to deliver an additional 15 PMDA approvals by year 10.