Insights

Every January, the biotech world connects in San Francisco for the J.P. Morgan Healthcare Conference. For RareMoon, JPM is where we kick off our year with conversations that accelerate rare disease therapies. As we enter our decade milestone, JPM 2026 marks an especially exciting year.

RareMoon's Clinical Operations Consultant offers her insights on liaising between regulatory teams and clinical/CRO partners, navigating IND submissions, and supporting start-ups facing clinical trials.

RareMoon's Senior Regulatory Expert offers her insights on advanced therapies, regulatory pathways, and strategic guidance for rare disease treatments.

Expert answers to fundamental questions about Orphan Drug Designation timing, FDA vs EMA differences, and strategic considerations for emerging biotech companies developing therapies for rare and ultra-rare diseases.

On September 3, 2025, the FDA announced the Rare Disease Evidence Principles (RDEP). This new regulatory framework is meant to provide more explicit guidance, enhanced communication and collaboration, and offer greater flexibility in how therapies for ultra-rare, genetically driven diseases are developed and reviewed.

An August 2025 Regulatory Focus article affirms a trend we have been experiencing at RareMoon: placing importance on early and effective communication leads to regulatory success.