Insights
Expert perspectives on regulatory strategy, market access, and therapeutic development from our team of former FDA reviewers and industry leaders.
Why RareMoon Shows Up for JPM, and Why It Remains Our Most Impactful Week of the Year
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.
Latest Insights
Ask the Expert: Sarah DiSalvatore, MPH
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.
Ask the Expert: S. Kaye Spratt, PhD
RareMoon's Senior Regulatory Expert offers her insights on advanced therapies, regulatory pathways, and strategic guidance for rare disease treatments.
Orphan Insights, Part 1: Orphan Drug Designation Strategy & Timing
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.
FDA's New RDEP Process: What It Means for Ultra-Rare Disease Drug Development
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.
Fostered Collaboration Results in Fewer Clinical Holds: A CBER OTP Pilot
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.
This Week's Update to the FDA's Commissioner's National Priority Voucher (CNPV) Pilot Program
Explore the FDA's new CNPV Pilot Program, a Commissioner-led initiative offering expedited review for therapies addressing critical public health priorities outside existing PRV pathways.
IMPACT: Regulatory Updates
Subscribe to RareMoon's quarterly insights regarding key regulatory developments shaping the future of orphan drugs and advanced therapies.
Whether you're launching a new program, approaching a critical milestone, or navigating global agency interactions, we're here to offer our experience and insight, keeping you informed, prepared, and aligned for success.
Ask the Expert
Submit your questions, and we'll have our experts answer them in our 'Ask the Expert' series or in an upcoming blog.