Insights
Expert perspectives on regulatory strategy, market access, and therapeutic development from our team of former FDA reviewers and industry leaders.
Accelerating Development in an Evolving FDA Landscape
With the FDA landscape shifting faster than ever, RareMoon is helping sponsors identify and leverage opportunities through our FDA Designation Action Plan, a structured approach to anticipate, prioritize, and pursue various FDA acceleration mechanisms.
Latest Insights
Orphan Drug Data Reflects a Consistent and Meaningful Trend
ODD activity and orphan drug approvals continue to show sustained momentum. A look at the numbers from 2021 through early 2026 and what they mean for rare disease programs.
Navigating FDA's Rare Disease Programs: A Practical Framework for Sponsors
A practical overview of the FDA rare disease and advanced therapy landscape in early 2026, with guidance on how to layer designations, pilots, and pathways to support program execution.
Plausible Mechanism & RDEP: Understanding Two Distinct Regulatory Tools in Rare Disease Development
A side-by-side comparison of the FDA's Plausible Mechanism Pathway and the Rare Disease Evidence Principles Pilot (RDEP) — two frameworks that are often discussed together but serve fundamentally different roles in rare disease development.
Rare Disease Accelerated Approval Updates (and Industry's Response)
An update on five gene therapy programs pursuing Accelerated Approval, emerging themes around surrogate endpoints, external controls, and evolving FDA evidentiary expectations for rare disease therapies.
A Magical Night at RareMoon's 10-Year Anniversary Celebration
From magic tricks and mind control to a GOAT Awards ceremony topped off with an MVP honor, RareMoon's 10th Anniversary celebration was unforgettable. It was a chance to laugh, connect, and reflect on our growth, our achievements, and the incredible team that makes RareMoon what it is today.
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.
IMPACT: Regulatory Updates
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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
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