Insights
Expert perspectives on regulatory strategy, market access, and therapeutic development from our team of former FDA reviewers and industry leaders.
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.
Latest Insights
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.
Keeping Pace with Innovation: Current Insights from Global Health Authority Leadership
This week, we heard from health authority leaders from the United States, Europe, the United Kingdom, and beyond at the DIA 2025 Global Annual Meeting in Washington, DC, and one theme stood out: modernizing regulation to match innovation, with leading health agencies at the forefront.
Ask the Expert: Lilia Bi, MD, PhD
Sponsors often struggle with early PIP submissions for pediatric gene therapies due to their complexity. This article highlights EMA's guidance, the stepwise PIP pilot, and approaches to harmonize global pediatric plans for regulatory success.
Dr. Vinay Prasad: Balancing Innovation and Evidence in Rare Disease Treatment
When Dr. Martin Makary took his seat as FDA Commissioner earlier this year, his early remarks signaled a fresh era for rare disease and gene therapy developers. From promising to 'customize the approval process to the condition' to voicing support for faster reviews in ultra-rare populations.
Hope on the Horizon: Breakthrough for MPS IIIA
The second half of 2025 is shaping up to be a pivotal period for advanced therapies, with the FDA poised to decide on a wave of gene and cell therapies. The outcomes of these reviews will offer critical insights into the future of Accelerated Approval for gene therapies.
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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