Insights

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

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.

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.

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.

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.