Insights

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.

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.

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.

Despite FDA staffing concerns, our experience has been steady—fast responses, consistent support, and strong collaboration continue.

Navigate regulatory uncertainty with strategy, early engagement, and thoughtful communication to build trust, reduce risk, and drive rare disease programs forward.

RareMoon exists to navigate rare disease drug development, overcoming regulatory hurdles to bring life-changing therapies to patients. #RareDiseaseDay

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.

RareMoon expert Mette Due Theilade Thomsen to lead DIA Global's virtual Paediatric Investigation Plans (PIP) training in November, offering key regulatory insights and practical skills.