Recent headlines have raised valid concerns about staffing and internal shifts at the FDA. The FDA Law Blog highlighted the strain on reviewers following the dismissal of senior staff, and Reuters reported that the FDA has let go of most of its negotiators for upcoming pharma user fee talks. These developments point to uncertainty, especially in areas like rare disease and advanced therapies, where predictability is paramount.
However, from where we stand — in daily, real-time interaction with FDA teams — the experience has been remarkably steady, even encouraging.
Our Real-World Experience
At RareMoon, we've recently cleared an IND for a first-in-clinic novel therapeutic product, received timely responses to RMAT submissions, and had record-fast meeting minutes returned. These aren't isolated events. We're in contact with CBER's Office of Therapeutic Products (OTP) multiple times a week and continue to be met with clarity, professionalism, and even gratitude from agency staff.
💡 Recent Achievements
• Cleared IND for first-in-clinic novel therapeutic product
• Received timely responses to RMAT submissions
• Record-fast meeting minutes returned
• Multiple weekly interactions with CBER's OTP
The Reality Check
Are timelines always a concern for our clients? Absolutely. But in reality, given the broader context, the FDA is currently exceeding our expectations. Our Regulatory Project Managers have remained consistent and engaged, and we've seen no drop in responsiveness or commitment to public health.
In fact, the agency's support has been instrumental in helping our clients navigate complex and time-sensitive regulatory paths. The dedication we've witnessed from FDA staff during this period of transition has been nothing short of remarkable.
What Makes the Difference
We also recognize that part of the reason our experience may differ is due to how we approach agency interactions. We make it a priority to submit clear, aligned meeting minutes that rarely require edits — and we show up prepared, respectful of the agency's time and process.
Our Collaborative Approach
- Clear Communication: Submit well-structured, aligned meeting minutes that minimize back-and-forth
- Thorough Preparation: Come to meetings with comprehensive data and clear questions
- Respectful Engagement: Honor the agency's time and regulatory processes
- Professional Relationships: Build trust through consistent, reliable interactions
- Solution-Oriented: Focus on finding pathways forward rather than obstacles
That level of collaboration makes a difference. When sponsors approach the FDA with respect, preparation, and a genuine commitment to patient safety, the agency responds in kind.
Acknowledging the Challenges
We continue to acknowledge the pressure the agency is under and the very real implications of staffing changes. The concerns raised by industry observers are valid, and we don't dismiss the broader challenges facing the FDA during this period of transition.
The dismissal of senior staff and changes to the negotiating team for user fee agreements are significant developments that deserve attention. These changes could have long-term implications for:
- Institutional knowledge retention
- Consistency in regulatory decision-making
- User fee negotiation outcomes
- Timeline predictability for complex submissions
The Bigger Picture
What we're witnessing may be a testament to the professionalism and dedication of the career scientists and regulatory professionals who remain at the agency. These individuals understand their critical role in advancing public health and continue to execute their responsibilities with remarkable commitment.
It's also worth noting that the FDA has faced staffing challenges and organizational changes throughout its history, yet has consistently maintained its core mission of protecting and promoting public health. The agency's resilience during periods of transition speaks to the strength of its institutional framework and the dedication of its workforce.
"From our seat at the table, the FDA continues to show up — for sponsors, for patients, and for progress. And for that, we're incredibly grateful!"
Looking Forward
While we remain cautiously optimistic about our continued positive interactions with the FDA, we also understand the importance of staying vigilant and adaptable. The regulatory landscape is always evolving, and successful navigation requires both preparation and flexibility.
For sponsors working in rare disease and advanced therapies, our advice remains consistent:
- Engage Early and Often: Establish relationships and seek guidance proactively
- Come Prepared: Invest in thorough preparation for all FDA interactions
- Be Respectful: Honor the agency's expertise and regulatory processes
- Stay Flexible: Be prepared to adapt to changing circumstances
- Focus on Patients: Keep patient benefit at the center of all discussions
A Message of Gratitude
As we navigate these uncertain times, we want to acknowledge the incredible work being done by FDA staff who continue to show up every day with professionalism, expertise, and dedication to public health. Their commitment to advancing innovative therapies, especially in rare disease areas where patients have few or no treatment options, deserves recognition and appreciation.
We also recognize that our positive experience may not be universal, and we remain committed to sharing both our successes and any challenges we encounter as they arise. Transparency in these discussions benefits the entire rare disease community.
From our seat at the table, the FDA continues to show up — for sponsors, for patients, and for progress. And for that, we're incredibly grateful!