We’re hearing the same concerns from clients across the board: “What now?” In a time of shifting agency dynamics, changing leadership, and uncertain timelines, here are five grounded strategies to help keep your program moving forward.
1. Stay the Course
Stay the course — and be mindful of the circumstances your review team is navigating. As much uncertainty as we may feel, they are just as much, if not more. Many are working under new or shifting leadership, unsure if they will retain their positions, and managing internal and external pressures. Extend professional courtesies, starting with your Regulatory Project Manager, and remember that these teams are working under intense scrutiny and timelines.
Be thoughtful in your interactions and submissions. Do not assume your reviewers are experts in your specific indication or modality — there are over 7,000 rare diseases and only a limited number of reviewers. Be precise, strategic, and also considerate when presenting your data and requests. A thoughtful approach not only serves your program but respects the reviewers who are essential to its progress.
2. Know Where You Stand.
Know where you stand. A regulatory plan, including a high-level gap assessment, allows sponsors to identify weaknesses or open questions in their development program — across nonclinical, CMC, and clinical disciplines, as well as regulatory uncertainty and gaps. Having a clear action plan is critical. Have a regulatory strategy, at least until your next milestone, (right now, for investor considerations, that’s looking like proof-of-concept). Investors and stakeholders are wrangling with the uncertainty as well. To overcome some of these, its helpful for Sponsors to understand the gaps, identify risks and have actionable plans to close them. With this, we demonstrate program readiness and reduce perceived risk by proactively addressing deficiencies and aligning the development plan with the current regulatory landscape. This makes your program more attractive for investment, partnerships, or further development amid changing regulatory environments.
3. Engage Early and Often with Health Agencies
Uncertainty should not delay agency engagement. Securing formal feedback from the FDA, EMA, or other regulators through INTERACT, Pre-IND, Scientific Advice, or similar meetings provides tangible evidence of program viability.
Meeting feedback is not only valuable scientifically but strategically—it shows investors and partners that your regulatory path is being validated by the right agencies. Early agency feedback also helps you preemptively adapt the development plans, reducing surprises later and building confidence in your program’s trajectory.
4. Have a Plan B.
In our experience, Health Canada and many EU national agencies consistently provide feedback well-aligned with FDAs, often with the added benefit of more flexible engagement models. Sponsors frequently overlook these opportunities, but they offer valuable insights, alternative perspectives, and, in some cases, a more efficient path forward, especially during times of uncertainty.
Consider engaging beyond the U.S. Identify your Key Opinion Leaders, understand where they are most active, and be prepared to engage them and regulatory agencies outside the U.S. You don’t have to go far to find proactive and highly collaborative regulators.
5. Make Your Regulatory Provider a PART of Your Team
Want to navigate this uncertainty? Get to know the people who engage with the FDA day in and day out. Don’t default to hiring a “Global Head of Regulatory” to lead a Pre-IND or Scientific Advice meeting — titles don’t substitute for real-time, working knowledge of agency expectations and processes.
Surround yourself with regulatory experts familiar with the nuances of these interactions—the questions that matter (not only for program advice but also considering how the investment community will interpret the feedback), the flexibilities available, and the informal norms that aren’t always written in guidance and often need to be teased out with savvy framing.
Engage us for more real-time advice. Don’t wait until you’ve made a misstep or submitted a package that leaves questions unanswered. Regular, proactive engagement with us isn’t just about navigating uncertainty — it’s about avoiding preventable delays and setting your program up for long-term success.
Contact RareMoon to discuss your specific situation and questions. We are here to support you and are happy to be a resource for you and your team.
