With all eyes on new leadership, it appears FDA aims to keep pace with the emerging and cutting-edge technologies biotech is bringing forward. With growing industry and public expectations and increasing pressure to accelerate therapies to patients, particularly within the rare disease community, FDA appears to be responding.
At this intersection, the FDA landscape is shifting faster than ever. New leadership, coupled with increasingly novel therapeutic and manufacturing innovations, is driving the expansion of expedited pathways, pilot programs, and a broader commitment to regulatory flexibility. While these changes are designed to accelerate development, and we anticipate they will do just that, they also introduce complexity and uncertainty. Sponsors struggle to keep pace with what's changing, what's emerging, and what it means for their programs.
At the same time, fundraising pressures demand faster, de-risked development strategies. To keep pace with this momentum and get ahead of investor expectations, sponsors must take a proactive approach. In response, RareMoon is helping sponsors identify and leverage opportunities through the launch of our FDA Designation Action Plan; a structured approach to anticipate, prioritize, and pursue various FDA mechanisms.
The Action Plan
RareMoon offers a comprehensive FDA Designation Action Plan, a structured, forward-looking assessment to identify, qualify, and leverage FDA acceleration mechanisms.
We will:
- Evaluate your program across all FDA designations, expedited pathways, and pilot programs (new and legacy)
- Conduct feasibility and strategic assessments
- Identify opportunities to stack and sequence designations
- Deliver a clear, timeline-driven action plan
Programs & Pathways Assessed
When thoughtfully sequenced, these mechanisms work together to reduce uncertainty, maintain momentum, and accelerate patient access while meeting regulatory expectations.
- Orphan Drug Designation
- Rare Pediatric Disease designation
- Support for Clinical Trials Advancing Rare Disease Therapeutics (START)
- Rare Disease Endpoint Advancement (RDEA)
- Fast Track Designation
- Regenerative Medicine Advanced Therapy (RMAT)
- Breakthrough Therapy Designation
- Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program
- Advanced Manufacturing Technology (AMT) designation
- CDER/CBER Rare Disease Evidence Principles (RDEP)
- Plausible Mechanism Approval Pathway
- Commissioners National Priority Review Voucher
- Qualified Infectious Disease Product (QIDP)
- Priority Review
- Priority Review Voucher
- Platform Technology Designation
- Emerging FDA initiatives as they evolve
Key Deliverables
- Comprehensive regulatory incentive landscape
- Feasibility and strategic fit analysis
- Prioritized opportunities
- Application and engagement timeline
- Strategic recommendations for stacking and sequencing
Why Now?
FDA programs and regulatory frameworks are evolving faster than most development teams can track. New pilots, updated guidance, and emerging acceleration models create both opportunity and uncertainty.
RareMoon continuously monitors FDA movement, ensuring sponsors stay ahead of unknowns and leverage acceleration mechanisms in a timely and effective manner.
Who This Is For
- Biotech startups preparing for funding rounds
- Advanced therapy developers (cell, gene, RNA, rare disease)
- Sponsors seeking regulatory clarity and acceleration
- Programs navigating evolving FDA pathways
Request a Regulatory Designation Assessment
Get in line! Gain an expert evaluation of your program's eligibility for FDA designations, expedited pathways, and pilot programs, including feasibility, sequencing, and timing considerations.
Contact RareMoon for your FDA Designation Action Plan.