RareMoon has successfully led numerous biotech companies through the Breakthrough Therapy Designation (BTD) process.
Our expertise includes preparing robust preliminary Breakthrough Therapy Designation (BTD) Advice Requests to facilitate early dialogue with the FDA, completing full BTD applications, and leading post-designation multidisciplinary meetings to secure alignment across FDA review divisions.
We possess a nuanced understanding of BTD’s qualifying criteria, data expectations, and the regulatory impact of designation—tailored for first-in-class or accelerated programs.
Our team includes former FDA regulators and seasoned strategists who have designed and reviewed BTD requests from both the Agency and sponsor sides. This dual perspective enables us to anticipate questions, prepare sponsors for pivotal regulatory discussions, and optimize the benefits of BTD — from increased and enhanced FDA interactions to the potential for Priority Review and Accelerated Approval.
How RareMoon supports Breakthrough Therapy Designation efforts
- Strategic assessment of BTD eligibility, including data readiness and precedent benchmarking.
- Development of a Preliminary BTD Advice Request, structured to initiate productive FDA engagement and reduce risk at formal submission.
- Preparation and authorship of the BTD request, ensuring alignment with CBER or CDER expectations.
- Regulatory positioning of early clinical data to highlight substantial improvement over existing therapies.
- Planning and leading the Comprehensive BTD Multidisciplinary Meeting, including briefing books, slide decks, and team coaching.
- Ongoing FDA engagement support, including updates, communications, and regulatory next steps post-designation.
