FDA Breakthrough Therapy Designation Services
RareMoon has successfully led numerous biotech companies through the Breakthrough Therapy Designation process.
Our expertise includes preparing robust preliminary Breakthrough Therapy Designation 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.
BTD Program Benefits
Programs granted Breakthrough Therapy Designation may benefit from:
- Intensive FDA guidance and enhanced communication throughout development
- Rolling review of application components as they become available
- Priority Review eligibility at the time of marketing application
- Accelerated Approval pathway based on surrogate or intermediate endpoints
- Organizational commitment involving senior FDA managers
How RareMoon Supports BTD 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
- Preparation and authorship of the BTD request, ensuring alignment with CBER or CDER expectations
- Regulatory positioning of early clinical data to highlight substantial improvement
- Planning and leading the Comprehensive BTD Multidisciplinary Meeting
- Ongoing FDA engagement support and regulatory next steps post-designation
RareMoon brings unmatched insight, proven execution, and sustained strategic guidance to help you progress your therapy from BTD request to regulatory approval.
About Breakthrough Therapy Designation
The FDA's Breakthrough Therapy Designation is intended to expedite the development and review of drugs that treat serious conditions and have preliminary clinical evidence indicating substantial improvement over existing treatments on clinically significant endpoints.