Comment from Cure MAPT FTD
AnonymousSupportAdvocacy
Summary: The Cure MAPT Frontotemporal Dementia (FTD) community strongly endorses the proposed framework as it aligns with patient priorities for accelerating therapies for rare genetic conditions. They argue that the framework addresses critical needs such as mechanism-based rationales, trial flexibility for small populations, and expedited pathways for mutation-specific diseases.
On behalf of the Cure MAPT Frontotemporal Dementia (FTD) community, we strongly endorse the Plausible Mechanism Framework as consistent with the priorities expressed by patients and families during our FDA Patient Listening Session on 5/23/25. Participants articulated seven key asks to accelerate the development of effective therapies for rare, genetically defined FTD:
1. Recognition of the urgent unmet medical need in MAPT FTD.
2. Acceptance of scientifically justified, mechanism-based rationales for therapeutic development.
3. Flexibility in trial design to accommodate small patient populations.
4. Incorporation of meaningful biomarkers and disease-specific endpoints.
5. Transparency in regulatory decision-making and clear communication with patients.
6. Engagement of patients and caregivers throughout the therapy development process.
7. Expedited pathways for therapies that show evidence of target engagement and potential clinical benefit.
The proposed framework directly addresses these asks by allowing evidence from small, mechanistically guided studies to inform regulatory decisions while maintaining rigorous standards for safety and efficacy. By formally adopting this approach, the FDA would honor the voice of the MAPT FTD patient community, ensure that families are not left behind, and create a clear path toward therapies tailored to ultra-rare, mutation-specific neurodegenerative diseases.