Abstract Details

Title Insights on FTD: An Externally-Led Patient-Focused Drug Development Meeting

Topic Aging, Dementia, and Behavioral Neurology

Presentation(s) P8 - Poster Session 8 (11:45 AM-12:45 PM)

Poster/Presentation
Number 3-004

Objective Understanding the lived experience of those affected by a disorder should be central to the development and evaluation of therapeutics.

Background Disease-specific Externally-Led Patient-Focused Drug Development (EL-PFDD) meetings were established by the FDA to hear directly from those living with a condition about experiences, symptoms, treatments, and hopes for new therapies. Such insights are particularly important for frontotemporal degeneration (FTD), a genetically and clinically heterogenous condition.

Design/Methods On March 5, 2021, AFTD hosted an EL-PFDD for FTD; 382 caregivers, family members, and persons with FTD participated, plus 171 professionals. In preparation, AFTD and the FTD Disorders Registry conducted the FTD Insights Survey, gathering information on clinical and genetic characteristics of respondents; the earliest, current, and most concerning symptoms; and perspectives on treatments and clinical trials. 1,241 persons with FTD, care partners/caregivers, and family members in the US, Canada and UK had completed the survey by October 31, 2020.

Results People living with all FTD syndromes described challenges in activities of daily life, including preparing meals, managing finances, driving, and conversing. Symptoms vary within and across disorders, encompassing language, motor function, thinking/decision-making, interpersonal/emotional functioning, among others. Many people diagnosed have limited awareness of their symptoms. In genetic FTD, the path to diagnosis can be simpler, yet a lifetime of perceived or known risk can dramatically influence life decisions. The lack of effective, approved therapies – disease-modifying or symptomatic – was a universal concern. Non-prescription and off-label prescription therapies may stabilize symptoms temporarily while prescribed acetylcholinesterase inhibitors can exacerbate symptoms. Many people are willing to participate in clinical research depending on illness stage and burden imposed by trial design.

Conclusions The community of those affected by FTD offers valuable insights into this condition as partners in care and research.

Authors/Disclosures
Diana Wheaton PRESENTER	Diana Wheaton has received personal compensation for serving as an employee of FTD Disorders Registry. The institution of Diana Wheaton has received research support from Rainwater Charitable Foundation. The institution of Diana Wheaton has received research support from NIA.
	No disclosure on file
Shana Dodge (Association for Frontotemporal Degeneration)	Shana Dodge has received personal compensation for serving as an employee of The Association for Frontotemporal Degeneration (AFTD).
	No disclosure on file
	No disclosure on file
Stephanie Cosentino, PhD (Columbia University Medical Center)	Dr. Cosentino has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Association for Frontotemporal Dementia. Dr. Cosentino has received personal compensation in the range of $500-$4,999 for serving as a Consultant for SAGE Pharmaceuticals.
	No disclosure on file
Penny Dacks, PhD (AFTD)	Penny Dacks has received personal compensation for serving as an employee of The Association for Frontotemporal Degeneration.
	No disclosure on file

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