Division of Dockets Management (HFA-305)
Food and Drug Administration
5630 Fishers Lane, Rm. 1061
Rockville, MD 20852
Docket No. FDA-2012-N-0967 Patient focused drug development public meetings comments
Dear Sir/Madam:
We strongly urge you to consider adding frontotemporal lobar degeneration (FTD) to your list of disease areas
selected for the PDUFA public meetings held by the FDA to learn about disease impact on patient’s daily lives and
the treatment benefits that matter most to patients and families. FTD has been identified under NAPA as one of
the Alzheimer’s disease related dementias (ADRD) that is a national priority. FTD is a rare disease and encompasses
a spectrum of disorders that include behavioral variant FTD (bvFTD, also called Pick’s disease), the primary
progressive aphasias (PPA), progressive supranuclear palsy (PSP), cortical basal syndrome (CBS) and more recently,
FTD-ALS. Patients affected demonstrate pronounced behavioral changes, loss of cognitive skills, difficulty with
language and movement. FTD is a young onset disease, affecting people in their forties and fifties. Persons with
FTD live on average 10 years or less after diagnosis, and currently there are no approved therapeutics for FTD.




FTD is a progressive condition that severely impacts the patient’s ability to carry out activities of daily
living without caregiver support. FTD patients commonly lose their jobs and exhibit impulsive or
inappropriate behaviors. Some lose the ability to use language and communicate with others; some show
progressive deterioration of motor coordination and are eventually confined to a wheelchair.
Clinical trials for neurological diseases do not capture or address essential aspects of FTD such as lack of
empathy, loss of interest in activities or feelings for others – symptoms which significantly impact their
relationships with family and friends.
Currently there are no therapies for FTD, and only partial, symptomatic management using medications
approved for other diseases, none of which improve survival or allow patients to return to their former
role in their families or community or workplace.
FTD negatively impacts the children in the family. School age children can be faced with a parent whose
behavior creates an unsafe environment in the home. Young adult children in their twenties can become
caregivers, disrupting plans for their education and future. Financial impact of the disease is often
devastating on a young family.
FTD patients and their caregivers are a community that is engaged and willing to participate in activities that will
support more patient-centric therapeutics and clinical trial development. This past summer, an online FTD disease
impact survey created by the Association for Frontotemporal Degeneration (AFTD) received over 900 submissions
from caregivers and patients in just over 90 days. This year, NIH has generously funded two clinical research
networks for FTD in aid of gathering sufficient clinical data to support drug development and to create an
infrastructure that coordinates efforts among top academic medical centers across the US. Giving FTD patients,
caregivers and other stakeholders a voice at the FDA PDUFA public meetings of 2016-17 would serve to better
inform these efforts as well as those of the FDA in their regulatory review process.
Sincerely,
Name, City, State
(indicate role/title, for example: “Caregiver for my wife, diagnosed with bvFTD”)