Externally Led Patient Focused Drug Development Meeting: Speaker Questionnaire*
The Patient Focused Drug Development initiative aims to obtain the patient perceptive on specific diseases and their treatments. Please complete this questionnaire as it relates to the patient. Each section should be filled out from the perspective of the patient or the caregiver as
**Please fill out one form per patient.
you have more than one patient story to share please fill out multiple forms.
Speaker Demographic Information
Are you a:
Have you lost any loved ones to this disease?*
forms for each individual Krabbe disease patient.
At what age and in what year did they pass?
Are you affiliated with a nonprofit association in any way? If so, which organization and in what way are you involved?
Do you have a conflict of interest to report?
Conflict of interest may include any financial interest or relationship with a pharmaceutical company or any other healthcare related for profit entity.
Patient Demographic Information
At what age and in what year was the patient diagnosed with Krabbe disease?
Have you or your loved one undergone a transplant?
What was the age of the patient and date they were transplanted?
If Yes did the patient experience any complications from the transplant?
Patient Impact: Please complete from patient perspective (you) or from the Caregiver perspective ( your loved one).
Can you describe in detail you or your loved one's Krabbe disease symptoms?
Has Krabbe disease affected you or your loved one's ability to live independently? If so, how?
How has Krabbe disease affected you or your loved one's relationships?
How did Krabbe disease impact you or your loved one's ability to complete school/the school day? (Leave blank if not applicable)
How does Krabbe disease impact you or your loved one's activities outside of school or work? (Leave Blank if not applicable)
How has Krabbe disease affected you or your loved one's career choice or ability to have a job? (Leave blank if not applicable)
How has Krabbe disease affected you or your loved one's intimacy and/or reproductive health? (Leave blank if not applicable)
What is the impact of medical care on you or your loved one's daily life?
How else has Krabbe disease affected you or your loved one's life?
On a scale of 1-10, 10 being severely and 1 being no impact, how much has Krabbe disease impacted you or your loved one's life?
Please comment on the most significant way Krabbe disease has impacted you or your loved one's life.
What Impact does care coordination have on you or your loved one's daily life?
Caregiver Impact (Please complete only if you are a Caregiver of a patient with Krabbe disease)
How has caring for a patient with Krabbe disease impacted your daily life?
How has Krabbe disease affected you and your family financially?
How has Krabbe disease affected your career choice or ability to have a job?
How has caring for a patient with Krabbe disease affected your relationships?
How has Krabbe disease affected your intimacy and relationship with a partner?
What is the impact of supporting medical care for your loved one on your daily life?
How else has caring for a patient with Krabbe disease affected your life?
Patient Care: If your loved one is deceased please respond based on the care that was provided to help them live their best life
What is currently being done to treat you or your loved one's condition and its symptoms?
Please elaborate on how well the current treatment supports the ability for you or your loved one to live your best life:
How many physicians and medical experts take an actual role in you or your loved one's care and in what clinical fields (i.e. neurologists, pulmonologists, primary care, etc.?)
What assisted medical equipment or devices do you or your loved one use on a daily basis?
What are the most significant disadvantages or complications of your or your loved one's current treatments and how do they affect daily life?
Please elaborate on any other complications that you or your loved one has experienced from Krabbe disease or its treatment:
Have you or your loved one considered or undergone any of the following treatment options? (
Please check all that apply
Anticonvulsant medications to mange seizures
Medications to ease muscle spasticity and irritability
Physical and occupational therapies to minimize deterioration of muscle tone
Nutritional support, such as the use of a G tube (gastric tube), to deliver fluids & nutrients directly into the stomach or feeding pumps
Oxygen, pulse ox and respiratory support for breathing and heart rate monitoring
Nebulizer, cough assist equipment, shaky vests to keep secretions loose for respiratory support
Suction machines to alleviate secretions
Adaptive and assistive mobility devices such as Kid Karts, Standers, wheelchairs and positioning chairs for seating
Orthotics to mange spasticity and bone deformation
Positioning devices for sleeping
Short of a complete cure, what specific things would you look for in an ideal treatment for this condition?
Have you or loved one ever been enrolled in a Krabbe disease clinical trial, registry or other research?
Please provide any details on any clinical trials or registries you have participated in previously
If you had the opportunity to consider participating in a clinical trial studying experimental treatments, what things would you consider when deciding whether or not to participate?
Do you have the ability to participate in a virtual style meeting which will include a prerecorded video testimonial and a live Q&A session via virtual conferencing on October 29, 2020?
How did you hear about this PFDD meeting for Krabbe Disease?
The National Organization for Rare Disorders (NORD)
Partners for Krabbe Research
The Legacy of Angels Foundaton
United Leukodystrophy Foundation
If Other Please specify